Notifications that the funding would not be extended were relayed May 30 to researchers, who were told by officials that the had elected “to go with currently available approaches to eliminate HIV” instead.

The cuts will shutter two major HIV vaccine research efforts first funded by the NIH at the Duke Human Vaccine Institute and the Scripps Research Institute, scientists said. A Moderna spokesperson said the vaccine manufacturer’s through the NIH’s HIV Vaccine Trials Network have also been put on pause.

One senior NIH official said the HHS had instructed the agency not to issue any more funding in the next fiscal year for HIV vaccine research, with only a handful of exceptions. 

A budgetary rule change specifically targeted at HIV vaccine research is also expected to lead to another cut to the NIH’s awards for studies initiated by scientists, an official said.

The change, to be finalized shortly, inflates the accounting for the upfront cost of studies into HIV vaccines funded by the agency. Instead of the cost of a five-year grant being spread over five years, the NIH plans to make HIV vaccine dollars from multiyear grants all count toward a single year, the official said, making it harder for them to get funded.

A spokesperson for HHS told CBS News that “complex and duplicative health programs have resulted in serious duplication of efforts,” saying that “27 separate programs that address HIV/AIDS” had spent $7.5 billion.

“The Administration believes the United States should have the best medical research in the world. To that end, we are advancing policies to maximize the impact of every federal taxpayer dollar and ensure proper oversight of this funding,” HHS spokesperson Emily Hilliard said.

Hilliard claimed “critical HIV/AIDS programs will continue” under the new agency that Health and Human Services Secretary Robert F. Kennedy Jr. has proposed creating, dubbed the Administration for a Healthy America.

“For HIV vaccine design and development, we’ve begun to see light at the end of the tunnel after many years of research. This is a terrible time to cut it off. We’re beginning to get close. We’re getting good results out of clinical trials,” said , an immunology professor at Scripps Research.

Burton warned that his institution’s HIV vaccine research could not simply be turned back on, even if a future administration decided to change course on HIV funding. He said that ongoing experiments would be shuttered and that researchers assembled to study the issue would be forced to refocus their careers on other topics.

“This is a decision with consequences that will linger. This is a setback of probably a decade for HIV vaccine research,” Burton said.

The cancellation of the funds comes weeks ahead of the FDA’s for deciding on approval of lenacapavir, a twice-yearly injectable drug to prevent HIV.

The drug, which is being brought to the commercial market by drugmaker Gilead Sciences, builds on into earlier HIV medications. The drug’s availability could lead to a significant drop in HIV cases worldwide, since it was 100% effective in preventing transmission.

An NIH official, who was not authorized to speak publicly, rebuked the claim that the effectiveness of current HIV prevention strategies meant a vaccine was no longer needed. “The only way of ending the HIV epidemic in the U.S. and AIDS pandemic worldwide” is with a vaccine, the official said.

Developing an effective HIV vaccine has been an elusive target for researchers, though scientists have hailed recent breakthroughs in the field.

“HIV has established roadblocks to us fighting it off, which are unparalleled in vaccinology. We’ve had to learn what each of the roadblocks are and to devise ways to overcome it. This virus mutates so quickly,” said Duke professor of medicine Barton Ford Haynes, who is part of the Duke Human Vaccine Institute.

Haynes said his institute’s work was essentially combining different vaccines as part of a strategy to design an effective HIV vaccine.

He praised lenacapavir as a “wonderful development for the field” but said there was still a need for a vaccine. Lenacapavir requires injections every six months to remain effective, a challenging proposition even before steep cuts to the Centers for Disease Control and Prevention’s domestic HIV programs and U.S.-backed HIV/AIDS foreign aid programs.

“The hope was that adding an HIV vaccine to all the preventive measures that we have would finally allow us to end the pandemic,” Haynes said.

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Hayes is too sick to work, so she has spent much of the last four years sitting on her beige couch, often curled up under an electric blanket.

“My blood flow now sucks, so my hands and my feet are freezing. Even if I’m sweating, my toes are cold,” , who lives in Western Pennsylvania. She misses feeling well enough to play with her 9-year-old son or attend her 17-year-old son’s baseball games.

Along with claiming the lives of 1.2 million Americans, the covid-19 pandemic has been described as a . Hayes is one of millions of Americans who suffer from long covid. Depending on the patient, the condition can rob someone of energy, scramble the autonomic nervous system, or fog their memory, among many other symptoms.

In addition to the brain fog and chronic fatigue, Hayes’ constellation of symptoms includes frequent hives and migraines. Also, her tongue is constantly swollen and dry.

“I’ve had multiple doctors look at it and tell me they don’t know what’s going on,” Hayes said about her tongue. 

Estimates of prevalence range considerably, depending on how researchers define long covid in a given study, but the Centers for Disease Control and Prevention puts it at 17 million adults.

Despite long covid’s vast reach, the federal government’s investment in researching the disease — to the tune of $1.15 billion as of December — has so far failed to bring any new treatments to market. 

This disappoints and angers the patient community, who say the National Institutes of Health should focus on ways to stop their suffering instead of simply trying to understand why they’re suffering.

“It’s unconscionable that more than four years since this began, we still don’t have one FDA-approved drug,” said , executive director of the , a patient-led advocacy organization. Stone was among several people with long covid who spoke at a workshop hosted by the NIH in September where patients, clinicians, and researchers discussed their priorities and frustrations around the agency’s approach to long-covid research.

Some doctors and researchers are also critical of the agency’s research initiative, called RECOVER, or Researching COVID to Enhance Recovery. Without clinical trials, physicians specializing in treating long covid must rely on hunches to guide their clinical decisions, said , chief of research and development with the .

“What [RECOVER] lacks, really, is clarity of vision and clarity of purpose,” said Al-Aly, saying he agrees that the NIH has had enough time and money to produce more meaningful progress.

Now the NIH is starting to determine how to allocate an additional of funding for long-covid research, of which is earmarked for clinical trials. These funds will be allocated over the next four years.

At the end of October, RECOVER for clinical trial ideas that look at potential therapies, including medications, saying its goal is “to work rapidly, collaboratively, and transparently to advance treatments for Long COVID.”

This turn suggests the NIH has begun to respond to patients. This has stirred cautious optimism among those who say that the agency’s approach to long covid has lacked urgency in the search for effective treatments.

Stone calls this $300 million a down payment. She warns it’s going to take a lot more money to help people like Hayes regain some degree of health.

“There really is a burden to make up this lost time now,” Stone said.

The NIH told ºÚÁϳԹÏÍø News and NPR via email that it recognizes the urgency in finding treatments. But to do that, there needs to be an understanding of the biological mechanisms that are making people sick, which is difficult to do with post-infectious conditions.

That’s why it has funded research into how long covid affects , or trying to understand why people are afflicted with the condition.

Good Science Takes Time

In December 2020, for the NIH to launch RECOVER, raising hopes in the long-covid patient community.

Then-NIH Director explained that was to better understand long covid as a disease and that clinical trials of potential treatments would come later.

According to RECOVER’s website, it has funded to test the safety and effectiveness of an experimental treatment or intervention. Just one of those trials has .

On the other hand, RECOVER has supported more than 200 observational studies, such as research on how long covid and on which symptoms are . And the initiative has funded more than 40 pathobiology studies, which focus on the basic cellular and molecular mechanisms of long covid.

RECOVER’s this research has led to crucial insights on the risk factors for developing long covid and on understanding how the disease interacts with preexisting conditions.

It notes that observational studies are important in helping scientists to design and launch evidence-based clinical trials.

Good science takes time, said , the co-principal investigator for the RECOVER-Adult Observational Cohort at New York University. And long covid is an “exceedingly complicated” illness that appears to affect nearly every organ system, she said. 

This makes it more difficult to study than many other diseases. Because long covid harms the body in so many ways, with widely variable symptoms, it’s harder to identify precise targets for treatment.

“I also will remind you that we’re only three, four years into this pandemic for most people,” Horwitz said. “We’ve been spending much more money than this, yearly, for 30, 40 years on other conditions.”

NYU received of RECOVER funds in 2021, which the institution is using to spearhead the collection of data and biospecimens from up to 40,000 patients. Horwitz said nearly 30,000 are enrolled so far.

This , Horwitz said, supports ongoing observational research, allowing scientists to understand what is happening biologically to people who don’t recover after an initial infection — and that will help determine which clinical trials for treatments are worth undertaking.

“Simply trying treatments because they are available without any evidence about whether or why they may be effective reduces the likelihood of successful trials and may put patients at risk of harm,” she said.

Delayed Hopes or Incremental Progress?

The NIH told ºÚÁϳԹÏÍø News and NPR that patients and caregivers have been central to RECOVER from the beginning, “playing critical roles in designing studies and clinical trials, responding to surveys, serving on governance and publication groups, and guiding the initiative.”

But the consensus from patient advocacy groups is that RECOVER should have done more to prioritize clinical trials from the outset. Patients also say RECOVER leadership ignored their priorities and experiences when determining which studies to fund.

RECOVER has scored some gains, said , co-director of . This includes findings on differences in long covid between adults and kids.

But Davids said the NIH shouldn’t have named the initiative “RECOVER,” since it wasn’t designed as a streamlined effort to develop treatments.

“The name’s a little cruel and misleading,” he said.

RECOVER’s initial allocation of $1.15 billion probably wasn’t enough to develop a new medication to treat long covid, said co-director of the University of Pennsylvania’s .

But, he said,  the results of preliminary clinical trials could have spurred pharmaceutical companies to fund more studies on drug development and test how existing drugs influence a patient’s immune response.

Emanuel is one of the authors of a March 2022 covid . He notes that RECOVER’s lack of focus on new treatments was a problem. “Only 15% of the budget is for clinical studies. That is a failure in itself — a failure of having the right priorities,” he told ºÚÁϳԹÏÍø News and NPR via email.

And though the NYU biobank has been impactful, Emanuel said there needs to be more focus on how existing drugs influence immune response.

He said some clinical trials that RECOVER has funded are “ridiculous,” because they’ve focused on symptom amelioration, for example to of over-the-counter medication to improve sleep. Other studies looked at non-pharmacological interventions, such as exercise and “” to help with cognitive fog.

People with long covid say this type of clinical research contributes to what many describe as the “gaslighting” they experience from doctors, who sometimes blame a patient’s symptoms on anxiety or depression, rather than acknowledging long covid as a real illness with a physiological basis.

“I’m just disgusted,” said long-covid patient Hayes. “You wouldn’t tell somebody with diabetes to breathe through it.”

, director and founder of the , said she’s even taken breaks from seeking treatment after getting fed up with being told that her symptoms were due to her diet or mental health.

“You’re at the whim of somebody who may not even understand the spectrum of long covid,” Sweeney said.

Insurance Battles Over Experimental Treatments

Since there are still no long-covid treatments approved by the Food and Drug Administration, anything a physician prescribes is classified as either experimental — for unproven treatments — or an off-label use of a drug approved for other conditions. This means patients can struggle to get insurance to cover prescriptions.

, medical director for — said he writes many appeal letters. And some people pay for their own treatment.

For example, intravenous immunoglobulin therapy, low-dose naltrexone, and hyperbaric oxygen therapy are all promising treatments, he said.

For hyperbaric oxygen, , randomized show improvements for the chronic fatigue and brain fog that often plague long-covid patients. The theory is that higher oxygen concentration and increased air pressure can help heal tissues that were damaged during a covid infection.

However, the out-of-pocket cost for a series of sessions in a hyperbaric chamber can run as much as $8,000, Brode said.

“Am I going to look a patient in the eye and say, ‘You need to spend that money for an unproven treatment’?” he said. “I don’t want to hype up a treatment that is still experimental. But I also don’t want to hide it.”

There’s a host of pharmaceuticals that have promising off-label uses for long covid, said microbiologist , president and chief scientific officer at the Massachusetts-based . For instance, she’s collaborating on a clinical study that repurposes two HIV drugs to treat long covid.

Proal said research on treatments can move forward based on what’s already understood about the disease. For instance, she said that scientists — partly due to — that some patients small amounts of viral material after a covid infection. She has not received RECOVER funds but is researching antivirals.

But to vet a range of possible treatments for the millions suffering now — and to develop new drugs specifically targeting long covid — clinical trials are needed. And that requires money.

Hayes said she would definitely volunteer for an experimental drug trial. For now, though, “in order to not be absolutely miserable,” she said she focuses on what she can do, like having dinner with her family.

At the same time, Hayes doesn’t want to spend the rest of her life on a beige couch. 

RECOVER’s deadline to submit research proposals for potential long-covid treatments is .

This article is from a partnership that includes and ºÚÁϳԹÏÍø News.

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“Right To Try” experimental drug program saved “thousands and thousands of lives”

Former President Donald Trump on Aug. 30

Former President Donald Trump has boasted in recent months about “Right To Try,” a law he signed in 2018. It’s aimed at boosting terminally ill patients’ access to potentially lifesaving medications not yet approved by the Food and Drug Administration.

“We have things to fight off diseases that will not be approved for another five or six years that people that are very sick, terminally ill, should be able to use. But there was no mechanism for doing it,” Trump , speaking in Washington, D.C., to supporters of the conservative parental rights advocacy group Moms for Liberty.

He also said that because of Right To Try, “we have saved thousands and thousands of lives.”

Trump similarly praised the program during an in Pennsylvania, in a with a conservative commentator, and during his Republican National Convention : “Right To Try is a big deal,” Trump said then.

Medical experts who’ve studied the experimental treatment program, however, say there’s no evidence to support Trump’s claims. These experts say Right To Try weakened regulations intended to protect patients.

What Is Right To Try?

The , aka Right To Try, passed Congress on a bipartisan basis and was signed into law in 2018. It sought to streamline the process for getting potentially lifesaving drugs that weren’t yet FDA-approved to terminally ill patients. The speed matters; industry groups say it takes on average for a new medicine to reach pharmacy shelves.

However, a similar FDA program, the pipeline, sometimes called “compassionate use,” has existed , and became law in 1987.

And that is the root of many criticisms of Right To Try.

“Right To Try is basically ‘expanded access light,’” said Alison Bateman-House, a medical ethicist who researches access to investigational medical products at New York University’s Grossman School of Medicine.

Right To Try caters to fewer patients than expanded access and offers them fewer treatments, Bateman-House said.

Easing Access or Erasing Safeguards?

Patients must meet specific, but different, criteria to qualify for either experimental medication program.

To qualify for expanded use, patients must have a “serious or immediately life-threatening disease or condition” for which there is no “comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the disease or condition,” according to . Clinical trials must be infeasible for the patients, and the use of these drugs must not interfere with any in-progress studies. Also, the potential benefits must justify the risks, according to the prescribing physicians.

Then, after identifying a treatment, the patient’s doctor must receive approval from its manufacturer, the FDA, and the institutional review board overseeing the medication’s clinical trials.

The FDA said these steps exist so the agency can “fairly weigh the risks and benefits” of the medication and protect the patient’s safety. The agency also collects data about the drugs’ clinical impact on the patient and any adverse effects to inform the wider approval process for the drug.

Right To Try sought to hasten this approval process. Under the new program, for instance, a doctor must merely identify an experimental medication and receive authorization to use it from the manufacturer. In most cases, the FDA has no authority to approve or deny the application, and there’s no review board process to navigate.

But, because of the Right To Try program’s definitions, fewer patients and fewer medicines qualify.

Under Right To Try, patients must have a “life-threatening” disease or condition, not just “serious,” as with expanded access. Experimental medications are available only after they’ve completed Phase 1 clinical trials; treatments accessed through the expanded access program can be administered during a Phase 1 study.

Right To Try, which includes liability protections for manufacturers and prescribing physicians, also weakens requirements that govern how doctors disclose experimental medications’ risks to patients, leaving informed consent undefined. And it prevents the FDA from using information about how patients tolerate the drugs to “delay or adversely affect the review or approval of such drug(s),” unless top officials justify the benefit to public health in writing.

Supporters say Right To Try is an example of successful deregulation and claim that its more efficient approval process saved lives. But critics see this as a key reason for concern, because it “opens up the opportunity of exploiting desperate patients,” said Holly Fernandez Lynch, a bioethicist who studies pharmaceutical policy at the University of Pennsylvania’s Perelman School of Medicine.

Government data shows regulatory agencies weren’t the main hurdle patients faced when seeking experimental drugs. The FDA almost always approved expanded access applications, and quickly by government standards.

According to a 2018 FDA report on the expanded access program, the FDA authorized 99% of the roughly 9,000 requests it received in the previous five years, approving emergency requests for experimental medications in less than one day on average. More shows that approval trend has continued, even as the number of applications has grown each year.

In rare cases in which the FDA didn’t automatically approve requests, regulators often didn’t deny them, but recommended tweaks to the requested dosage to address safety and effectiveness concerns.

Right To Try by the Numbers

The FDA does not share detailed information about the number of doses provided or patients treated under Right To Try. Instead, it posts only an showing how many drugs have been approved under the program. The agency says that since Right To Try began in 2018 it has approved 16 treatments: 12 from 2018 to 2022 and four last year.

The FDA declined to provide additional information about the number of Right To Try requests or approvals.

Although the 16 medications approved through Right To Try were possibly provided to more than one patient each, experts said it’s extremely unlikely thousands of patients were involved, as Trump said.

Trump’s claim represents an “egregious overestimate of the number of people who are using Right To Try,” said Fernandez Lynch, noting she believes the real numbers are “very, very low.”

The Trump campaign did not respond to multiple inquiries about the source of the former president’s statistics. Karoline Leavitt, the campaign’s national press secretary, told ºÚÁϳԹÏÍø News that in a second term “President Trump will of course remain open to other pathways to expand ‘Right to Try’ to save more American lives.”

It remains unclear how Trump might expand the program, though the conservative Goldwater Institute is advocating for “,” which it claims will let patients receive individualized therapeutics.

Experts noted such drugs are already accessible through the expanded access program.

Meanwhile, evidence shows that the high price of experimental treatments, which are sometimes available through certain drug company programs but not typically covered by insurance, is a greater hurdle to patients than regulatory guardrails are.

“I don’t think that people are having a problem with the FDA blocking access to individualized therapeutics,” Bateman-House said. “I think the problem is that individualized therapeutics are incredibly expensive, and there’s only a very small number of researchers in the country who know how to make them.”

Our Ruling

Trump has claimed throughout the campaign that his Right To Try program is novel and has saved thousands of lives. But a similar program has existed for decades, and there is no evidence Right To Try has had anywhere close to the impact Trump said it has had.

Neither the Trump campaign nor Right To Try advocates provided evidence to back claims of widespread benefit. And government data shows only 16 medications have been approved under the program in its first six years, with no accounting of how many patients used those medications or their clinical outcomes.

Moreover, public health experts have said Right To Try weakens patient protections and fails to address the true barriers to experimental medications.

We rate Trump’s claim False.

our sources:

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To bring a new drug to market, the FDA requires pharmaceutical firms to perform extensive studies to demonstrate safety and efficacy, which are often expensive and time-consuming to conduct to the agency’s specifications. Getting a drug to market a few months sooner and for less expense than usual can translate into millions in profit for the manufacturer.

That is why a private equity-backed startup like Headlands Research saw an opportunity in creating a network of clinical sites and wringing greater efficiency out of businesses, to perform this critical scientific work faster. And why Moderna, Pfizer, Biogen, and other drug industry bigwigs have been willing to hire it — even though it’s a relatively new player in the field, formed in 2018 by investment giant KKR.

In July 2020, Headlands it won coveted contracts to run clinical trials of covid-19 vaccines, which would include shots for AstraZeneca, Johnson & Johnson, Moderna, and Pfizer.

In marketing its services, Headlands described its mission to “profoundly impact” clinical trials — including boosting participation among racial and ethnic minorities who have long been underrepresented in such research.

“We are excited,” CEO Mark Blumling said in a statement, to bring “COVID-19 studies to the ethnically diverse populations represented at our sites.” Blumling, a drug industry veteran with venture capital and private equity experience, told KHN that KKR backed him to start the company, which has grown by buying established trial sites and opening new ones.

Finding and enrolling patients is often the limiting and most costly part of trials, said Dr. Marcella Alsan, a public policy professor at Harvard Kennedy School and an expert on diverse representation in clinical trials, which have a median cost of $19 million for new drugs, .

Before covid hit, Headlands acquired research centers in McAllen, Texas; Houston; metro Atlanta; and Lake Charles, Louisiana, saying those locations would help it boost recruitment of diverse patients — an urgent priority during the pandemic in studying vaccines to ward off a disease disproportionately killing Black, Hispanic, and Native Americans.

Headlands’ sites also ran, among other things, clinical studies on treatments to combat Type 2 diabetes, postpartum depression, asthma, liver disease, migraines, and endometriosis, according to a review of website archives and the federal website ClinicalTrials.gov. But within two years, some of Headlands’ alluring promises would fall flat.

In September, Headlands shuttered locations in Houston — one of the nation’s largest metro areas and home to major medical centers and research universities — and Lake Charles, a move Blumling attributed to problems finding “experienced, highly qualified staff” to carry out the complex and highly specialized work of clinical research. The McAllen site is not taking on new research as Headlands shifts operations to another South Texas location it launched with Pfizer.

What impact did those sites have? Blumling declined to provide specifics on whether enrollment targets for covid vaccine trials, including by race and ethnicity, were met for those locations, citing confidentiality. He noted that for any given trial, data is aggregated across all sites and the drug company sponsoring it is the only entity that has seen the data for each site once the trial is completed.

A fragmented clinical trials industry has made it a prime target for private equity, which often consolidates markets by merging companies. But Headlands’ trajectory shows the potential risks of trying to combine independent sites and squeeze efficiency out of studies that will affect the health of millions.

, a health economist at Johns Hopkins who has studied private equity acquisitions of physician practices, said consolidation has potential downsides. Singh and her colleagues in September analyzing acquisitions in dermatology, gastroenterology, and ophthalmology that found physician practices — a business with parallels to clinical trial companies — charged higher prices after acquisition.

“We’ve seen reduced market competition in a variety of settings to be associated with increases in prices, reduction in access and choice for patients, and so on,” Singh said. “So it’s a delicate balance.”

, a professor of medicine at Harvard Medical School, called private equity involvement in trials “concerning.”

“We need to make sure that patients” know enough to provide “adequate, informed consent,” he said, and ensure “protections about the privacy of the data.”

“We don’t want those kinds of things to be lost in the shuffle in the goals of making money,” he said.

Blumling said trial sites Headlands acquired are not charging higher prices than before. He said privacy “is one of our highest concerns. Headlands holds itself to the highest standard.”

Good or bad, clinical trials have become a big, profitable business in the private equity sphere, data shows.

Eleven of the 25 private equity firms identified by industry tracker PitchBook as the top investors in health care have bought stakes in clinical research companies, a KHN analysis found. Those companies have been involved in studies ranging from to treatments for ovarian cancer, Parkinson’s disease, and Alzheimer’s.

Contracted firms also analyze patient data and prepare materials to secure approval from regulatory agencies, in hopes of getting more drugs to market faster. And a big draw for investors: Clinical research companies make money whether or not a drug succeeds, making it less risky than investing in a drug company.

The number of clinical trials has exploded to more than 434,000 this year as of late November, more than triple the number a decade ago.

Still, most trial sites are physician practices that don’t consistently perform studies, by Boston-based investment firm Provident Healthcare Partners.

“Independent sites are being purchased by private equity, and they’re moving into larger site groups of 30, 40, and then their game plan is to roll that up into a business and then sell it again,” said Linda Moore Schipani, CEO of Clinical Research Associates, a Nashville-based company that worked on covid vaccine trials for AstraZeneca, Novavax, and Pfizer. “That’s kind of the endgame.”

Headlands is a prime example. in November 2019 that it would acquire six centers in the U.S. and Canada, including three sites in Texas and Louisiana owned by Centex Studies that would help improve participation among Hispanics and African Americans.

It has made other acquisitions since then and opened new sites in areas with “extremely limited trial options,” something Blumling says distinguishes his company.

“I’m not an evangelist for private equity,” Blumling said. “The ability of KKR to be willing to invest in something that is a three- to five-year return versus a one- to two-year return is something that you won’t see out there.”

A research center in Brownsville, Texas — a stone’s throw from the U.S.-Mexico border and where 95% of the population is Hispanic or Latino — is one of several where it is partnering with Pfizer to boost patient diversity.

To recruit patients, Headlands “is really going beyond what a lot of sites do, which is social media,” Blumling said in an interview. “It’s going within churches, community fairs, really getting out into as much as possible the broader community.”

Headlands closed the Houston and Lake Charles sites because of staffing issues, Blumling said, and finished or moved their studies elsewhere. Blumling said the decision to close those locations “did not have anything to do with the speed of trials.”

Similarly, he said, Headlands is moving the McAllen site’s operations to Brownsville “because it had a larger population of trained personnel.”

“We want to continue to grow sites and do great work,” Blumling said. “If we can’t find the people in order to do that at the quality that we demand, which is at the highest level, then it doesn’t make sense to keep those sites.”

‘The Writing to Me Was on the Wall’

In 2006, Devora Torrence co-founded Centex Studies, which she described as “my little mom and pop business” in a about female entrepreneurs in science. She said a flurry of interest from private equity came at the end of 2018. The appeal was evident: Drug companies were relying on bigger clinical trial networks.

“The thing is speed, getting it to market. With a bigger network, you get that speed,” Torrence said on the podcast. “The writing to me was on the wall that either I get some outside investment and scale up myself, or kind of listen to these guys and see if maybe now would be the right time to exit.”

Joining Headlands had its benefits during the pandemic because she could “lean on” its other sites with experience running vaccine trials. “Had we not gotten those … we may not still be here,” Torrence said.

Torrence, whose LinkedIn profile said she left the company in 2021, didn’t respond to messages from KHN.

Lyndon Fullen, a health care consultant and former Centex employee, said private equity provides funding that allows companies to add study sites.

“I completely support it,” he said. “If it’s about reaching that large patient population, it’s of course better to have larger groups with that funding.”

Opportunity in Long Covid

Contract research organization Parexel saw opportunity in the covid pandemic — millions of people were developing long covid after infection and there were few, if any, meaningful treatment options.

The company, which employs more than 19,000 people, was acquired in 2021 by EQT Private Equity and Goldman Sachs’ private equity arm , billions more than the $4.5 billion that private equity firm when it took Parexel private in 2017.

A growing body of research shows the debilitating effects of long covid, including a of tens of thousands of patients in Scotland where nearly half had not fully recovered months later. But treatments addressing its root causes could be years away. “It’s a huge number of people,” said Dr. Nathalie Sohier, who leads Parexel’s infectious diseases and vaccines franchise. “There’s a lot of need.”

Long covid represents the promise and peril of the work to develop new drugs: Millions of patients create a potentially lucrative market for drug companies, and yet researchers and industry experts say they are reluctant to jump in. In part, that’s because “it’s not a well-defined disease, and that really makes it highly risky for companies to invest in research,” said Cecil Nick, a vice president for Parexel.

“How are we going to be able to tell the FDA that our drug works? We can’t count the number of people who died; we can’t count the number of people in the hospital,” said , a University of California-San Francisco professor who is running an observational study on long covid patients.

As of August, among more than 4,400 covid studies, only 304 focused on long covid. A third of those were related to drug development, Sohier said.

Sohier said “there are few” companies in its long covid program. That hasn’t stopped Parexel from as the ideal partner to shepherd new products, including by doing regulatory work and using remote technology to retain patients in trials. Parexel has worked on nearly 300 covid-related studies in more than 50 countries, spokesperson Danaka Williams said.

Michael Fenne, research and campaign coordinator with the Private Equity Stakeholder Project, which studies private equity investments, said Parexel and other contract research organizations are beefing up their data capacity. The aim? To have better information on patients.

“It kind of ties into access and control of patients,” Fenne said. “Technology makes accessing patients, and then also having more reliable information on them, easier.”

KHN senior correspondent Fred Schulte and Megan Kalata contributed to this report.

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There were no drugs against covid-19, and no way to predict which infected patients would develop pneumonia or fall into an inflammatory tailspin leading to severe illness or death. In desperation, Vinetz and countless other doctor-scientists trawled the literature for existing medicines that might help.

“We were in the hospital. We had nothing,” Vinetz said. “I was one of tens of thousands of doctors around the world who said, ‘We gotta figure out what to do.’”

On April 16, 2020, Vinetz saw an about a drug called camostat, licensed in to treat inflammation of the pancreas. Research during the first SARS epidemic, in 2004, had shown the drug had a plausible biochemical mechanism for slowing coronavirus infections, so Vinetz and his colleagues quickly organized a small clinical trial on outpatients with mild to moderate symptoms.

In those days, before covid vaccines and covid-specific treatments appeared on the market, Vinetz’s experiment was one of thousands conducted by doctors who hoped older vaccines and drugs, usually cheap and off-patent, might provide them with options.

Mostly, the drugs were too toxic or had no clear effect. Of the trials for potential covid drugs listed on the website of the National Institutes of Health — including antivirals, anti-inflammatories, and drugs used for cancer, asthma, heart disease, and dozens of other conditions — few have produced helpful medicines.

In fact, only one older drug is routinely used to fight covid. That’s the steroid dexamethasone, to help keep hospitalized patients from requiring supplemental oxygen or intubation.

Drugs like hydroxychloroquine and ivermectin showed hints of value initially but failed in clinical trials — only to remain in circulation, at least partly because their use symbolized affinity in the culture war for some of President Donald Trump’s followers.

A few old drugs still show promise, but they’ve had trouble getting traction. The ivermectin and hydroxychloroquine fiascoes soured doctors on repurposed medications, and the pharmaceutical industry has shown little interest in testing them, especially when it can earn billions from even mediocre new ones, scientists tracking the field say.

American and European scientists have confirmed the theoretical basis for camostat’s impact on covid. But evidence for its effects is weak; last year the from a big NIH trial comparing various treatments.

A more promising story emerged with fluvoxamine, licensed under the brand name Luvox in 1994 to treat obsessive-compulsive disorder. The drug is in the same class as common antidepressants such as Prozac, Lexapro, and Zoloft.

A child psychiatrist noticed fluvoxamine might be good for covid. In March 2020, while recovering from a bout of covid, Dr. Angela Reiersen of Washington University in St. Louis saw a 2019 study in mice that showed how fluvoxamine could activate a protein similar to one with Wolfram syndrome, a genetic disease that causes diabetes, neurological issues, and, eventually, death.

Reiersen and her colleague Dr. Eric Lenze, a geriatric psychiatrist, began a in people with symptoms of covid. Of the 80 in the fluvoxamine group, none suffered a serious decline, while six of 72 patients given sugar pills got pneumonia, and four were hospitalized.

In a follow-up in Brazil, people who took at least 80% of their fluvoxamine pills were 66% less likely to require emergency care or hospitalization than those who got sugar pills. Only one died, compared with 11 in the placebo group.

Since October, when the Brazilian study was published, fluvoxamine’s future has dimmed. Neither the NIH nor the recommends fluvoxamine to prevent respiratory distress. The NIH panelists in the Brazilian trial were only statistically significant among those who remained in the trial. (Because of nausea and other side effects, only 74% of trial participants in the fluvoxamine wing took all their pills, compared with 82% in the placebo wing.)

The NIH panel also was put off by the fact that the Brazilian trial counted hospitalizations as well as people put under a doctor’s care for six hours or more — not a standard measure. Trial organizers said that was necessary because Brazilian hospitals were so packed with covid patients that many people got their care in makeshift outdoor shelters.

Regulators and experts are awaiting results from two other big trials, one of universities and hospitals, . But both studies are using doses of 100 milligrams of fluvoxamine a day, compared with 200 or 300 milligrams in the successful trials.

“I have concerns that they are not using a high-enough dose,” Reiersen said, given that fluvoxamine operates on a different biochemical pathway to fight covid than the one involved in psychiatric treatment.

The concern is shared by Craig Rayner, a former drug company scientist who worked on the Brazilian trial and other big tests of repurposed drugs. “You can do the largest, most well-funded study in the world,” he said, “but if you choose the wrong dose, it’s rubbish in, rubbish out.”

The team overseeing NIH’s trial opted for a lower dose because higher doses had already been used in the earlier trials — and often caused side effects, said Sarah Dunsmore, a program director at NIH’s National Center for Advancing Translational Sciences.

On Dec. 21, David Boulware, a University of Minnesota infectious-disease expert, petitioned the FDA to approve a change in fluvoxamine’s label stating it can be used to prevent respiratory distress in at-risk patients with mild to moderate covid. He hasn’t received a response yet.

It’s a different story for big drug companies. Two days after Boulware’s submission, to market its drug molnupiravir, which in its clinical trial showed about as much effectiveness as fluvoxamine, and also had side effects like nausea and dizziness. Fluvoxamine also can cause insomnia and anxiety; molnupiravir is , male or female, having unprotected sex, because it caused genetic and fetal damage in test animals.

Still, federal guidelines in certain settings, and the government has bought for about $2.2 billion, or $733 per dose. Fluvoxamine, a generic, goes for less than $5 a pill.

“You hate to say that Big Pharma has a lot of influence, but clearly they do,” Boulware said. “The molnupiravir data was not that great, but we’re spending billions on the drug and it got fast-track emergency use authorization” while fluvoxamine remains in a gray area.

With the arrival of effective vaccines and the trickle of antiviral treatments, the urgency of rehabilitating old drugs for U.S. patients has ebbed. But the need remains high in lower- and middle-income countries where vaccines and new covid treatments remain unavailable.

It’s not rare for a pharmaceutical company to synthesize or study a drug for one purpose, only to discover it works better for something else. The classic instance is sildenafil, or Viagra, which was being developed as a drug for hypertension when scientists noticed a remarkable side effect. Remdesivir, now a front-line drug against covid, was aimed at treating Ebola.

It’s less common for a drug marketed for one use to acquire an entirely different purpose, but the pandemic drove scientists to try. They tested thousands of compounds in petri dishes for their virus-killing power, but the journey from test tube to human remedy is long, said Rayner, who is also a professor of pharmaceutical sciences at Monash University in Melbourne, Australia.

If fluvoxamine were a new drug, the company sponsoring it would have spent the money needed to get the drug approved and to show the FDA it has the means to monitor the drug’s safety and efficacy. Since it’s an old drug, it will be up to independent scientists, or perhaps a reluctant generics manufacturer, to sponsor safety monitoring should the FDA provide an emergency use authorization, Rayner said.

An EUA or approval “comes with strings. You have to continue to monitor the safety, to make sure no signals pop up when you move it from thousands to millions of patients,” he said. “That’s very expensive.”

U.S. physicians can prescribe drugs off label, but most are leery of doing so until a drug has won approval for the new use. That’s especially true now.

Definitive answers on some repurposed drugs were slow in coming because there were too many small, poorly designed studies by “every man and his dog,” Rayner said. He calculates up to $5.6 billion has been wasted on hydroxychloroquine clinical trials alone.

A recent called for better coordination and information-sharing among those organizing trials so that definitive answers can be obtained quickly with big pots of data.

As for camostat, Vinetz said those who took the drug felt better than those who got a placebo. “It basically prevented loss of smell and taste, which people really bitterly care about,” he said. “That means there’s a real biological effect. That merits further exploration.”

But will that happen? Vinetz’s team has sought publication of their research for five months with no success. He’d like to see whether camostat can prevent long covid, but such investigations cost millions. Camostat’s Japanese manufacturer as a covid drug after its own small, unsuccessful trial.

“When there’s no profit motive, it’s tough,” Vinetz said. Meanwhile, he’s resumed his research into controlling a neglected tropical disease: .

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Even as the war in Ukraine has prompted an exodus of international companies — from fast-food chains and oil producers to luxury retailers — from Russia, U.S. and global drug companies said they would continue manufacturing and selling their products there.

Airlines, automakers, banks, and technology giants — at least 320 companies — are among the businesses curtailing operations or making high-profile exits from Russia as its invasion of Ukraine intensifies. McDonald’s, Starbucks, and Coca-Cola announced a pause in sales this week.

But drugmakers, medical device manufacturers, and health care companies, which are exempted from U.S. and European sanctions, said Russians need access to medicines and medical equipment and contend that international humanitarian law requires they keep supply chains open.

“As a health care company, we have an important purpose, which is why at this time we continue to serve people in all countries in which we operate who depend on us for essential products, some life-sustaining,” said Scott Stoffel, divisional vice president for Illinois-based Abbott Laboratories, which manufactures and sells medicines in Russia for oncology, women’s health, pancreatic insufficiency, and liver health.

Johnson & Johnson — which has corporate offices in Moscow, Novosibirsk, St. Petersburg, and Yekaterinburg — said in a statement, “We remain committed to providing essential health products to those in need in Ukraine, Russia, and the region, in compliance with current sanctions and while adapting to the rapidly changing situation on the ground.”

The reluctance of drugmakers to pause operations in Russia is being met with a growing chorus of criticism.

Pharmaceutical companies that say they must continue to manufacture drugs in Russia for humanitarian reasons are “being misguided at best, cynical in the medium case, and outright deplorably misleading and deceptive,” said , a professor at the Yale School of Management who is have curtailed operations in Russia. He noted that banks and technology companies also provide essential services.

“Russians are put in a tragic position of unearned suffering. If we continue to make life palatable for them, then we are continuing to support the regime,” Sonnenfeld said. “These drug companies will be seen as complicit with the most vicious operation on the planet. Instead of protecting life, they are going to be seen as destroying life. The goal here is to show that Putin is not in control of all sectors of the economy.”

U.S. pharmaceutical and medical companies have operated in Russia for decades, and many ramped up operations after Russia invaded and annexed Crimea in 2014, navigating the fraught relationship between the U.S. and Russia amid sanctions. In 2010, Vladimir Putin, then Russian prime minister, announced an ambitious national plan for the Russian pharmaceutical industry that would be a pillar in his efforts to reestablish his country as an influential superpower and wean the country off Western pharmaceutical imports. Under the plan, called “Pharma-2020” and “Pharma-2030,” the government required Western pharmaceutical companies eager to sell to Russia’s growing middle class to locate production inside the country.

Pfizer, Johnson & Johnson, Novartis, and Abbott are among the drugmakers that manufacture pharmaceutical drugs at facilities in St. Petersburg and elsewhere in the country and typically sell those drugs as branded generics or under Russian brands.

Pfizer’s CEO, Albert Bourla, said on CBS that the giant drugmaker is not going to make further investments in Russia, but that it will not cut ties with Russia, as multinational companies in other industries are doing.

Pharmaceutical manufacturing plants in Kaluga, a major manufacturing center for Volkswagen and Volvo southwest of Moscow, have been funded through a partnership between Rusnano, a state-owned venture that promotes the development of high-tech enterprises, and U.S. venture capital firms.

Russia also has sought to position itself as an attractive research market, offering an inexpensive and lax regulatory environment for clinical drug trials. Last year, Pfizer conducted in Russia clinical trials of Paxlovid, its experimental antiviral pill to treat covid-19. Before the invasion began in late February, 3,072 trials were underway in Russia and 503 were underway in Ukraine, according to BioWorld, a reporting hub focused on drug development that features data from Cortellis.

AstraZeneca is the top sponsor of clinical trials in Russia, with 49 trials, followed by a subsidiary of Merck, with 48 trials.

So far, drugmakers’ response to the Ukraine invasion has largely centered on public pledges to donate essential medicines and vaccines to Ukrainian patients and refugees. They’ve also made general comments about the need to keep open the supply of medicines flowing within Russia.

$2 million to support humanitarian efforts in Ukraine, and Pfizer, based in New York, said it has supplied $1 million in humanitarian grants. Swiss drug maker Novartis said it was expanding humanitarian efforts in Ukraine and working to “ensure the continued supply of our medicines in Ukraine.”

But no major pharmaceutical or medical device maker has announced plans to shutter manufacturing plants or halt sales inside Russia.

In , hundreds of leaders of mainly smaller biotechnology companies have called on industry members to cease business activities in Russia, including “investment in Russian companies and new investment within the borders of Russia,” and to halt trade and collaboration with Russian companies, except for supplying food and medicines. How many of the signatories have business operations in Russia was unclear.

, director for market access at Janssen, a Johnson & Johnson company, was among those who signed the letter, but whether he was speaking for the company was unclear. In its own statement , the company said it’s “committed to providing access to our essential medical products in the countries where we operate, in compliance with current international sanctions.”

GlaxoSmithKline, headquartered in the United Kingdom, said that it’s stopping all advertising in Russia and will not enter into contracts that “directly support the Russian administration or military.” But the company said that as a “supplier of needed medicines, vaccines and everyday health products, we have a responsibility to do all we can to make them available. For this reason, we will continue to supply our products to the people of Russia, while we can.”

Nell Minow, vice chair of ValueEdge Advisors, an investment consulting firm, noted that drug companies have been treated differently than other industries during previous global conflicts. For example, some corporate ethicists advised against pharmaceutical companies’ total divestment from South Africa’s apartheid regime to ensure essential medicines flowed to the country.

“There is a difference between a hamburger and a pill,” Minow said. Companies should strongly condemn Russia’s actions, she said, but unless the U.S. enters directly into a war with Russia, companies that make essential medicines and health care products should continue to operate. Before U.S. involvement in World War II, she added, there were “some American companies that did business with Germany until the last minute.”

KHN senior correspondent Arthur Allen contributed to this article.

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But you’d be wrong.

Opinions about Aduhelm (also known as aducanumab) in the dementia community are diverse, ranging from “we want the government to cover this drug” to “we’re concerned about this medication and think it should be studied further.”

The Alzheimer’s Association and UsAgainstAlzheimer’s, the most influential advocacy organizations in the field, are in the former camp.

Both are pushing for Medicare to cover Aduhelm’s $28,000 annual cost and fiercely oppose the Centers for Medicare & Medicaid Services’ to restrict coverage only to people enrolled in clinical trials. Nearly were received on that proposal, and a final decision is expected in April.

“With respect, we have no more time for debate or delay,” the Alzheimer’s Association national Early-Stage Advisory Group wrote in a Feb. 10 comment. “Every passing day without access to potential treatments subjects us to a future of irreversible decline.” For its part, UsAgainstAlzheimer’s called CMS’ proposal “anti-patient.”

Yet the scientific evidence behind Aduhelm is inconclusive, its efficacy in preventing the progression of Alzheimer’s remains unproved, and there are concerns about its safety. The FDA to the medication last June but ordered the drugmaker, Biogen, to conduct a new clinical trial to verify its benefit. And the agency’s decision came despite a 10-0 recommendation against doing so from its scientific advisory committee. (One committee member abstained, citing uncertainty.)

Other organizations representing people living with dementia are more cautious, calling for more research about Aduhelm’s effectiveness and potential side effects. More than 40% of people who take the medication have swelling or bleeding in the brain — complications that need to be carefully monitored.

The Dementia Action Alliance, which supports people living with dementia, is among them. In a statement forwarded to me by CEO Karen Love, the organization said, “DAA strongly supports CMS’s decision to limit access to aducanumab to people enrolled in qualifying clinical trials in order to better study aducanumab’s efficacy and adverse effects.”

Meanwhile, Dementia Alliance International — the world’s largest organization run by and for people with dementia, with more than 5,000 members — has not taken a position on Aduhelm. “We felt that coming out with a statement on one side or another would split our organization,” said Diana Blackwelder, its treasurer, who lives in Washington, D.C.

Blackwelder, 60, who was diagnosed with early-onset Alzheimer’s in 2017, told me, “To say that millions of people afflicted with a disease are all up in arms against CMS’s proposal is just wrong. We’re all individuals, not a collective.”

“I understand the need for hope,” she said, expressing a personal opinion, “but people living with dementia need to be protected as well. This drug has very serious, frequent side effects. My concern is that whatever CMS decides, they at least put in some guardrails so that people taking this drug get proper workups and monitoring.”

The debate over Medicare’s decision on Aduhelm is crucial, since most people with Alzheimer’s are older or seriously disabled and covered by the government health program.

To learn more, I talked to several people living with dementia. Here’s some of what they told me:

Jay Reinstein, 60, is married and lives in Raleigh, North Carolina. He was diagnosed with early-onset Alzheimer’s disease three years ago and formerly served on the national board of directors of the Alzheimer’s Association.

“I understand [Aduhelm] is controversial, but to me it’s a risk I’m willing to take because there’s nothing else out there,” Reinstein said, noting that people he’s met through support groups have progressed in their disease very quickly. “Even if it’s a 10% chance of slowing [Alzheimer’s] down by six months, I am still willing to take it. While I am progressing slowly, I want more time.”

Laurie Scherrer of Albertville, Alabama, was diagnosed with early-onset Alzheimer’s and in 2013, at age 55.

Early on, she was prescribed Aricept (donepezil), one of a that address Alzheimer’s symptoms. “I became totally confused and disoriented, I couldn’t think, I couldn’t concentrate,” she told me. After stopping the medication, those symptoms went away.

“I am not for CMS approving this drug, and I wouldn’t take it,” Scherrer said. At discussion groups on Aduhelm hosted by the Dementia Action Alliance (Scherrer is on the board), only two of 50 participants wanted the drug to be made widely available. The reason, she said: “They don’t think there are enough benefits to counteract the possible harms.”

Rebecca Chopp, 69, of Broomfield, Colorado, was diagnosed with early-onset Alzheimer’s in March 2019. She’s a former chancellor of the University of Denver.

Chopp is a member of a newly formed group of five people with dementia who meet regularly, “support one another,” and want to “tell the story of Alzheimer’s from our perspective,” she said.

Two people in the group have taken Aduhelm, and both report that it has improved their well-being. “I believe in science, and I am very respectful of the large number of scientists who feel that [Aduhelm] should not have been approved,” she told me. “But I’m equally compassionate toward those who are desperate and who feel this [drug] might help them.”

Chopp opposes CMS’ decision because “Aduhelm has been FDA-approved and I think it should be funded for those who choose to take it.”

Joanna Fix, 53, of Colorado Springs was diagnosed with early-onset Alzheimer’s disease in October 2016. She, too, developed serious complications after taking Aricept and another dementia medication, Namenda (memantine).

“I would love it if tomorrow somebody said, ‘Here’s something that can cure you,’ but I don’t think we’re at that point with Aduhelm,” Fix told me. “We haven’t been looking at this [drug] long enough. It feels like this is just throwing something at the disease because there’s nothing else to do.”

“Please, please take it from someone living with this disease: There is more to life than taking a magic pill,” Fix continued. “All I care about is my quality of life. My marriage. Educating and helping other people living with dementia. And what I can still do day to day.”

Phil Gutis, 60, of Solebury, Pennsylvania, has participated in clinical trials and taken Aduhelm for 5½ years after being diagnosed with early-onset Alzheimer’s in 2016.

He’s convinced the medication has helped him. “I don’t know how to describe it other than to say my head feels so much clearer now,” he told me. “I feel much more capable of doing things now. It’s not like I’ve gained my memories back, but I certainly haven’t deteriorated.”

Gutis thinks CMS’ proposed restrictions on Aduhelm are misguided. “When the FDA approved it, there was this sense of excitement — oh, we’re getting somewhere. With the CMS decision, I feel we are setting the field back again. It’s this constant feeling that progress is being made and then — whack.”

Christine Thelker, 62, is a widow who lives alone in Vernon, British Columbia. She was diagnosed with vascular dementia seven years ago and is a board member for Dementia Advocacy Canada, which supports restrictions on Aduhelm’s availability.

“Most of us who live with dementia understand a cure is not likely: There are too many different types of dementia, and it’s just too complicated,” Thelker told me. “To think we’re just going to take a pill and be better is not realistic. Don’t give us false hope.”

What people with Alzheimer’s and other types of dementia need, instead, is “various types of rehabilitation and assistance that can improve our quality of life and help us maintain a sense of hope and purpose,” Thelker said.

Jim Taylor of New York City and Sherman, Connecticut, is a caregiver for his wife, Geri Taylor, 78, who has moderate Alzheimer’s. She joined a clinical trial for Aduhelm in 2015 and has been on the drug since, with the exception of about 12 months when Biogen temporarily stopped the clinical trial. “In that period, her short-term memory and communications skills noticeably declined,” Jim Taylor said.

“We’re convinced the medication is a good thing, though we know it’s not helpful for everybody,” Taylor continued. “It really boosts [Geri’s] spirits to think she’s part of research and doing everything she can.

“If it’s helpful for some and it can be monitored so that any side effects are caught in a timely way, then I think [Aduhelm] should be available. That decision should be left up to the person with the disease and their care partner.”

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So it’s no surprise that some of the same drugs are being reformulated and renamed by manufacturers as a new obesity treatment. No longer limited to the crowded field of treatments for Type 2 diabetes, which of Americans, they join the far smaller number of drugs for obesity, and is ready to be mined for profit.

One that recently hit the market — winning Food and Drug Administration approval in June — is Novo Nordisk’s Wegovy, a higher-dose version of the company’s injectable diabetes drug, Ozempic.

Ozempic’s peppy ads suggest that people who use it might lose weight, but also include a disclaimer: that it “is not a weight loss drug.” Now — with a new name — it is. And clinical trials showed for many patients.

“People who go on this medication lose more weight than with any drug we’ve seen, ever,” said Dr. Fatima Cody Stanford, an obesity medicine specialist at Massachusetts General Hospital and Harvard Medical School who was not involved with any of the clinical trials.

But that leaves employers and insurers in the uncomfortable position of deciding if it’s worth it.

Wegovy’s monthly wholesale price tag — set at $1,349 — is about 58% more than Ozempic’s, although, the company points out, the drug’s injector pens contain more than twice as much of the active ingredient. Studies so far show that patients may need to take it indefinitely to maintain weight loss, translating to a tab that could top $323,000 over 20 years at the current price. Weight loss treatments are not universally covered by insurance policies.

The arrival of this new class of weight loss drugs — may soon follow — has created a thicket of issues for those who will pay for them. The decision is complicated by many unknowables concerning their long-term use and whether competition might eventually lower the price.

“The metric we try to use is value,” said James Gelfand, senior vice president for health policy at the ERISA Industry Committee, or ERIC, which represents large, self-insured employers. “If we pay for this drug, how much is this going to cost and how much value will it provide to the beneficiaries?”

have had a lackluster past in this regard, with only modest results. Many employers and insurers likely remember , a combination of fenfluramine and dexfenfluramine that was pulled from the market in the late 1990s for causing heart valve problems.

New drugs like Wegovy, more effective but also pricier than previous weight loss treatments, will add more fuel to that debate.

Past treatments were shown to prompt weight loss in the range of 5% to 10% of body weight. But many had relatively serious or unpleasant .

Wegovy, however, helped patients lose an average of 15% of their body weight over 68 weeks in the that led to its approval. A comparison group that got a placebo injection lost an average of 2.5% over the same period. On the high end, nearly a third of patients in the treatment group lost 20% or more. Both groups had counseling on diet and exercise.

Side effects, generally considered mild, included nausea, diarrhea, vomiting and constipation. A few patients developed pancreatitis, a serious inflammation of the pancreas. Like the diabetes medication, the drug carries a warning about a potential risk of a type of thyroid cancer.

Weight loss in those taking Wegovy puts it close to the 20% to 25% losses seen with bariatric surgery, said Stanford at Mass General, and well above the 3% to 4% seen with diet and other lifestyle changes alone.

Participants also saw reductions in their waistlines and improvements in their blood pressure and blood sugar levels, which may mean they won’t develop diabetes, said Dr. Sean Wharton, an internal medicine specialist and adjunct professor at York University in Toronto who was among the co-authors of the report outlining the results of the on Wegovy.

Since weight loss is known to reduce the risk of heart attack, high blood pressure and diabetes, might the new drug type be worth it?

Covering such treatment would be a sea change for Medicare, which specifically bars coverage for obesity medications or drugs for “anorexia, weight loss or weight gain,” although it does pay for bariatric surgery. Pharmaceutical companies, patient advocates and some medical professionals are backing proposed federal legislation to allow coverage. But the legislation, the Treat and Reduce Obesity Act, has not made progress despite being reintroduced every year since 2012, and sponsors are now instead to rewrite existing rules.

Private insurers will have to consider a cost-benefit analysis of adding Wegovy to their list of covered treatments, either broadly or with limits. Obesity was first recognized as a disease by the American Medical Association, easing the path for insurance coverage, in .

“Employers are going to have a bit of a challenge” deciding whether to add the benefit to insurance offerings, said Steve Pearson, founder and president of the Institute for Clinical and Economic Review, which provides cost-benefit analyses of medical treatments but has not yet looked at Wegovy.

The trade-offs are embodied in patients like Phylander Pannell, a 49-year-old Largo, Maryland, woman who said she lost 65 pounds in a clinical trial of Wegovy. That study gave the drug to all participants for the first 20 weeks, then randomly assigned patients to get either the drug or a placebo for the next 48 weeks to determine what happens when the medication is stopped. Only after the trial ended did she find out she was in the treatment group the entire time.

Her weight fell slowly at first, then ramped up, eventually bringing her 190-pound frame down to about 125. Pains in her joints eased; she felt better all around.

“I definitely feel the drug was it for me,” said Pannell, who also followed the trial’s guidance on diet and exercise.

The found that both groups lost weight in the initial 20 weeks, but those who continued to get the drug lost an additional average of 7.9% of their body weight. Those who got a placebo gained back nearly 7%.

After the trial ended, and the covid-19 pandemic hit, Pannell regained some weight and is now at 155. She is eager to get back on the medication and hopes her job-based insurance will cover it.

Many employers do cover obesity drugs. For example, about 40% of private employer plans include Novo Nordisk’s once-daily injection called Saxenda on their health plans, said Michael Bachner, Novo Nordisk’s director of media relations.

He said the $1,349-a-month wholesale acquisition price of Wegovy was determined by making it equivalent to that of Saxenda, which is less effective.

Still, that is more than the $851 monthly wholesale price of Ozempic. But, he points out, the recommended dosage of Wegovy is more than twice that of Ozempic. Four milligrams come in the Ozempic injector pens for the month, while Wegovy has 9.6.

“There’s more drug in the pen,” Bachner said. “That drives the price up.”

He added: “This is not a 20-year-old drug that we now have a new indication for and are pricing it higher. It’s a whole different clinical program,” which required new trials.

Now scientists, employers, physicians and patients will have to decide whether the new drugs are worth it.

Earlier estimates — some commissioned by Novo Nordisk — of the potential cost of adding an obesity drug benefit to Medicare showed an overall reduction in spending when better health from the resulting weight loss was factored in.

Still, those considered much less expensive drugs, including a range of generic and branded drugs costing as little as $7 a month to more than $300, a small fraction of Wegovy’s cost.

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“There’s lots of things that she used to be able to do just fine,” said her mom, Londen Tabor, who lives with her daughter in Gillette, Wyoming. Autumn’s speech has become slurred and her cognitive skills slower. She needs help with many tasks, such as writing, showering and dressing, and while she can walk, her balance is off.

Autumn has been turned down for clinical trials because she is too young.

“It is so frustrating to me,” Tabor said. “I would sell my soul to try to get any type [of treatment] to help my daughter.”

For patients like Autumn with serious or immediately life-threatening conditions who do not qualify for clinical trials and have exhausted all treatment options, there may be another option: seeking approval from the Food and Drug Administration for , or compassionate use, of experimental therapies.

Definitive numbers are hard to find, but studies from researchers, actions by drugmakers and insights from experts suggest that getting expanded access to unproven therapies for rare diseases is more difficult than for more common illnesses, such as cancer.

Even with experimental treatments on the rise, patients with rare diseases frequently face an unwillingness by drug companies to provide them before clinical studies are completed. Developing drugs for these diseases is an especially fragile process because the patient populations are small and often diverse, having different genetics, symptoms and other characteristics, which makes studying the drugs’ effects difficult.

Drugmakers believe offering a drug before studies are finished could impair its development and jeopardize FDA approval.

Companies working on therapies for rare diseases, especially smaller ones, could feel those repercussions acutely, said Lisa Kearns, a researcher in the ethics division of New York University’s medical school and member of the division’s working group on compassionate use and preapproval access. “There’s not as much investment in rare diseases, so an [adverse] event could frighten the already limited number of potential investors.”

Drugs that were not made available for compassionate use last year until studies were completed include , ; Enspryng, for an ; and , .

A spokesperson for Roche, which makes Evrysdi and Enspryng and is working on a treatment for Huntington’s disease, said the decision was tied not to the type of disease but to company policy: Roche does not set up expanded access programs for any drugs until results are available from a phase 3 clinical trial. (Those phase 3 studies are typically the last testing done before the company seeks drug approval.)

Another company’s experimental drug for , an autoimmune disease that leads to skeletal muscle weakness, similarly was not available through until , and no programs have started for a therapy being studied in and , a fatal neurodegenerative disease often referred to as Lou Gehrig’s disease.

One slight, but notable, deviation: Drugmaker Biogen this year to allow certain ALS patients to receive an experimental drug , after the testing was to be completed but before the results are known.

Dr. Merit Cudkowicz, a neurologist at Massachusetts General Hospital in Boston, has helped patients get therapies through expanded access. Since September 2018, she and colleagues being developed by drug companies, but only about 120 patients have received therapies this way. More than 16,000 people in the United States in 2015 to have ALS and do not qualify for clinical trials because of the progression of their disease or very strict eligibility requirements.

These examples contrast with some drugs for more common problems. , for leukemia, was offered to thousands of patients through expanded access programs before the manufacturer completed the clinical studies that led to FDA approval. , for HIV/AIDS, and , for the most common type of lung cancer, were similarly offered to large numbers of patients even as clinical trials were ongoing.

Last year, Novartis gave more than 7,000 patients worldwide early access to cancer drugs.

Doctors also report that getting experimental drugs for cancer patients is relatively simple. More than 200 physicians around the country were surveyed, and among those who applied for access, said they had secured drugs still being investigated for patients who were not responding to approved therapies.

California researchers found similar trends in a of 23 social media campaigns launched by patients between 2005 and 2015 seeking a variety of experimental treatments. While seven of the 19 patients with cancer received early access to requested drugs, no access was allowed for three patients with rare diseases, although one of those patients was allowed to enroll in a clinical trial.

Companies base their decisions on whether to provide a therapy through expanded access on a number of factors, said Jess Rabourn, CEO of WideTrial, which helps pharmaceutical companies run compassionate use programs. In general, there should be evidence that patients can tolerate the treatment and an expectation that any benefit outweighs the risk, he said.

“This idea that you have to wait until the research is done is baloney,” he said. “We’re talking about patients who are going to die if they’re told to wait.”

But drugmakers often view it differently, even though evidence suggests that granting early access drug approval.

Kearns explained that companies often wait until phase 3, or after, because they can be “relatively” confident of a drug’s safety and effectiveness. “They don’t want to harm patients, of course, but they also do not want to threaten the drug’s eventual regulatory approval with an adverse event in [a] very sick patient population.”

Melissa Hogan, who consults on clinical trials for rare diseases and is an , attributes the lack of access to the high cost of therapies and the tightknit nature of the rare disease community, where patients and their families often set up social media groups and exchange ideas and treatment plans. Companies “know that if one patient gains access, other patients will know” and ask for access, said Hogan, who has a son with mucopolysaccharidosis type II. That could overwhelm small drugmakers with little manufacturing capacity.

These concerns cause “many companies [to] just throw up their hands and take a hard line of no [expanded access] until they reach approval stage,” said Hogan.

The 2018 offers another option for some patients. Unlike expanded access, the law applies only to requests for medicines — not medical devices — and does not require approval from the FDA or an , a committee that reviews and monitors people participating in research for their protection. The legislation, however, doesn’t oblige companies to grant a request.

For Cali Orsulak, expanded access may be her husband’s only option. He was diagnosed with ALS in 2019 at age 43.

“We did our best with the skill level we had to search clinical trials all over Canada and the U.S., and then covid hit and it became increasingly difficult,” said Orsulak, explaining that they live in Canada but seek medical care in the United States. “Now that my husband has progressed, it’s even harder to get into clinical trials.”

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By that evening, though, he had a fever of 101 degrees and was clearly ill. “I felt like I had been hit by a truck,” recalled Mutter, who lives near Seattle.

The next day he was diagnosed with COVID-19. By Saturday, the 58-year-old was enrolled in a clinical trial for the same antibody cocktail that President Donald Trump claimed was responsible for his

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Notifications that the funding would not be extended were relayed May 30 to researchers, who were told by officials that the had elected “to go with currently available approaches to eliminate HIV” instead.

The cuts will shutter two major HIV vaccine research efforts first funded by the NIH at the Duke Human Vaccine Institute and the Scripps Research Institute, scientists said. A Moderna spokesperson said the vaccine manufacturer’s through the NIH’s HIV Vaccine Trials Network have also been put on pause.

One senior NIH official said the HHS had instructed the agency not to issue any more funding in the next fiscal year for HIV vaccine research, with only a handful of exceptions. 

A budgetary rule change specifically targeted at HIV vaccine research is also expected to lead to another cut to the NIH’s awards for studies initiated by scientists, an official said.

The change, to be finalized shortly, inflates the accounting for the upfront cost of studies into HIV vaccines funded by the agency. Instead of the cost of a five-year grant being spread over five years, the NIH plans to make HIV vaccine dollars from multiyear grants all count toward a single year, the official said, making it harder for them to get funded.

A spokesperson for HHS told CBS News that “complex and duplicative health programs have resulted in serious duplication of efforts,” saying that “27 separate programs that address HIV/AIDS” had spent $7.5 billion.

“The Administration believes the United States should have the best medical research in the world. To that end, we are advancing policies to maximize the impact of every federal taxpayer dollar and ensure proper oversight of this funding,” HHS spokesperson Emily Hilliard said.

Hilliard claimed “critical HIV/AIDS programs will continue” under the new agency that Health and Human Services Secretary Robert F. Kennedy Jr. has proposed creating, dubbed the Administration for a Healthy America.

“For HIV vaccine design and development, we’ve begun to see light at the end of the tunnel after many years of research. This is a terrible time to cut it off. We’re beginning to get close. We’re getting good results out of clinical trials,” said , an immunology professor at Scripps Research.

Burton warned that his institution’s HIV vaccine research could not simply be turned back on, even if a future administration decided to change course on HIV funding. He said that ongoing experiments would be shuttered and that researchers assembled to study the issue would be forced to refocus their careers on other topics.

“This is a decision with consequences that will linger. This is a setback of probably a decade for HIV vaccine research,” Burton said.

The cancellation of the funds comes weeks ahead of the FDA’s for deciding on approval of lenacapavir, a twice-yearly injectable drug to prevent HIV.

The drug, which is being brought to the commercial market by drugmaker Gilead Sciences, builds on into earlier HIV medications. The drug’s availability could lead to a significant drop in HIV cases worldwide, since it was 100% effective in preventing transmission.

An NIH official, who was not authorized to speak publicly, rebuked the claim that the effectiveness of current HIV prevention strategies meant a vaccine was no longer needed. “The only way of ending the HIV epidemic in the U.S. and AIDS pandemic worldwide” is with a vaccine, the official said.

Developing an effective HIV vaccine has been an elusive target for researchers, though scientists have hailed recent breakthroughs in the field.

“HIV has established roadblocks to us fighting it off, which are unparalleled in vaccinology. We’ve had to learn what each of the roadblocks are and to devise ways to overcome it. This virus mutates so quickly,” said Duke professor of medicine Barton Ford Haynes, who is part of the Duke Human Vaccine Institute.

Haynes said his institute’s work was essentially combining different vaccines as part of a strategy to design an effective HIV vaccine.

He praised lenacapavir as a “wonderful development for the field” but said there was still a need for a vaccine. Lenacapavir requires injections every six months to remain effective, a challenging proposition even before steep cuts to the Centers for Disease Control and Prevention’s domestic HIV programs and U.S.-backed HIV/AIDS foreign aid programs.

“The hope was that adding an HIV vaccine to all the preventive measures that we have would finally allow us to end the pandemic,” Haynes said.

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Hayes is too sick to work, so she has spent much of the last four years sitting on her beige couch, often curled up under an electric blanket.

“My blood flow now sucks, so my hands and my feet are freezing. Even if I’m sweating, my toes are cold,” , who lives in Western Pennsylvania. She misses feeling well enough to play with her 9-year-old son or attend her 17-year-old son’s baseball games.

Along with claiming the lives of 1.2 million Americans, the covid-19 pandemic has been described as a . Hayes is one of millions of Americans who suffer from long covid. Depending on the patient, the condition can rob someone of energy, scramble the autonomic nervous system, or fog their memory, among many other symptoms.

In addition to the brain fog and chronic fatigue, Hayes’ constellation of symptoms includes frequent hives and migraines. Also, her tongue is constantly swollen and dry.

“I’ve had multiple doctors look at it and tell me they don’t know what’s going on,” Hayes said about her tongue. 

Estimates of prevalence range considerably, depending on how researchers define long covid in a given study, but the Centers for Disease Control and Prevention puts it at 17 million adults.

Despite long covid’s vast reach, the federal government’s investment in researching the disease — to the tune of $1.15 billion as of December — has so far failed to bring any new treatments to market. 

This disappoints and angers the patient community, who say the National Institutes of Health should focus on ways to stop their suffering instead of simply trying to understand why they’re suffering.

“It’s unconscionable that more than four years since this began, we still don’t have one FDA-approved drug,” said , executive director of the , a patient-led advocacy organization. Stone was among several people with long covid who spoke at a workshop hosted by the NIH in September where patients, clinicians, and researchers discussed their priorities and frustrations around the agency’s approach to long-covid research.

Some doctors and researchers are also critical of the agency’s research initiative, called RECOVER, or Researching COVID to Enhance Recovery. Without clinical trials, physicians specializing in treating long covid must rely on hunches to guide their clinical decisions, said , chief of research and development with the .

“What [RECOVER] lacks, really, is clarity of vision and clarity of purpose,” said Al-Aly, saying he agrees that the NIH has had enough time and money to produce more meaningful progress.

Now the NIH is starting to determine how to allocate an additional of funding for long-covid research, of which is earmarked for clinical trials. These funds will be allocated over the next four years.

At the end of October, RECOVER for clinical trial ideas that look at potential therapies, including medications, saying its goal is “to work rapidly, collaboratively, and transparently to advance treatments for Long COVID.”

This turn suggests the NIH has begun to respond to patients. This has stirred cautious optimism among those who say that the agency’s approach to long covid has lacked urgency in the search for effective treatments.

Stone calls this $300 million a down payment. She warns it’s going to take a lot more money to help people like Hayes regain some degree of health.

“There really is a burden to make up this lost time now,” Stone said.

The NIH told ºÚÁϳԹÏÍø News and NPR via email that it recognizes the urgency in finding treatments. But to do that, there needs to be an understanding of the biological mechanisms that are making people sick, which is difficult to do with post-infectious conditions.

That’s why it has funded research into how long covid affects , or trying to understand why people are afflicted with the condition.

Good Science Takes Time

In December 2020, for the NIH to launch RECOVER, raising hopes in the long-covid patient community.

Then-NIH Director explained that was to better understand long covid as a disease and that clinical trials of potential treatments would come later.

According to RECOVER’s website, it has funded to test the safety and effectiveness of an experimental treatment or intervention. Just one of those trials has .

On the other hand, RECOVER has supported more than 200 observational studies, such as research on how long covid and on which symptoms are . And the initiative has funded more than 40 pathobiology studies, which focus on the basic cellular and molecular mechanisms of long covid.

RECOVER’s this research has led to crucial insights on the risk factors for developing long covid and on understanding how the disease interacts with preexisting conditions.

It notes that observational studies are important in helping scientists to design and launch evidence-based clinical trials.

Good science takes time, said , the co-principal investigator for the RECOVER-Adult Observational Cohort at New York University. And long covid is an “exceedingly complicated” illness that appears to affect nearly every organ system, she said. 

This makes it more difficult to study than many other diseases. Because long covid harms the body in so many ways, with widely variable symptoms, it’s harder to identify precise targets for treatment.

“I also will remind you that we’re only three, four years into this pandemic for most people,” Horwitz said. “We’ve been spending much more money than this, yearly, for 30, 40 years on other conditions.”

NYU received of RECOVER funds in 2021, which the institution is using to spearhead the collection of data and biospecimens from up to 40,000 patients. Horwitz said nearly 30,000 are enrolled so far.

This , Horwitz said, supports ongoing observational research, allowing scientists to understand what is happening biologically to people who don’t recover after an initial infection — and that will help determine which clinical trials for treatments are worth undertaking.

“Simply trying treatments because they are available without any evidence about whether or why they may be effective reduces the likelihood of successful trials and may put patients at risk of harm,” she said.

Delayed Hopes or Incremental Progress?

The NIH told ºÚÁϳԹÏÍø News and NPR that patients and caregivers have been central to RECOVER from the beginning, “playing critical roles in designing studies and clinical trials, responding to surveys, serving on governance and publication groups, and guiding the initiative.”

But the consensus from patient advocacy groups is that RECOVER should have done more to prioritize clinical trials from the outset. Patients also say RECOVER leadership ignored their priorities and experiences when determining which studies to fund.

RECOVER has scored some gains, said , co-director of . This includes findings on differences in long covid between adults and kids.

But Davids said the NIH shouldn’t have named the initiative “RECOVER,” since it wasn’t designed as a streamlined effort to develop treatments.

“The name’s a little cruel and misleading,” he said.

RECOVER’s initial allocation of $1.15 billion probably wasn’t enough to develop a new medication to treat long covid, said co-director of the University of Pennsylvania’s .

But, he said,  the results of preliminary clinical trials could have spurred pharmaceutical companies to fund more studies on drug development and test how existing drugs influence a patient’s immune response.

Emanuel is one of the authors of a March 2022 covid . He notes that RECOVER’s lack of focus on new treatments was a problem. “Only 15% of the budget is for clinical studies. That is a failure in itself — a failure of having the right priorities,” he told ºÚÁϳԹÏÍø News and NPR via email.

And though the NYU biobank has been impactful, Emanuel said there needs to be more focus on how existing drugs influence immune response.

He said some clinical trials that RECOVER has funded are “ridiculous,” because they’ve focused on symptom amelioration, for example to of over-the-counter medication to improve sleep. Other studies looked at non-pharmacological interventions, such as exercise and “” to help with cognitive fog.

People with long covid say this type of clinical research contributes to what many describe as the “gaslighting” they experience from doctors, who sometimes blame a patient’s symptoms on anxiety or depression, rather than acknowledging long covid as a real illness with a physiological basis.

“I’m just disgusted,” said long-covid patient Hayes. “You wouldn’t tell somebody with diabetes to breathe through it.”

, director and founder of the , said she’s even taken breaks from seeking treatment after getting fed up with being told that her symptoms were due to her diet or mental health.

“You’re at the whim of somebody who may not even understand the spectrum of long covid,” Sweeney said.

Insurance Battles Over Experimental Treatments

Since there are still no long-covid treatments approved by the Food and Drug Administration, anything a physician prescribes is classified as either experimental — for unproven treatments — or an off-label use of a drug approved for other conditions. This means patients can struggle to get insurance to cover prescriptions.

, medical director for — said he writes many appeal letters. And some people pay for their own treatment.

For example, intravenous immunoglobulin therapy, low-dose naltrexone, and hyperbaric oxygen therapy are all promising treatments, he said.

For hyperbaric oxygen, , randomized show improvements for the chronic fatigue and brain fog that often plague long-covid patients. The theory is that higher oxygen concentration and increased air pressure can help heal tissues that were damaged during a covid infection.

However, the out-of-pocket cost for a series of sessions in a hyperbaric chamber can run as much as $8,000, Brode said.

“Am I going to look a patient in the eye and say, ‘You need to spend that money for an unproven treatment’?” he said. “I don’t want to hype up a treatment that is still experimental. But I also don’t want to hide it.”

There’s a host of pharmaceuticals that have promising off-label uses for long covid, said microbiologist , president and chief scientific officer at the Massachusetts-based . For instance, she’s collaborating on a clinical study that repurposes two HIV drugs to treat long covid.

Proal said research on treatments can move forward based on what’s already understood about the disease. For instance, she said that scientists — partly due to — that some patients small amounts of viral material after a covid infection. She has not received RECOVER funds but is researching antivirals.

But to vet a range of possible treatments for the millions suffering now — and to develop new drugs specifically targeting long covid — clinical trials are needed. And that requires money.

Hayes said she would definitely volunteer for an experimental drug trial. For now, though, “in order to not be absolutely miserable,” she said she focuses on what she can do, like having dinner with her family.

At the same time, Hayes doesn’t want to spend the rest of her life on a beige couch. 

RECOVER’s deadline to submit research proposals for potential long-covid treatments is .

This article is from a partnership that includes and ºÚÁϳԹÏÍø News.

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“Right To Try” experimental drug program saved “thousands and thousands of lives”

Former President Donald Trump on Aug. 30

Former President Donald Trump has boasted in recent months about “Right To Try,” a law he signed in 2018. It’s aimed at boosting terminally ill patients’ access to potentially lifesaving medications not yet approved by the Food and Drug Administration.

“We have things to fight off diseases that will not be approved for another five or six years that people that are very sick, terminally ill, should be able to use. But there was no mechanism for doing it,” Trump , speaking in Washington, D.C., to supporters of the conservative parental rights advocacy group Moms for Liberty.

He also said that because of Right To Try, “we have saved thousands and thousands of lives.”

Trump similarly praised the program during an in Pennsylvania, in a with a conservative commentator, and during his Republican National Convention : “Right To Try is a big deal,” Trump said then.

Medical experts who’ve studied the experimental treatment program, however, say there’s no evidence to support Trump’s claims. These experts say Right To Try weakened regulations intended to protect patients.

What Is Right To Try?

The , aka Right To Try, passed Congress on a bipartisan basis and was signed into law in 2018. It sought to streamline the process for getting potentially lifesaving drugs that weren’t yet FDA-approved to terminally ill patients. The speed matters; industry groups say it takes on average for a new medicine to reach pharmacy shelves.

However, a similar FDA program, the pipeline, sometimes called “compassionate use,” has existed , and became law in 1987.

And that is the root of many criticisms of Right To Try.

“Right To Try is basically ‘expanded access light,’” said Alison Bateman-House, a medical ethicist who researches access to investigational medical products at New York University’s Grossman School of Medicine.

Right To Try caters to fewer patients than expanded access and offers them fewer treatments, Bateman-House said.

Easing Access or Erasing Safeguards?

Patients must meet specific, but different, criteria to qualify for either experimental medication program.

To qualify for expanded use, patients must have a “serious or immediately life-threatening disease or condition” for which there is no “comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the disease or condition,” according to . Clinical trials must be infeasible for the patients, and the use of these drugs must not interfere with any in-progress studies. Also, the potential benefits must justify the risks, according to the prescribing physicians.

Then, after identifying a treatment, the patient’s doctor must receive approval from its manufacturer, the FDA, and the institutional review board overseeing the medication’s clinical trials.

The FDA said these steps exist so the agency can “fairly weigh the risks and benefits” of the medication and protect the patient’s safety. The agency also collects data about the drugs’ clinical impact on the patient and any adverse effects to inform the wider approval process for the drug.

Right To Try sought to hasten this approval process. Under the new program, for instance, a doctor must merely identify an experimental medication and receive authorization to use it from the manufacturer. In most cases, the FDA has no authority to approve or deny the application, and there’s no review board process to navigate.

But, because of the Right To Try program’s definitions, fewer patients and fewer medicines qualify.

Under Right To Try, patients must have a “life-threatening” disease or condition, not just “serious,” as with expanded access. Experimental medications are available only after they’ve completed Phase 1 clinical trials; treatments accessed through the expanded access program can be administered during a Phase 1 study.

Right To Try, which includes liability protections for manufacturers and prescribing physicians, also weakens requirements that govern how doctors disclose experimental medications’ risks to patients, leaving informed consent undefined. And it prevents the FDA from using information about how patients tolerate the drugs to “delay or adversely affect the review or approval of such drug(s),” unless top officials justify the benefit to public health in writing.

Supporters say Right To Try is an example of successful deregulation and claim that its more efficient approval process saved lives. But critics see this as a key reason for concern, because it “opens up the opportunity of exploiting desperate patients,” said Holly Fernandez Lynch, a bioethicist who studies pharmaceutical policy at the University of Pennsylvania’s Perelman School of Medicine.

Government data shows regulatory agencies weren’t the main hurdle patients faced when seeking experimental drugs. The FDA almost always approved expanded access applications, and quickly by government standards.

According to a 2018 FDA report on the expanded access program, the FDA authorized 99% of the roughly 9,000 requests it received in the previous five years, approving emergency requests for experimental medications in less than one day on average. More shows that approval trend has continued, even as the number of applications has grown each year.

In rare cases in which the FDA didn’t automatically approve requests, regulators often didn’t deny them, but recommended tweaks to the requested dosage to address safety and effectiveness concerns.

Right To Try by the Numbers

The FDA does not share detailed information about the number of doses provided or patients treated under Right To Try. Instead, it posts only an showing how many drugs have been approved under the program. The agency says that since Right To Try began in 2018 it has approved 16 treatments: 12 from 2018 to 2022 and four last year.

The FDA declined to provide additional information about the number of Right To Try requests or approvals.

Although the 16 medications approved through Right To Try were possibly provided to more than one patient each, experts said it’s extremely unlikely thousands of patients were involved, as Trump said.

Trump’s claim represents an “egregious overestimate of the number of people who are using Right To Try,” said Fernandez Lynch, noting she believes the real numbers are “very, very low.”

The Trump campaign did not respond to multiple inquiries about the source of the former president’s statistics. Karoline Leavitt, the campaign’s national press secretary, told ºÚÁϳԹÏÍø News that in a second term “President Trump will of course remain open to other pathways to expand ‘Right to Try’ to save more American lives.”

It remains unclear how Trump might expand the program, though the conservative Goldwater Institute is advocating for “,” which it claims will let patients receive individualized therapeutics.

Experts noted such drugs are already accessible through the expanded access program.

Meanwhile, evidence shows that the high price of experimental treatments, which are sometimes available through certain drug company programs but not typically covered by insurance, is a greater hurdle to patients than regulatory guardrails are.

“I don’t think that people are having a problem with the FDA blocking access to individualized therapeutics,” Bateman-House said. “I think the problem is that individualized therapeutics are incredibly expensive, and there’s only a very small number of researchers in the country who know how to make them.”

Our Ruling

Trump has claimed throughout the campaign that his Right To Try program is novel and has saved thousands of lives. But a similar program has existed for decades, and there is no evidence Right To Try has had anywhere close to the impact Trump said it has had.

Neither the Trump campaign nor Right To Try advocates provided evidence to back claims of widespread benefit. And government data shows only 16 medications have been approved under the program in its first six years, with no accounting of how many patients used those medications or their clinical outcomes.

Moreover, public health experts have said Right To Try weakens patient protections and fails to address the true barriers to experimental medications.

We rate Trump’s claim False.

our sources:

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To bring a new drug to market, the FDA requires pharmaceutical firms to perform extensive studies to demonstrate safety and efficacy, which are often expensive and time-consuming to conduct to the agency’s specifications. Getting a drug to market a few months sooner and for less expense than usual can translate into millions in profit for the manufacturer.

That is why a private equity-backed startup like Headlands Research saw an opportunity in creating a network of clinical sites and wringing greater efficiency out of businesses, to perform this critical scientific work faster. And why Moderna, Pfizer, Biogen, and other drug industry bigwigs have been willing to hire it — even though it’s a relatively new player in the field, formed in 2018 by investment giant KKR.

In July 2020, Headlands it won coveted contracts to run clinical trials of covid-19 vaccines, which would include shots for AstraZeneca, Johnson & Johnson, Moderna, and Pfizer.

In marketing its services, Headlands described its mission to “profoundly impact” clinical trials — including boosting participation among racial and ethnic minorities who have long been underrepresented in such research.

“We are excited,” CEO Mark Blumling said in a statement, to bring “COVID-19 studies to the ethnically diverse populations represented at our sites.” Blumling, a drug industry veteran with venture capital and private equity experience, told KHN that KKR backed him to start the company, which has grown by buying established trial sites and opening new ones.

Finding and enrolling patients is often the limiting and most costly part of trials, said Dr. Marcella Alsan, a public policy professor at Harvard Kennedy School and an expert on diverse representation in clinical trials, which have a median cost of $19 million for new drugs, .

Before covid hit, Headlands acquired research centers in McAllen, Texas; Houston; metro Atlanta; and Lake Charles, Louisiana, saying those locations would help it boost recruitment of diverse patients — an urgent priority during the pandemic in studying vaccines to ward off a disease disproportionately killing Black, Hispanic, and Native Americans.

Headlands’ sites also ran, among other things, clinical studies on treatments to combat Type 2 diabetes, postpartum depression, asthma, liver disease, migraines, and endometriosis, according to a review of website archives and the federal website ClinicalTrials.gov. But within two years, some of Headlands’ alluring promises would fall flat.

In September, Headlands shuttered locations in Houston — one of the nation’s largest metro areas and home to major medical centers and research universities — and Lake Charles, a move Blumling attributed to problems finding “experienced, highly qualified staff” to carry out the complex and highly specialized work of clinical research. The McAllen site is not taking on new research as Headlands shifts operations to another South Texas location it launched with Pfizer.

What impact did those sites have? Blumling declined to provide specifics on whether enrollment targets for covid vaccine trials, including by race and ethnicity, were met for those locations, citing confidentiality. He noted that for any given trial, data is aggregated across all sites and the drug company sponsoring it is the only entity that has seen the data for each site once the trial is completed.

A fragmented clinical trials industry has made it a prime target for private equity, which often consolidates markets by merging companies. But Headlands’ trajectory shows the potential risks of trying to combine independent sites and squeeze efficiency out of studies that will affect the health of millions.

, a health economist at Johns Hopkins who has studied private equity acquisitions of physician practices, said consolidation has potential downsides. Singh and her colleagues in September analyzing acquisitions in dermatology, gastroenterology, and ophthalmology that found physician practices — a business with parallels to clinical trial companies — charged higher prices after acquisition.

“We’ve seen reduced market competition in a variety of settings to be associated with increases in prices, reduction in access and choice for patients, and so on,” Singh said. “So it’s a delicate balance.”

, a professor of medicine at Harvard Medical School, called private equity involvement in trials “concerning.”

“We need to make sure that patients” know enough to provide “adequate, informed consent,” he said, and ensure “protections about the privacy of the data.”

“We don’t want those kinds of things to be lost in the shuffle in the goals of making money,” he said.

Blumling said trial sites Headlands acquired are not charging higher prices than before. He said privacy “is one of our highest concerns. Headlands holds itself to the highest standard.”

Good or bad, clinical trials have become a big, profitable business in the private equity sphere, data shows.

Eleven of the 25 private equity firms identified by industry tracker PitchBook as the top investors in health care have bought stakes in clinical research companies, a KHN analysis found. Those companies have been involved in studies ranging from to treatments for ovarian cancer, Parkinson’s disease, and Alzheimer’s.

Contracted firms also analyze patient data and prepare materials to secure approval from regulatory agencies, in hopes of getting more drugs to market faster. And a big draw for investors: Clinical research companies make money whether or not a drug succeeds, making it less risky than investing in a drug company.

The number of clinical trials has exploded to more than 434,000 this year as of late November, more than triple the number a decade ago.

Still, most trial sites are physician practices that don’t consistently perform studies, by Boston-based investment firm Provident Healthcare Partners.

“Independent sites are being purchased by private equity, and they’re moving into larger site groups of 30, 40, and then their game plan is to roll that up into a business and then sell it again,” said Linda Moore Schipani, CEO of Clinical Research Associates, a Nashville-based company that worked on covid vaccine trials for AstraZeneca, Novavax, and Pfizer. “That’s kind of the endgame.”

Headlands is a prime example. in November 2019 that it would acquire six centers in the U.S. and Canada, including three sites in Texas and Louisiana owned by Centex Studies that would help improve participation among Hispanics and African Americans.

It has made other acquisitions since then and opened new sites in areas with “extremely limited trial options,” something Blumling says distinguishes his company.

“I’m not an evangelist for private equity,” Blumling said. “The ability of KKR to be willing to invest in something that is a three- to five-year return versus a one- to two-year return is something that you won’t see out there.”

A research center in Brownsville, Texas — a stone’s throw from the U.S.-Mexico border and where 95% of the population is Hispanic or Latino — is one of several where it is partnering with Pfizer to boost patient diversity.

To recruit patients, Headlands “is really going beyond what a lot of sites do, which is social media,” Blumling said in an interview. “It’s going within churches, community fairs, really getting out into as much as possible the broader community.”

Headlands closed the Houston and Lake Charles sites because of staffing issues, Blumling said, and finished or moved their studies elsewhere. Blumling said the decision to close those locations “did not have anything to do with the speed of trials.”

Similarly, he said, Headlands is moving the McAllen site’s operations to Brownsville “because it had a larger population of trained personnel.”

“We want to continue to grow sites and do great work,” Blumling said. “If we can’t find the people in order to do that at the quality that we demand, which is at the highest level, then it doesn’t make sense to keep those sites.”

‘The Writing to Me Was on the Wall’

In 2006, Devora Torrence co-founded Centex Studies, which she described as “my little mom and pop business” in a about female entrepreneurs in science. She said a flurry of interest from private equity came at the end of 2018. The appeal was evident: Drug companies were relying on bigger clinical trial networks.

“The thing is speed, getting it to market. With a bigger network, you get that speed,” Torrence said on the podcast. “The writing to me was on the wall that either I get some outside investment and scale up myself, or kind of listen to these guys and see if maybe now would be the right time to exit.”

Joining Headlands had its benefits during the pandemic because she could “lean on” its other sites with experience running vaccine trials. “Had we not gotten those … we may not still be here,” Torrence said.

Torrence, whose LinkedIn profile said she left the company in 2021, didn’t respond to messages from KHN.

Lyndon Fullen, a health care consultant and former Centex employee, said private equity provides funding that allows companies to add study sites.

“I completely support it,” he said. “If it’s about reaching that large patient population, it’s of course better to have larger groups with that funding.”

Opportunity in Long Covid

Contract research organization Parexel saw opportunity in the covid pandemic — millions of people were developing long covid after infection and there were few, if any, meaningful treatment options.

The company, which employs more than 19,000 people, was acquired in 2021 by EQT Private Equity and Goldman Sachs’ private equity arm , billions more than the $4.5 billion that private equity firm when it took Parexel private in 2017.

A growing body of research shows the debilitating effects of long covid, including a of tens of thousands of patients in Scotland where nearly half had not fully recovered months later. But treatments addressing its root causes could be years away. “It’s a huge number of people,” said Dr. Nathalie Sohier, who leads Parexel’s infectious diseases and vaccines franchise. “There’s a lot of need.”

Long covid represents the promise and peril of the work to develop new drugs: Millions of patients create a potentially lucrative market for drug companies, and yet researchers and industry experts say they are reluctant to jump in. In part, that’s because “it’s not a well-defined disease, and that really makes it highly risky for companies to invest in research,” said Cecil Nick, a vice president for Parexel.

“How are we going to be able to tell the FDA that our drug works? We can’t count the number of people who died; we can’t count the number of people in the hospital,” said , a University of California-San Francisco professor who is running an observational study on long covid patients.

As of August, among more than 4,400 covid studies, only 304 focused on long covid. A third of those were related to drug development, Sohier said.

Sohier said “there are few” companies in its long covid program. That hasn’t stopped Parexel from as the ideal partner to shepherd new products, including by doing regulatory work and using remote technology to retain patients in trials. Parexel has worked on nearly 300 covid-related studies in more than 50 countries, spokesperson Danaka Williams said.

Michael Fenne, research and campaign coordinator with the Private Equity Stakeholder Project, which studies private equity investments, said Parexel and other contract research organizations are beefing up their data capacity. The aim? To have better information on patients.

“It kind of ties into access and control of patients,” Fenne said. “Technology makes accessing patients, and then also having more reliable information on them, easier.”

KHN senior correspondent Fred Schulte and Megan Kalata contributed to this report.

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There were no drugs against covid-19, and no way to predict which infected patients would develop pneumonia or fall into an inflammatory tailspin leading to severe illness or death. In desperation, Vinetz and countless other doctor-scientists trawled the literature for existing medicines that might help.

“We were in the hospital. We had nothing,” Vinetz said. “I was one of tens of thousands of doctors around the world who said, ‘We gotta figure out what to do.’”

On April 16, 2020, Vinetz saw an about a drug called camostat, licensed in to treat inflammation of the pancreas. Research during the first SARS epidemic, in 2004, had shown the drug had a plausible biochemical mechanism for slowing coronavirus infections, so Vinetz and his colleagues quickly organized a small clinical trial on outpatients with mild to moderate symptoms.

In those days, before covid vaccines and covid-specific treatments appeared on the market, Vinetz’s experiment was one of thousands conducted by doctors who hoped older vaccines and drugs, usually cheap and off-patent, might provide them with options.

Mostly, the drugs were too toxic or had no clear effect. Of the trials for potential covid drugs listed on the website of the National Institutes of Health — including antivirals, anti-inflammatories, and drugs used for cancer, asthma, heart disease, and dozens of other conditions — few have produced helpful medicines.

In fact, only one older drug is routinely used to fight covid. That’s the steroid dexamethasone, to help keep hospitalized patients from requiring supplemental oxygen or intubation.

Drugs like hydroxychloroquine and ivermectin showed hints of value initially but failed in clinical trials — only to remain in circulation, at least partly because their use symbolized affinity in the culture war for some of President Donald Trump’s followers.

A few old drugs still show promise, but they’ve had trouble getting traction. The ivermectin and hydroxychloroquine fiascoes soured doctors on repurposed medications, and the pharmaceutical industry has shown little interest in testing them, especially when it can earn billions from even mediocre new ones, scientists tracking the field say.

American and European scientists have confirmed the theoretical basis for camostat’s impact on covid. But evidence for its effects is weak; last year the from a big NIH trial comparing various treatments.

A more promising story emerged with fluvoxamine, licensed under the brand name Luvox in 1994 to treat obsessive-compulsive disorder. The drug is in the same class as common antidepressants such as Prozac, Lexapro, and Zoloft.

A child psychiatrist noticed fluvoxamine might be good for covid. In March 2020, while recovering from a bout of covid, Dr. Angela Reiersen of Washington University in St. Louis saw a 2019 study in mice that showed how fluvoxamine could activate a protein similar to one with Wolfram syndrome, a genetic disease that causes diabetes, neurological issues, and, eventually, death.

Reiersen and her colleague Dr. Eric Lenze, a geriatric psychiatrist, began a in people with symptoms of covid. Of the 80 in the fluvoxamine group, none suffered a serious decline, while six of 72 patients given sugar pills got pneumonia, and four were hospitalized.

In a follow-up in Brazil, people who took at least 80% of their fluvoxamine pills were 66% less likely to require emergency care or hospitalization than those who got sugar pills. Only one died, compared with 11 in the placebo group.

Since October, when the Brazilian study was published, fluvoxamine’s future has dimmed. Neither the NIH nor the recommends fluvoxamine to prevent respiratory distress. The NIH panelists in the Brazilian trial were only statistically significant among those who remained in the trial. (Because of nausea and other side effects, only 74% of trial participants in the fluvoxamine wing took all their pills, compared with 82% in the placebo wing.)

The NIH panel also was put off by the fact that the Brazilian trial counted hospitalizations as well as people put under a doctor’s care for six hours or more — not a standard measure. Trial organizers said that was necessary because Brazilian hospitals were so packed with covid patients that many people got their care in makeshift outdoor shelters.

Regulators and experts are awaiting results from two other big trials, one of universities and hospitals, . But both studies are using doses of 100 milligrams of fluvoxamine a day, compared with 200 or 300 milligrams in the successful trials.

“I have concerns that they are not using a high-enough dose,” Reiersen said, given that fluvoxamine operates on a different biochemical pathway to fight covid than the one involved in psychiatric treatment.

The concern is shared by Craig Rayner, a former drug company scientist who worked on the Brazilian trial and other big tests of repurposed drugs. “You can do the largest, most well-funded study in the world,” he said, “but if you choose the wrong dose, it’s rubbish in, rubbish out.”

The team overseeing NIH’s trial opted for a lower dose because higher doses had already been used in the earlier trials — and often caused side effects, said Sarah Dunsmore, a program director at NIH’s National Center for Advancing Translational Sciences.

On Dec. 21, David Boulware, a University of Minnesota infectious-disease expert, petitioned the FDA to approve a change in fluvoxamine’s label stating it can be used to prevent respiratory distress in at-risk patients with mild to moderate covid. He hasn’t received a response yet.

It’s a different story for big drug companies. Two days after Boulware’s submission, to market its drug molnupiravir, which in its clinical trial showed about as much effectiveness as fluvoxamine, and also had side effects like nausea and dizziness. Fluvoxamine also can cause insomnia and anxiety; molnupiravir is , male or female, having unprotected sex, because it caused genetic and fetal damage in test animals.

Still, federal guidelines in certain settings, and the government has bought for about $2.2 billion, or $733 per dose. Fluvoxamine, a generic, goes for less than $5 a pill.

“You hate to say that Big Pharma has a lot of influence, but clearly they do,” Boulware said. “The molnupiravir data was not that great, but we’re spending billions on the drug and it got fast-track emergency use authorization” while fluvoxamine remains in a gray area.

With the arrival of effective vaccines and the trickle of antiviral treatments, the urgency of rehabilitating old drugs for U.S. patients has ebbed. But the need remains high in lower- and middle-income countries where vaccines and new covid treatments remain unavailable.

It’s not rare for a pharmaceutical company to synthesize or study a drug for one purpose, only to discover it works better for something else. The classic instance is sildenafil, or Viagra, which was being developed as a drug for hypertension when scientists noticed a remarkable side effect. Remdesivir, now a front-line drug against covid, was aimed at treating Ebola.

It’s less common for a drug marketed for one use to acquire an entirely different purpose, but the pandemic drove scientists to try. They tested thousands of compounds in petri dishes for their virus-killing power, but the journey from test tube to human remedy is long, said Rayner, who is also a professor of pharmaceutical sciences at Monash University in Melbourne, Australia.

If fluvoxamine were a new drug, the company sponsoring it would have spent the money needed to get the drug approved and to show the FDA it has the means to monitor the drug’s safety and efficacy. Since it’s an old drug, it will be up to independent scientists, or perhaps a reluctant generics manufacturer, to sponsor safety monitoring should the FDA provide an emergency use authorization, Rayner said.

An EUA or approval “comes with strings. You have to continue to monitor the safety, to make sure no signals pop up when you move it from thousands to millions of patients,” he said. “That’s very expensive.”

U.S. physicians can prescribe drugs off label, but most are leery of doing so until a drug has won approval for the new use. That’s especially true now.

Definitive answers on some repurposed drugs were slow in coming because there were too many small, poorly designed studies by “every man and his dog,” Rayner said. He calculates up to $5.6 billion has been wasted on hydroxychloroquine clinical trials alone.

A recent called for better coordination and information-sharing among those organizing trials so that definitive answers can be obtained quickly with big pots of data.

As for camostat, Vinetz said those who took the drug felt better than those who got a placebo. “It basically prevented loss of smell and taste, which people really bitterly care about,” he said. “That means there’s a real biological effect. That merits further exploration.”

But will that happen? Vinetz’s team has sought publication of their research for five months with no success. He’d like to see whether camostat can prevent long covid, but such investigations cost millions. Camostat’s Japanese manufacturer as a covid drug after its own small, unsuccessful trial.

“When there’s no profit motive, it’s tough,” Vinetz said. Meanwhile, he’s resumed his research into controlling a neglected tropical disease: .

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Even as the war in Ukraine has prompted an exodus of international companies — from fast-food chains and oil producers to luxury retailers — from Russia, U.S. and global drug companies said they would continue manufacturing and selling their products there.

Airlines, automakers, banks, and technology giants — at least 320 companies — are among the businesses curtailing operations or making high-profile exits from Russia as its invasion of Ukraine intensifies. McDonald’s, Starbucks, and Coca-Cola announced a pause in sales this week.

But drugmakers, medical device manufacturers, and health care companies, which are exempted from U.S. and European sanctions, said Russians need access to medicines and medical equipment and contend that international humanitarian law requires they keep supply chains open.

“As a health care company, we have an important purpose, which is why at this time we continue to serve people in all countries in which we operate who depend on us for essential products, some life-sustaining,” said Scott Stoffel, divisional vice president for Illinois-based Abbott Laboratories, which manufactures and sells medicines in Russia for oncology, women’s health, pancreatic insufficiency, and liver health.

Johnson & Johnson — which has corporate offices in Moscow, Novosibirsk, St. Petersburg, and Yekaterinburg — said in a statement, “We remain committed to providing essential health products to those in need in Ukraine, Russia, and the region, in compliance with current sanctions and while adapting to the rapidly changing situation on the ground.”

The reluctance of drugmakers to pause operations in Russia is being met with a growing chorus of criticism.

Pharmaceutical companies that say they must continue to manufacture drugs in Russia for humanitarian reasons are “being misguided at best, cynical in the medium case, and outright deplorably misleading and deceptive,” said , a professor at the Yale School of Management who is have curtailed operations in Russia. He noted that banks and technology companies also provide essential services.

“Russians are put in a tragic position of unearned suffering. If we continue to make life palatable for them, then we are continuing to support the regime,” Sonnenfeld said. “These drug companies will be seen as complicit with the most vicious operation on the planet. Instead of protecting life, they are going to be seen as destroying life. The goal here is to show that Putin is not in control of all sectors of the economy.”

U.S. pharmaceutical and medical companies have operated in Russia for decades, and many ramped up operations after Russia invaded and annexed Crimea in 2014, navigating the fraught relationship between the U.S. and Russia amid sanctions. In 2010, Vladimir Putin, then Russian prime minister, announced an ambitious national plan for the Russian pharmaceutical industry that would be a pillar in his efforts to reestablish his country as an influential superpower and wean the country off Western pharmaceutical imports. Under the plan, called “Pharma-2020” and “Pharma-2030,” the government required Western pharmaceutical companies eager to sell to Russia’s growing middle class to locate production inside the country.

Pfizer, Johnson & Johnson, Novartis, and Abbott are among the drugmakers that manufacture pharmaceutical drugs at facilities in St. Petersburg and elsewhere in the country and typically sell those drugs as branded generics or under Russian brands.

Pfizer’s CEO, Albert Bourla, said on CBS that the giant drugmaker is not going to make further investments in Russia, but that it will not cut ties with Russia, as multinational companies in other industries are doing.

Pharmaceutical manufacturing plants in Kaluga, a major manufacturing center for Volkswagen and Volvo southwest of Moscow, have been funded through a partnership between Rusnano, a state-owned venture that promotes the development of high-tech enterprises, and U.S. venture capital firms.

Russia also has sought to position itself as an attractive research market, offering an inexpensive and lax regulatory environment for clinical drug trials. Last year, Pfizer conducted in Russia clinical trials of Paxlovid, its experimental antiviral pill to treat covid-19. Before the invasion began in late February, 3,072 trials were underway in Russia and 503 were underway in Ukraine, according to BioWorld, a reporting hub focused on drug development that features data from Cortellis.

AstraZeneca is the top sponsor of clinical trials in Russia, with 49 trials, followed by a subsidiary of Merck, with 48 trials.

So far, drugmakers’ response to the Ukraine invasion has largely centered on public pledges to donate essential medicines and vaccines to Ukrainian patients and refugees. They’ve also made general comments about the need to keep open the supply of medicines flowing within Russia.

$2 million to support humanitarian efforts in Ukraine, and Pfizer, based in New York, said it has supplied $1 million in humanitarian grants. Swiss drug maker Novartis said it was expanding humanitarian efforts in Ukraine and working to “ensure the continued supply of our medicines in Ukraine.”

But no major pharmaceutical or medical device maker has announced plans to shutter manufacturing plants or halt sales inside Russia.

In , hundreds of leaders of mainly smaller biotechnology companies have called on industry members to cease business activities in Russia, including “investment in Russian companies and new investment within the borders of Russia,” and to halt trade and collaboration with Russian companies, except for supplying food and medicines. How many of the signatories have business operations in Russia was unclear.

, director for market access at Janssen, a Johnson & Johnson company, was among those who signed the letter, but whether he was speaking for the company was unclear. In its own statement , the company said it’s “committed to providing access to our essential medical products in the countries where we operate, in compliance with current international sanctions.”

GlaxoSmithKline, headquartered in the United Kingdom, said that it’s stopping all advertising in Russia and will not enter into contracts that “directly support the Russian administration or military.” But the company said that as a “supplier of needed medicines, vaccines and everyday health products, we have a responsibility to do all we can to make them available. For this reason, we will continue to supply our products to the people of Russia, while we can.”

Nell Minow, vice chair of ValueEdge Advisors, an investment consulting firm, noted that drug companies have been treated differently than other industries during previous global conflicts. For example, some corporate ethicists advised against pharmaceutical companies’ total divestment from South Africa’s apartheid regime to ensure essential medicines flowed to the country.

“There is a difference between a hamburger and a pill,” Minow said. Companies should strongly condemn Russia’s actions, she said, but unless the U.S. enters directly into a war with Russia, companies that make essential medicines and health care products should continue to operate. Before U.S. involvement in World War II, she added, there were “some American companies that did business with Germany until the last minute.”

KHN senior correspondent Arthur Allen contributed to this article.

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But you’d be wrong.

Opinions about Aduhelm (also known as aducanumab) in the dementia community are diverse, ranging from “we want the government to cover this drug” to “we’re concerned about this medication and think it should be studied further.”

The Alzheimer’s Association and UsAgainstAlzheimer’s, the most influential advocacy organizations in the field, are in the former camp.

Both are pushing for Medicare to cover Aduhelm’s $28,000 annual cost and fiercely oppose the Centers for Medicare & Medicaid Services’ to restrict coverage only to people enrolled in clinical trials. Nearly were received on that proposal, and a final decision is expected in April.

“With respect, we have no more time for debate or delay,” the Alzheimer’s Association national Early-Stage Advisory Group wrote in a Feb. 10 comment. “Every passing day without access to potential treatments subjects us to a future of irreversible decline.” For its part, UsAgainstAlzheimer’s called CMS’ proposal “anti-patient.”

Yet the scientific evidence behind Aduhelm is inconclusive, its efficacy in preventing the progression of Alzheimer’s remains unproved, and there are concerns about its safety. The FDA to the medication last June but ordered the drugmaker, Biogen, to conduct a new clinical trial to verify its benefit. And the agency’s decision came despite a 10-0 recommendation against doing so from its scientific advisory committee. (One committee member abstained, citing uncertainty.)

Other organizations representing people living with dementia are more cautious, calling for more research about Aduhelm’s effectiveness and potential side effects. More than 40% of people who take the medication have swelling or bleeding in the brain — complications that need to be carefully monitored.

The Dementia Action Alliance, which supports people living with dementia, is among them. In a statement forwarded to me by CEO Karen Love, the organization said, “DAA strongly supports CMS’s decision to limit access to aducanumab to people enrolled in qualifying clinical trials in order to better study aducanumab’s efficacy and adverse effects.”

Meanwhile, Dementia Alliance International — the world’s largest organization run by and for people with dementia, with more than 5,000 members — has not taken a position on Aduhelm. “We felt that coming out with a statement on one side or another would split our organization,” said Diana Blackwelder, its treasurer, who lives in Washington, D.C.

Blackwelder, 60, who was diagnosed with early-onset Alzheimer’s in 2017, told me, “To say that millions of people afflicted with a disease are all up in arms against CMS’s proposal is just wrong. We’re all individuals, not a collective.”

“I understand the need for hope,” she said, expressing a personal opinion, “but people living with dementia need to be protected as well. This drug has very serious, frequent side effects. My concern is that whatever CMS decides, they at least put in some guardrails so that people taking this drug get proper workups and monitoring.”

The debate over Medicare’s decision on Aduhelm is crucial, since most people with Alzheimer’s are older or seriously disabled and covered by the government health program.

To learn more, I talked to several people living with dementia. Here’s some of what they told me:

Jay Reinstein, 60, is married and lives in Raleigh, North Carolina. He was diagnosed with early-onset Alzheimer’s disease three years ago and formerly served on the national board of directors of the Alzheimer’s Association.

“I understand [Aduhelm] is controversial, but to me it’s a risk I’m willing to take because there’s nothing else out there,” Reinstein said, noting that people he’s met through support groups have progressed in their disease very quickly. “Even if it’s a 10% chance of slowing [Alzheimer’s] down by six months, I am still willing to take it. While I am progressing slowly, I want more time.”

Laurie Scherrer of Albertville, Alabama, was diagnosed with early-onset Alzheimer’s and in 2013, at age 55.

Early on, she was prescribed Aricept (donepezil), one of a that address Alzheimer’s symptoms. “I became totally confused and disoriented, I couldn’t think, I couldn’t concentrate,” she told me. After stopping the medication, those symptoms went away.

“I am not for CMS approving this drug, and I wouldn’t take it,” Scherrer said. At discussion groups on Aduhelm hosted by the Dementia Action Alliance (Scherrer is on the board), only two of 50 participants wanted the drug to be made widely available. The reason, she said: “They don’t think there are enough benefits to counteract the possible harms.”

Rebecca Chopp, 69, of Broomfield, Colorado, was diagnosed with early-onset Alzheimer’s in March 2019. She’s a former chancellor of the University of Denver.

Chopp is a member of a newly formed group of five people with dementia who meet regularly, “support one another,” and want to “tell the story of Alzheimer’s from our perspective,” she said.

Two people in the group have taken Aduhelm, and both report that it has improved their well-being. “I believe in science, and I am very respectful of the large number of scientists who feel that [Aduhelm] should not have been approved,” she told me. “But I’m equally compassionate toward those who are desperate and who feel this [drug] might help them.”

Chopp opposes CMS’ decision because “Aduhelm has been FDA-approved and I think it should be funded for those who choose to take it.”

Joanna Fix, 53, of Colorado Springs was diagnosed with early-onset Alzheimer’s disease in October 2016. She, too, developed serious complications after taking Aricept and another dementia medication, Namenda (memantine).

“I would love it if tomorrow somebody said, ‘Here’s something that can cure you,’ but I don’t think we’re at that point with Aduhelm,” Fix told me. “We haven’t been looking at this [drug] long enough. It feels like this is just throwing something at the disease because there’s nothing else to do.”

“Please, please take it from someone living with this disease: There is more to life than taking a magic pill,” Fix continued. “All I care about is my quality of life. My marriage. Educating and helping other people living with dementia. And what I can still do day to day.”

Phil Gutis, 60, of Solebury, Pennsylvania, has participated in clinical trials and taken Aduhelm for 5½ years after being diagnosed with early-onset Alzheimer’s in 2016.

He’s convinced the medication has helped him. “I don’t know how to describe it other than to say my head feels so much clearer now,” he told me. “I feel much more capable of doing things now. It’s not like I’ve gained my memories back, but I certainly haven’t deteriorated.”

Gutis thinks CMS’ proposed restrictions on Aduhelm are misguided. “When the FDA approved it, there was this sense of excitement — oh, we’re getting somewhere. With the CMS decision, I feel we are setting the field back again. It’s this constant feeling that progress is being made and then — whack.”

Christine Thelker, 62, is a widow who lives alone in Vernon, British Columbia. She was diagnosed with vascular dementia seven years ago and is a board member for Dementia Advocacy Canada, which supports restrictions on Aduhelm’s availability.

“Most of us who live with dementia understand a cure is not likely: There are too many different types of dementia, and it’s just too complicated,” Thelker told me. “To think we’re just going to take a pill and be better is not realistic. Don’t give us false hope.”

What people with Alzheimer’s and other types of dementia need, instead, is “various types of rehabilitation and assistance that can improve our quality of life and help us maintain a sense of hope and purpose,” Thelker said.

Jim Taylor of New York City and Sherman, Connecticut, is a caregiver for his wife, Geri Taylor, 78, who has moderate Alzheimer’s. She joined a clinical trial for Aduhelm in 2015 and has been on the drug since, with the exception of about 12 months when Biogen temporarily stopped the clinical trial. “In that period, her short-term memory and communications skills noticeably declined,” Jim Taylor said.

“We’re convinced the medication is a good thing, though we know it’s not helpful for everybody,” Taylor continued. “It really boosts [Geri’s] spirits to think she’s part of research and doing everything she can.

“If it’s helpful for some and it can be monitored so that any side effects are caught in a timely way, then I think [Aduhelm] should be available. That decision should be left up to the person with the disease and their care partner.”

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So it’s no surprise that some of the same drugs are being reformulated and renamed by manufacturers as a new obesity treatment. No longer limited to the crowded field of treatments for Type 2 diabetes, which of Americans, they join the far smaller number of drugs for obesity, and is ready to be mined for profit.

One that recently hit the market — winning Food and Drug Administration approval in June — is Novo Nordisk’s Wegovy, a higher-dose version of the company’s injectable diabetes drug, Ozempic.

Ozempic’s peppy ads suggest that people who use it might lose weight, but also include a disclaimer: that it “is not a weight loss drug.” Now — with a new name — it is. And clinical trials showed for many patients.

“People who go on this medication lose more weight than with any drug we’ve seen, ever,” said Dr. Fatima Cody Stanford, an obesity medicine specialist at Massachusetts General Hospital and Harvard Medical School who was not involved with any of the clinical trials.

But that leaves employers and insurers in the uncomfortable position of deciding if it’s worth it.

Wegovy’s monthly wholesale price tag — set at $1,349 — is about 58% more than Ozempic’s, although, the company points out, the drug’s injector pens contain more than twice as much of the active ingredient. Studies so far show that patients may need to take it indefinitely to maintain weight loss, translating to a tab that could top $323,000 over 20 years at the current price. Weight loss treatments are not universally covered by insurance policies.

The arrival of this new class of weight loss drugs — may soon follow — has created a thicket of issues for those who will pay for them. The decision is complicated by many unknowables concerning their long-term use and whether competition might eventually lower the price.

“The metric we try to use is value,” said James Gelfand, senior vice president for health policy at the ERISA Industry Committee, or ERIC, which represents large, self-insured employers. “If we pay for this drug, how much is this going to cost and how much value will it provide to the beneficiaries?”

have had a lackluster past in this regard, with only modest results. Many employers and insurers likely remember , a combination of fenfluramine and dexfenfluramine that was pulled from the market in the late 1990s for causing heart valve problems.

New drugs like Wegovy, more effective but also pricier than previous weight loss treatments, will add more fuel to that debate.

Past treatments were shown to prompt weight loss in the range of 5% to 10% of body weight. But many had relatively serious or unpleasant .

Wegovy, however, helped patients lose an average of 15% of their body weight over 68 weeks in the that led to its approval. A comparison group that got a placebo injection lost an average of 2.5% over the same period. On the high end, nearly a third of patients in the treatment group lost 20% or more. Both groups had counseling on diet and exercise.

Side effects, generally considered mild, included nausea, diarrhea, vomiting and constipation. A few patients developed pancreatitis, a serious inflammation of the pancreas. Like the diabetes medication, the drug carries a warning about a potential risk of a type of thyroid cancer.

Weight loss in those taking Wegovy puts it close to the 20% to 25% losses seen with bariatric surgery, said Stanford at Mass General, and well above the 3% to 4% seen with diet and other lifestyle changes alone.

Participants also saw reductions in their waistlines and improvements in their blood pressure and blood sugar levels, which may mean they won’t develop diabetes, said Dr. Sean Wharton, an internal medicine specialist and adjunct professor at York University in Toronto who was among the co-authors of the report outlining the results of the on Wegovy.

Since weight loss is known to reduce the risk of heart attack, high blood pressure and diabetes, might the new drug type be worth it?

Covering such treatment would be a sea change for Medicare, which specifically bars coverage for obesity medications or drugs for “anorexia, weight loss or weight gain,” although it does pay for bariatric surgery. Pharmaceutical companies, patient advocates and some medical professionals are backing proposed federal legislation to allow coverage. But the legislation, the Treat and Reduce Obesity Act, has not made progress despite being reintroduced every year since 2012, and sponsors are now instead to rewrite existing rules.

Private insurers will have to consider a cost-benefit analysis of adding Wegovy to their list of covered treatments, either broadly or with limits. Obesity was first recognized as a disease by the American Medical Association, easing the path for insurance coverage, in .

“Employers are going to have a bit of a challenge” deciding whether to add the benefit to insurance offerings, said Steve Pearson, founder and president of the Institute for Clinical and Economic Review, which provides cost-benefit analyses of medical treatments but has not yet looked at Wegovy.

The trade-offs are embodied in patients like Phylander Pannell, a 49-year-old Largo, Maryland, woman who said she lost 65 pounds in a clinical trial of Wegovy. That study gave the drug to all participants for the first 20 weeks, then randomly assigned patients to get either the drug or a placebo for the next 48 weeks to determine what happens when the medication is stopped. Only after the trial ended did she find out she was in the treatment group the entire time.

Her weight fell slowly at first, then ramped up, eventually bringing her 190-pound frame down to about 125. Pains in her joints eased; she felt better all around.

“I definitely feel the drug was it for me,” said Pannell, who also followed the trial’s guidance on diet and exercise.

The found that both groups lost weight in the initial 20 weeks, but those who continued to get the drug lost an additional average of 7.9% of their body weight. Those who got a placebo gained back nearly 7%.

After the trial ended, and the covid-19 pandemic hit, Pannell regained some weight and is now at 155. She is eager to get back on the medication and hopes her job-based insurance will cover it.

Many employers do cover obesity drugs. For example, about 40% of private employer plans include Novo Nordisk’s once-daily injection called Saxenda on their health plans, said Michael Bachner, Novo Nordisk’s director of media relations.

He said the $1,349-a-month wholesale acquisition price of Wegovy was determined by making it equivalent to that of Saxenda, which is less effective.

Still, that is more than the $851 monthly wholesale price of Ozempic. But, he points out, the recommended dosage of Wegovy is more than twice that of Ozempic. Four milligrams come in the Ozempic injector pens for the month, while Wegovy has 9.6.

“There’s more drug in the pen,” Bachner said. “That drives the price up.”

He added: “This is not a 20-year-old drug that we now have a new indication for and are pricing it higher. It’s a whole different clinical program,” which required new trials.

Now scientists, employers, physicians and patients will have to decide whether the new drugs are worth it.

Earlier estimates — some commissioned by Novo Nordisk — of the potential cost of adding an obesity drug benefit to Medicare showed an overall reduction in spending when better health from the resulting weight loss was factored in.

Still, those considered much less expensive drugs, including a range of generic and branded drugs costing as little as $7 a month to more than $300, a small fraction of Wegovy’s cost.

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“There’s lots of things that she used to be able to do just fine,” said her mom, Londen Tabor, who lives with her daughter in Gillette, Wyoming. Autumn’s speech has become slurred and her cognitive skills slower. She needs help with many tasks, such as writing, showering and dressing, and while she can walk, her balance is off.

Autumn has been turned down for clinical trials because she is too young.

“It is so frustrating to me,” Tabor said. “I would sell my soul to try to get any type [of treatment] to help my daughter.”

For patients like Autumn with serious or immediately life-threatening conditions who do not qualify for clinical trials and have exhausted all treatment options, there may be another option: seeking approval from the Food and Drug Administration for , or compassionate use, of experimental therapies.

Definitive numbers are hard to find, but studies from researchers, actions by drugmakers and insights from experts suggest that getting expanded access to unproven therapies for rare diseases is more difficult than for more common illnesses, such as cancer.

Even with experimental treatments on the rise, patients with rare diseases frequently face an unwillingness by drug companies to provide them before clinical studies are completed. Developing drugs for these diseases is an especially fragile process because the patient populations are small and often diverse, having different genetics, symptoms and other characteristics, which makes studying the drugs’ effects difficult.

Drugmakers believe offering a drug before studies are finished could impair its development and jeopardize FDA approval.

Companies working on therapies for rare diseases, especially smaller ones, could feel those repercussions acutely, said Lisa Kearns, a researcher in the ethics division of New York University’s medical school and member of the division’s working group on compassionate use and preapproval access. “There’s not as much investment in rare diseases, so an [adverse] event could frighten the already limited number of potential investors.”

Drugs that were not made available for compassionate use last year until studies were completed include , ; Enspryng, for an ; and , .

A spokesperson for Roche, which makes Evrysdi and Enspryng and is working on a treatment for Huntington’s disease, said the decision was tied not to the type of disease but to company policy: Roche does not set up expanded access programs for any drugs until results are available from a phase 3 clinical trial. (Those phase 3 studies are typically the last testing done before the company seeks drug approval.)

Another company’s experimental drug for , an autoimmune disease that leads to skeletal muscle weakness, similarly was not available through until , and no programs have started for a therapy being studied in and , a fatal neurodegenerative disease often referred to as Lou Gehrig’s disease.

One slight, but notable, deviation: Drugmaker Biogen this year to allow certain ALS patients to receive an experimental drug , after the testing was to be completed but before the results are known.

Dr. Merit Cudkowicz, a neurologist at Massachusetts General Hospital in Boston, has helped patients get therapies through expanded access. Since September 2018, she and colleagues being developed by drug companies, but only about 120 patients have received therapies this way. More than 16,000 people in the United States in 2015 to have ALS and do not qualify for clinical trials because of the progression of their disease or very strict eligibility requirements.

These examples contrast with some drugs for more common problems. , for leukemia, was offered to thousands of patients through expanded access programs before the manufacturer completed the clinical studies that led to FDA approval. , for HIV/AIDS, and , for the most common type of lung cancer, were similarly offered to large numbers of patients even as clinical trials were ongoing.

Last year, Novartis gave more than 7,000 patients worldwide early access to cancer drugs.

Doctors also report that getting experimental drugs for cancer patients is relatively simple. More than 200 physicians around the country were surveyed, and among those who applied for access, said they had secured drugs still being investigated for patients who were not responding to approved therapies.

California researchers found similar trends in a of 23 social media campaigns launched by patients between 2005 and 2015 seeking a variety of experimental treatments. While seven of the 19 patients with cancer received early access to requested drugs, no access was allowed for three patients with rare diseases, although one of those patients was allowed to enroll in a clinical trial.

Companies base their decisions on whether to provide a therapy through expanded access on a number of factors, said Jess Rabourn, CEO of WideTrial, which helps pharmaceutical companies run compassionate use programs. In general, there should be evidence that patients can tolerate the treatment and an expectation that any benefit outweighs the risk, he said.

“This idea that you have to wait until the research is done is baloney,” he said. “We’re talking about patients who are going to die if they’re told to wait.”

But drugmakers often view it differently, even though evidence suggests that granting early access drug approval.

Kearns explained that companies often wait until phase 3, or after, because they can be “relatively” confident of a drug’s safety and effectiveness. “They don’t want to harm patients, of course, but they also do not want to threaten the drug’s eventual regulatory approval with an adverse event in [a] very sick patient population.”

Melissa Hogan, who consults on clinical trials for rare diseases and is an , attributes the lack of access to the high cost of therapies and the tightknit nature of the rare disease community, where patients and their families often set up social media groups and exchange ideas and treatment plans. Companies “know that if one patient gains access, other patients will know” and ask for access, said Hogan, who has a son with mucopolysaccharidosis type II. That could overwhelm small drugmakers with little manufacturing capacity.

These concerns cause “many companies [to] just throw up their hands and take a hard line of no [expanded access] until they reach approval stage,” said Hogan.

The 2018 offers another option for some patients. Unlike expanded access, the law applies only to requests for medicines — not medical devices — and does not require approval from the FDA or an , a committee that reviews and monitors people participating in research for their protection. The legislation, however, doesn’t oblige companies to grant a request.

For Cali Orsulak, expanded access may be her husband’s only option. He was diagnosed with ALS in 2019 at age 43.

“We did our best with the skill level we had to search clinical trials all over Canada and the U.S., and then covid hit and it became increasingly difficult,” said Orsulak, explaining that they live in Canada but seek medical care in the United States. “Now that my husband has progressed, it’s even harder to get into clinical trials.”

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By that evening, though, he had a fever of 101 degrees and was clearly ill. “I felt like I had been hit by a truck,” recalled Mutter, who lives near Seattle.

The next day he was diagnosed with COVID-19. By Saturday, the 58-year-old was enrolled in a clinical trial for the same antibody cocktail that President Donald Trump claimed was responsible for his

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