WASHINGTON 鈥 Wearing a blue聽T-shirt and worn jeans, Marc Yale stood near the welcome pole at the annual Rare Disease Legislative Advocates conference handing out money.
Envelopes in his left hand held $750 checks to help patients with a rare disease cover the cost of a whirlwind trip to the nation鈥檚 capital. The money was donated by the pharmaceutical industry.
鈥淥bviously, we wanted to send as many patients to advocate on Capitol Hill as possible,鈥 said Yale, the director of the International Pemphigus & Pemphigoid Foundation.
Yale collected $7,500 in donations from a specialty pharmacy and a drugmaker that manufactures treatments for the rare diseases that afflict his members. The money was intended to help offset patients鈥 travel expenses.
The pemphigus/pemphigoid group, representing people with聽a rare autoimmune disease that often聽affects the skin and mucus membranes, was just one of many small patient advocacy organizations represented at the late February conference, where sick patients and family members learn how to lobby and test their new skills. Rare diseases in the U.S. are defined as those affecting fewer than 200,000 people.
The pharmaceutical industry is teaming up with advocacy groups that are training and even paying聽for patients who need their medicines to promote their causes in Washington.
National polls identify high drug prices as Americans鈥 No. 1聽health care complaint, and President Donald Trump has declared that pharmaceutical companies are 鈥済etting away with murder.鈥
But these behind-the-scenes partnerships between the pharmaceutical industry and advocacy groups may work against lowering the price of high-price drugs. Critics say vulnerable patients are being manipulated and the goals promoted are skewed by the pharma benefactors who want faster government approval for new products and to get insurers or government programs to pay for them, whatever the price tag.
Dr. Ezekiel Emanuel, a bioethicist who has studied the issue, said he questions whether patient advocacy groups truly are 鈥渨hite knights defending the good fight.鈥澛燞e said research suggests that the conflicts of interest that occur when drug companies train and finance patient groups are 鈥減retty rampant.鈥 Emanuel co-authored a March that found 83 percent of the 104 largest聽patient advocacy groups take money from the drug, medical device and biotech industries. Smaller organizations are even more likely to be disproportionately dependent on industry funding for their operating budgets, he said.
Among repeat attendees at this year鈥檚 conference were 17-year-olds Shira Strongin and Emily Muller, who both suffer from a debilitating connective tissue disease known as Ehlers-Danlos Syndromes.
Strongin said the 鈥減atient voice鈥 has helped pass laws, such as the 21st Century Cures Act that patients聽lobbied Congress for the previous year.
鈥淗earing personal stories makes things just hit home more,鈥 she added.
Muller and Strongin give their presentation on advocacy at the Rare Disease Legislative Advocates conference. (H. Darr Beiser for KHN)
But some conference first-timers聽were skeptical about the motivations: 鈥淲hat I鈥檓 seeing just doesn鈥檛 feel right,鈥 said Aaron Motschenbacher, who participated because his three young children suffer from deadly and progressive multiple sulfatase deficiency, a rare hereditary metabolic disorder.
Before paying his own way to take an early flight home, he complained that patients and their families had no 鈥渋nvolvement drafting, creating or discussing鈥 the messaging delivered to Congress, adding: 鈥淚t鈥檚 like marketing, I鈥檓 pushing an agenda by and through a sob story.鈥
Sharing Their Stories
The patient-lobbying conference, organized by the EveryLife Foundation for Rare Diseases, underlines how the financial interests of manufacturers and the medical needs of patients are intertwined.
More than 300聽patients and advocates attended, and nearly all聽took part in a subsequent 鈥淟obby Day鈥 to visit congressional staff and lawmakers. They permitted a reporter from Kaiser Health News to observe聽and also join in a reception showcasing art by some of the patients.
Many patients were visibly sick or terminally ill. When deployed to pay visits to politicians, they add a human face to lobbying efforts around proposed legislation that affects pharma. Legislation like the Cures Act might increase spending on drug development or grease the pathway of drugs to market and with fewer regulations.
Before going to Capitol Hill, the patients and their families underwent聽a day of training, learning how to tell their stories. If at a loss for what to talk about, they were provided talking points on what EveryLife staffers called potential 鈥渁sks.鈥
The group鈥檚 president, Dr. Emil Kakkis, is a drug industry executive. He said the foundation doesn鈥檛 鈥渢ell patients what to do on the Hill. They are given options.鈥
During one session called 鈥淭ricks of the Trade: Preparing for a Successful Meeting,鈥 Soapbox Consulting chief executive Christopher Kush walked the audience through logistics for the next day.
The attendees were given a mobile app, which shows each advocate鈥檚 prearranged meeting list. Checking a map, Kush looked at the audience and said: 鈥淚f you see a little dot where you live, you may have a new member of Congress 鈥 or a green check on your state, that means you have a new senator.鈥
He asked the audience to raise their hands if they saw dots and checks.
鈥淥h, here you come,鈥 Kush said. 鈥淚t鈥檚 going to be like an army walking up the hallways [of Congress]. I just got chills.鈥
The foundation also enlists patient advocates to woo Congress聽members to join its rare disease caucus聽and to support legislation to expedite drug development.
For attendees, the meetings do have benefits.
Advocate Janice Ragazzo said EveryLife training and Hill visits taught her how to create a disease registry, grow her disease organization鈥檚 outreach and how to talk with regulators. Her goal is to develop federal billing codes that could help track the prevalence of her daughter鈥檚 genetic disease.
They also learn from each other: Lisa Douthit, the leader of a different rare disease group, shook her head admiringly as Yale explained his fundraising strategy as he passed out the $750 checks.
鈥淚t鈥檚 great PR for the pharmaceutical company,鈥 she said.
More than that, perhaps, such events allow parents and patients with rare disease to connect with each other 鈥 many are delighted to realize they鈥檙e not alone in their suffering.
鈥淚鈥檝e watched kids sometimes who felt so timid and afraid and shy to share their stories learn to embrace it as part of their life,鈥 Muller said. 鈥淎nd I think that鈥檚 been the most incredible part for me.鈥
Strongin injects fondaparinux, a blood thinner she uses to prevent transient ischemic attacks, as Muller watches. (H. Darr Beiser for KHN)
A Coordinated Strategy
One focus of this year鈥檚 conference was the OPEN Act, which provides a financial reward for drugmakers who do clinical trials to repurpose an existing drug聽to treat聽a rare disease. A white paper explained the bill to participants and gave tips for聽what to say on the Hill.
In what appeared to be a coordinated strategy, Rep. Gus Bilirakis (R-Fla.)聽聽the OPEN (officially the Orphan Product Extensions Now, Accelerating Cures and Treatments) Act on the House floor聽the week of the conference. Bilirakis has worked with EveryLife on the bill for several years and it had been included in the House version of the聽Cures Act, though it was dropped from the final law. More than 220 patient organizations have signed on to support it and it has steadily gained co-sponsors, said EveryLife鈥檚 Julia Jenkins. Jenkins said the bill is expected to be introduced in the Senate soon.
If聽approved, it would give drugmakers six months of extra market exclusivity to repurpose a drug in addition to the patents or other exclusivity periods it may already have.
EveryLife wrote in a March Health Affairs that the law would 鈥渄ouble the number of treatments available鈥 and generate lower-cost medicines for the rare disease community.
But in a , Harvard鈥檚 Dr. Aaron Kesselheim argued that the law offers unnecessary incentives for manufacturers that already have blockbuster drugs.
Mixed Motivations
The EveryLife Foundation鈥檚 Rare Giving program covered about 100 travel stipends for conference attendees. While that is largely paid for by the foundation鈥檚 board members, including Kakkis, Jenkins said the group is聽鈥渁ctively trying to get industry to fund the program.鈥
Top donors to EveryLife include pharmaceutical companies AbbVie, Alexion, BioMarin, Raptor Pharmaceutical and the Kakkis family,聽according to the foundation鈥檚聽most recent annual report. EveryLife聽 income of nearly $1.8 million in 2015.
Kakkis, a successful medical researcher, founded Novato, Calif.-based Ultragenyx Pharmaceutical in 2010 to develop more ultra-rare disease drugs. Last year, he earned a base salary of $567,200 with a potential performance-based cash bonus that could equal up to 60 percent of his salary, according to corporate financial .
Emil Kakkis, president and founder of EveryLife Foundation (H. Darr Beiser for KHN)
Kakkis is forthright about wearing multiple hats 鈥 posting his affiliations聽openly on his on the Ultragenyx website. With a charming and compelling personality, he is also well-known for his ability to get things done.
Late last year, Kakkis鈥 foundation 鈥 with his army of advocates 鈥 lobbied for the Cures Act. One of the patients, Max Schill, was the little boy who 聽congressional leaders as they signed the bill to send to President Barack Obama. Key backers included EveryLife鈥檚 legislative caucus for rare diseases, which counts more than 100 House members and five senators among its ranks. Since 2011, Kakkis has individually contributed $48,100 to political committees, including $6,000 to Bilirakis.
Kakkis explained in a June 2010 Senate appropriations subcommittee hearing that he started EveryLife after working through the Food and Drug Administration鈥檚 process to win approval for Aldurazyme, an enzyme replacement therapy he developed to treat one form (MPS I) of a聽group of disorders known as mucopolysaccharidoses.
Eventually the drug was approved, he said, but with聽a three-year delay. And, during that time, 鈥渁 number of MPS I patients passed away,鈥 Kakkis said.
In Kakkis鈥 view, drug companies and patients have a shared interest in getting drugs developed and approved.
鈥淚 do think the company鈥檚 interest [and patients鈥 interests] are not misaligned,鈥 he said.
鈥楾hat鈥檚 The Way Washington Works鈥
Emanuel said he believes that patient advocacy groups should openly state their potential conflicts while participating in regulatory meetings. In addition, Emanuel said, drug and device manufacturers should 聽how much they pay patient advocacy groups just as they do with physicians and teaching hospitals.
In response to rising conflict-of-interest concerns, the National Health Council, a coalition of patient advocacy groups and other organizations, updated its in March and now requires member advocacy groups to divulge all contributions from pharmaceutical, biotechnology or device companies that exceed a certain threshold.
Dr. Christopher Austin of the National Institutes of Health invited several patient advocacy groups 鈥 including EveryLife 鈥 to speak at NIH during the agency鈥檚 annual rare disease day.
鈥淚 have no idea what their funding is, we just look at the work they do,鈥 said Austin, director of the National Center for Advancing Translational Sciences.
Speaker Max Bronstein, EveryLife鈥檚 chief advocacy and science policy officer, thanked the audience for advocating on behalf of the 21st Century Cures Act and talked about the OPEN Act, saying it is 鈥渢he legislation we鈥檝e been working on for a few years.鈥
Austin said he was aware that Kakkis was both the head of EveryLife as well as Ultragenyx.
鈥淵ou know, Washington is full of those people,鈥 Austin said. 鈥淎s I鈥檝e learned being here, that鈥檚 the way Washington works, for better or worse. It鈥檚 a little stinky frankly, but there you are.鈥
KHN鈥檚 coverage of prescription drug development, costs and pricing is supported in part by the .