Para VanOrden, como para millones de estadounidenses, esa es la causa más común de , una afección en la que la tiroides, una glándula con forma de mariposa ubicada en el cuello, no produce suficientes hormonas necesarias para que el cuerpo regule el metabolismo.

No hay cura para la enfermedad de Hashimoto o el hipotiroidismo. Pero VanOrden, que vive en Atenas, Texas, comenzó a tomar levotiroxina, una hormona tiroidea sintética muy recetada, que se usa para tratar síntomas comunes, como fatiga, aumento de peso, caída del cabello y sensibilidad al frío.

A la mayoría de los pacientes les va bien con la levotiroxina y sus síntomas desaparecen. Sin embargo, para otros, como VanOrden, el fármaco no es tan eficaz.

Para la mujer, significó ir de médico en médico, de prueba en prueba y de tratamiento en tratamiento, gastando alrededor de $5,000 al año.

“Luzco y actúo como una persona bastante enérgica”, dijo VanOrden, de 38 años, explicando que sus síntomas no son visibles. “Pero hay un agujero en mi tanque de gasolina”, dijo. Y “el estrés hace que el agujero sea más grande”.

Las enfermedades autoinmunes ocurren cuando el sistema inmunológico ataca y daña por error células y tejidos sanos. Otros ejemplos comunes incluyen la artritis reumatoide, el lupus, la enfermedad celíaca y la enfermedad inflamatoria intestinal. Hay más de , que afectan a , desproporcionadamente a las mujeres. En general, el costo del tratamiento de enfermedades autoinmunes en el país se estima en .

A pesar de ser muy frecuentes, encontrar ayuda para muchas enfermedades autoinmunes puede resultar frustrante y costoso. Recibir un diagnóstico puede ser un obstáculo importante porque la gama de síntomas se parece mucho a la de otras afecciones y, a menudo, no existen pruebas de identificación definitivas, explicó Sam Lim, director clínico de la División de Reumatología de la Facultad de Medicina de la Universidad Emory en Atlanta.

Además, algunos pacientes sienten que tienen que luchar para que les crean, incluso el médico. Y después de un diagnóstico, gastan fortunas explorando opciones de tratamiento.

“A menudo están molestos. Los pacientes se sienten desestimados”, dijo Elizabeth McAninch, endocrinóloga y experta en tiroides de la Universidad de Stanford, sobre algunos pacientes que acuden a ella en busca de ayuda.

La educación médica insuficiente y la falta de inversión en nuevas investigaciones son dos factores que dificultan la comprensión general del hipotiroidismo, según Antonio Bianco, endocrinólogo de la Universidad de Chicago y experto líder en esta afección.

Algunos pacientes se enojan cuando sus síntomas no responden a los tratamientos estándar, ya sea levotiroxina sola o en combinación con otra hormona, dijo Douglas Ross, endocrinólogo del Hospital General de Massachusetts, en Boston. “Tendremos que permanecer abiertos a la posibilidad de que nos falte algo aquí”, dijo.

Jennifer Ryan, de 42 años, dijo que ha gastado “miles de dólares de su bolsillo” buscando respuestas. Los médicos no recomendaron medicamentos con hormona tiroidea a la residente de Huntsville, Alabama, diagnosticada con Hashimoto después de años de fatiga y aumento de peso, porque sus niveles parecían normales. Recientemente cambió de médico y tiene esperanzas.

“No andas sufriendo todo el día y no tienes nada malo”, dijo Ryan.

Y las aseguradoras de salud suelen negar la cobertura de nuevos tratamientos para el hipotiroidismo, dijo Brittany Henderson, endocrinóloga y fundadora del Charleston Thyroid Center en Carolina del Sur, que atiende a pacientes de los 50 estados. “Las compañías de seguros quieren que se utilicen genéricos a pesar de que a muchos pacientes no les va bien con estos tratamientos”, dijo.

Mientras tanto, la magnitud de los problemas de tiroides de los estadounidenses se puede ver en la venta de medicamentos. La levotiroxina se encuentra entre los cada año en el país. Sin embargo, las investigaciones apuntan a una del fármaco en personas con hipotiroidismo leve.

Un , financiado por AbbVie (fabricante de Synthroid, una versión de marca de levotiroxina) dijo que una base de datos de reclamos médicos y farmacéuticos mostró que la prevalencia del hipotiroidismo, incluidas las formas más leves, aumentó del 9,5% de los estadounidenses en 2012 al 11,7%. en 2019.

El número de personas diagnosticadas aumentará a medida que la población envejezca, afirmó McAninch. Los disruptores endócrinos (químicos naturales o sintéticos que pueden afectar las hormonas) podrían explicar parte de ese aumento, dijo.

En su búsqueda de respuestas, los pacientes a veces se conectan a las redes sociales, donde hacen preguntas y describen sus niveles de hormona tiroidea, regímenes farmacológicos y síntomas. Algunas plataformas en línea ofrecen información que es, en el mejor de los casos, dudosa, pero en general, los medios de comunicación sociales han aumentado la comprensión de los pacientes sobre los síntomas difíciles de resolver, dijo Bianco.

También se animan unos a otros.

VanOrden, que ha estado activa en Reddit, tiene este consejo para otros pacientes: “No se rindan. Sigan siendo sus propios defensores. En algún lugar hay un médico que te escuchará”. Ha comenzado un tratamiento alternativo (medicación disecada para la tiroides, una opción no aprobada por la FDA) más una dosis baja de naltrexona, una droga para la adicción, aunque los datos son limitados. Ahora se está sintiendo mejor.

La investigación de la enfermedad tiroidea autoinmune recibe poca financiación, por lo que las causas subyacentes de la disfunción inmune no están bien estudiadas, afirmó Henderson. El establishment médico no ha reconocido plenamente a los pacientes con hipotiroidismo difíciles de tratar, pero un mayor reconocimiento de ellos y de sus síntomas ayudaría a financiar la investigación, afirmó Bianco.

“Me gustaría un reconocimiento muy claro y sólido de que estos pacientes existen”, dijo. “Estas personas son reales”.

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For her and millions of other Americans, that’s the most common cause of hypothyroidism, a condition in which the thyroid, a butterfly-shaped gland in the neck, doesn’t produce enough of the hormones needed for the body to regulate metabolism.

There’s no cure for Hashimoto’s or hypothyroidism. But VanOrden, who lives in Athens, Texas, started taking levothyroxine, a much-prescribed synthetic thyroid hormone used to treat common symptoms, like fatigue, weight gain, hair loss, and sensitivity to cold.

Most patients do well on levothyroxine and their symptoms resolve. Yet for others, like VanOrden, the drug is not as effective.

For her, that meant floating from doctor to doctor, test to test, and treatment to treatment, spending about $5,000 a year.

“I look and act like a pretty energetic person,” said VanOrden, 38, explaining that her symptoms are not visible. “But there is a hole in my gas tank,” she said. And “stress makes the hole bigger.”

Autoimmune diseases occur when the immune system mistakenly attacks and damages healthy cells and tissues. Other common examples include rheumatoid arthritis, lupus, celiac disease, and inflammatory bowel disease. There are more than , affecting up to an estimated , disproportionately women. Overall, the cost of treating autoimmune diseases is estimated at more than in the U.S.

Despite their frequency, finding help for many autoimmune diseases can prove frustrating and expensive. Getting diagnosed can be a major hurdle because the range of symptoms looks a lot like those of other medical conditions, and there are often no definitive identifying tests, said Sam Lim, clinical director of the Division of Rheumatology at Emory University School of Medicine in Atlanta. In addition, some patients feel they have to fight to be believed, even by a clinician. And after a diagnosis, many autoimmune patients rack up big bills as they explore treatment options.

“They’re often upset. Patients feel dismissed,” Elizabeth McAninch, an endocrinologist and thyroid expert at Stanford University, said of some patients who come to her for help.

Insufficient medical education and lack of investment in new research are two factors that hinder overall understanding of hypothyroidism, according to Antonio Bianco, a University of Chicago endocrinologist and leading expert on the condition.

Some patients become angry when their symptoms don’t respond to standard treatments, either levothyroxine or that drug in combination with another hormone, said Douglas Ross, an endocrinologist at Massachusetts General Hospital in Boston. “We will have to remain open to the possibility that we’re missing something here,” he said.

Jennifer Ryan, 42, said she has spent “thousands of dollars out-of-pocket” looking for answers. Doctors did not recommend thyroid hormone medication for the Huntsville, Alabama, resident — diagnosed with Hashimoto’s after years of fatigue and weight gain — because her levels appeared normal. She recently switched doctors and hopes for the best.

“You don’t walk around hurting all day long and have nothing wrong,” Ryan said.

And health insurers typically deny coverage of novel hypothyroidism treatments, said Brittany Henderson, an endocrinologist and founder of the Charleston Thyroid Center in South Carolina, which sees patients from all 50 states. “Insurance companies want you to use the generics even though many patients don’t do well with these treatments,” she said.

Meanwhile, the extent of Americans’ thyroid problems can be seen in drug sales. Levothyroxine is among the medications in the U.S. every year. Yet of the drug for those with mild hypothyroidism.

, paid for by AbbVie — maker of Synthroid, a brand-name version of levothyroxine — said a medical and pharmacy claims database showed that the prevalence of hypothyroidism, including milder forms, rose from 9.5% of Americans in 2012 to 11.7% in 2019.

The number of people diagnosed will rise as the population ages, said McAninch. Endocrine disruptors — natural or synthetic chemicals that can affect hormones — could account for some of that increase, she said.

In their search for answers, patients sometimes connect on social media, where they ask questions and describe their thyroid hormone levels, drug regimens, and symptoms. Some online platforms offer information that’s dubious at best, but overall, social media outlets have increased patients’ understanding of hard-to-resolve symptoms, Bianco said.

They also offer one another encouragement.

VanOrden, who has been active on Reddit, has this advice for other patients: “Don’t give up. Continue to advocate for yourself. Somewhere out there is a doctor who will listen to you.” She has started an alternative treatment — desiccated thyroid medication, an option not approved by the FDA — plus a low dose of the addiction drug naltrexone, though the data is limited. She’s feeling better now.

Research of autoimmune thyroid disease gets little funding, so the underlying causes of immune dysfunction are not well studied, Henderson said. The medical establishment hasn’t fully recognized hard-to-treat hypothyroid patients, but increased acknowledgment of them and their symptoms would help fund research, Bianco said.

“I would like a very clear, solid acknowledgment that these patients exist,” he said. “These people are real.”

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And since he wasn’t accepting new patients, she would have to find a new primary care physician.

“I think it’s unconscionable,” Siegel said, noting that many patients may have stayed away from the doctor’s office the past few years because of the covid pandemic. “There was no notification to patients that they’re on the verge of losing their doctor.”

Though it is dismaying to learn you’ve been dropped from a physician’s practice because a few years have passed since your last visit, the approach isn’t uncommon. Exactly how widespread the experience is, no one can say. But specialists also do this.

The argument for dropping the occasional patient makes some sense. Since many primary care doctors have a waiting list of prospective patients, removing those they rarely see opens up patient slots and improves access for others.

“Most primary care practices are incredibly busy, in part due to pent-up demand due to covid,” said , director of Harvard Medical School’s Center for Primary Care and a general internist at Beth Israel Deaconess Medical Center.

“Even though continuity of care is important, if the patient hasn’t been in and we don’t know if they’re going to come in, it’s hard to leave space for them,” he said.

Patients often move away or find a different doctor when their insurance changes without notifying the practice, experts say. In addition, physicians may seek to classify people they haven’t seen in a long time as new patients since their medical, family, and social history may require a time-consuming update after a lengthy break. Patient status is one element that determines how much doctors get paid.

Still, the transition can be trying for patients.

“I can completely understand the patient’s perspective,” said Courtney Jones, a senior director of case management at the Patient Advocate Foundation. “You believe you have a medical team that you’ve trusted previously to help you make decisions, and now you have to find another trusted team.”

Siegel said she rarely went to the doctor, adhering to her physician father’s counsel that people shouldn’t go unless they’re sick. Although she hadn’t been to her doctor’s office in person recently, Siegel said she had corresponded with the practice staff, including keeping them up to date on her covid vaccination status.

After receiving the online dismissal through the patient portal for the Jefferson Health system, Siegel called the family medicine practice’s patient line directly. They told her three years was the protocol and they had to follow it.

“I asked, ‘What about the patient?’” Siegel said. “They didn’t have an answer for that.”

It was a month before Siegel, who has coverage under Medicare’s traditional fee-for-service program, could see a doctor who was accepting new patients. By that time, her stomach virus symptoms had resolved.

Jefferson Health doesn’t have a policy that patients lose their doctor if they’re not seen regularly, according to a statement from spokesperson Damien Woods.

However, he said, “Patients not seen by their provider for three years or more are classified in the electronic medical records as new patients (rather than established patients), per Center for Medicare and Medicaid Services (CMS) guidance. Whenever possible, Jefferson works with these patients to keep them with their primary care provider and offers options for new providers in certain circumstances.”

American Medical Association recommend that physicians notify patients in advance when they’re withdrawing from a case so they have time to find another physician.

But the organization, which represents physicians, has no guidance about maintaining a panel of patients, said AMA spokesperson Robert Mills.

The American Academy of Family Physicians, which represents and advocates for family physicians, declined to comment for this story.

A primary care physician’s panel of patients typically includes those who have been seen in the past two years, said Phillips, of Harvard. Doctors may have 2,000 or more patients, studies show. Maintaining a workable number of patients is crucial, both for effective patient care and for the doctors.

“Practices realize that a major contributor to physician burnout is having more patients than you can deal with,” Phillips said.

Demand for physician services is expected to continue to outstrip supply in the coming decades, as people age and need more care at the same time the number of retiring physicians is on the upswing. According to , by 2034 there will be a shortage of up to 48,000 primary care physicians.

Maintaining a regular relationship with a primary care provider can help people manage chronic conditions and promptly identify new issues. Regularly checking in also helps ensure people receive important routine services such as immunizations and blood pressure checks, said , a former primary care physician who is president of the Commonwealth Fund, a research and policy organization.

Health care organizations increasingly focus on requiring doctors to meet certain quality metrics, such as managing patients’ high blood pressure or providing comprehensive diabetes care. In this environment, “it could be problematic for physicians to be accountable for the health of patients who do not see them,” Blumenthal said.

Money also figures into it. Steady visits are good for a practice’s bottom line. Practices may also decide to avoid new Medicare patients or those with certain types of insurance because the payments are too low, said , a consultant with Medical Group Management Association, an organization for health care managers.

In general, doctors aren’t obligated to continue seeing a patient. A doctor might dismiss patients because they aren’t following clinical recommendations or routinely cancel or miss appointments. Belligerent or abusive behavior is also grounds for dropping a patient.

In certain instances, physicians may be legally liable for “patient abandonment,” a form of medical malpractice. State rules vary, but there are common elements. Those rules generally apply when a doctor harms a patient by dropping them abruptly at a critical stage of treatment. It would generally not apply if a patient has not seen the physician for several years.

Even though quietly dropping a seldom-seen patient might not have an immediate medical consequence, patients ought to be informed, experts said.

“It’s really good customer service to explain the situation,” said , senior vice president at the Healthcare Financial Management Association, an organization for finance professionals. As for Siegel, he said, “This woman should not be left hanging. If you’re the patient, the physician should be proactive.”

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Los nuevos requisitos de personal del estado para estos hogares, aprobados discretamente en el proyecto de ley de presupuesto del año pasado, no parecen gozar de gran popularidad. Los defensores de los pacientes dicen que las nuevas regulaciones no son lo suficientemente estrictas, y que los centros con poco personal ponen en riesgo a sus residentes. Por su parte, los operadores de las residencias argumentan que no pueden contratar más personal para cumplir con la normativa.

Bajo las nuevas reglas, que entraron en vigencia en julio, pero que aún no se han hecho cumplir, los centros especializados deben proporcionar al menos 3,5 horas de atención directa por residente al día, en comparación con las 3,2 horas de atención que se viene proporcionando. Ese cuidado puede incluir desde insertar una sonda de alimentación hasta cambiar un pañal para adultos o ayudar a los residentes a comer y bañarse.

Se espera que el Departamento de Salud Pública de California, que supervisa las residencias para adultos mayores, anuncie a finales de enero qué centros —si los hay— estarán exentos de las nuevas regulaciones. Pero a algunos defensores de los pacientes no les gusta que las instalaciones opongan resistencia.

“Estamos consternados por el sistema de exención. Se está enviando el peor mensaje posible a los hogares para adultos mayores de California al aceptar un número de personal que podría poner en peligro a los residentes”, dijo Mike Connors de California Advocates for Nursing Home Reform, un grupo de defensa del consumidor.

(Haz clic y para ver qué residencias de California han solicitado exenciones por escasez de personal).

Los investigadores han vinculado la cantidad de personal a una mejor atención, y algunos expertos recomiendan de 3,8 a 4,1 horas de atención al día por paciente, como mínimo, para poder ofrecer atención de calidad. Tener suficiente personal ayuda a prevenir las caídas, las llagas por presión y otros problemas que pueden llevar a los residentes más frágiles al hospital.

reciente de Kaiser Health News (KHN) encontró que durante años hogares en todo el país inflaron el número de trabajadores ante el gobierno federal. Ahora, las residencias están obligadas a reportar los registros de nómina reales si quieren seguir siendo elegibles para los pagos de Medicare y Medicaid.

Durante el primer trimestre de 2018, el 58% de los centros especializados de California promediaron al menos 3,5 horas de atención al paciente al día, según un análisis de KHN de los registros de nómina presentados al gobierno federal. Llegando al 76% si se incluían las residencias en las que también se contaba a los administradores.

California es uno de los pocos estados que establecen requisitos mínimos para el personal de los hogares de adultos mayores. La mayoría de los estados cumplen con las normas del gobierno federal que exigen que los centros especializados que reciben dinero de Medicare o Medicaid tengan suficiente personal para satisfacer las necesidades de los residentes, según expresó Robyn Grant, directora de políticas públicas y abogacía del National Consumer Voice for Quality Long-Term Care.

Illinois requiere que las residencias para personas mayores proporcionen un mínimo de 3,8 horas de atención por paciente al día y el Distrito de Columbia requiere 4,1 horas, explicó Grant. Maine y Oklahoma adoptan un enfoque diferente, estableciendo una proporción entre el personal y los pacientes, en lugar de horas de atención.

Los gerentes de las residencias, y sus lobistas, dicen que es difícil contratar enfermeras y asistentes calificados en la robusta economía de California, y lamentan lo que describen como un reembolso inadecuado de Medicare y Medicaid. También han criticado una disposición de los nuevos requisitos según la cual 2,4 de las 3,5 horas de atención al paciente debe ser proporcionada por un asistente de enfermería certificado, en lugar de cualquier otro profesional de enfermería.

Las residencias necesitan flexibilidad porque “no todos los pacientes son iguales, no todos los diagnósticos son iguales”, señaló Matt Robinson, director de asuntos legislativos de la California Association of Health Facilities. “No nos oponemos a que haya más personal. Pero queremos personal de calidad. Queremos asegurarnos que haya una fuerza laboral sostenible para cumplir con ese mandato, de lo contrario es sólo un mandato vacío”.

Robinson dijo que los centros están solicitando exenciones sobre una base de “buena fe”. Si no se conceden las solicitudes de exención, añadió, las residencias podrían reducir sus camas o incluso cerrar.

En Los Ángeles, el Kei-Ai Los Angeles Healthcare Center, con 300 camas, ha solicitado una exención alegando “escasez de mano de obra”. Para Cynthia Sakaki Sirlin eso “está muy mal”. El padre de Sakaki Sirlin vive ahí, tiene 86 años y es veterano de la Guerra de Corea.

“No sé por qué están haciendo esto. Necesitan más personal, no menos, para mejorar la atención al paciente, según muestra la investigación. Entonces, ¿por qué piden una exención? ¿Por qué el estado lo permite? Eso sólo recompensa a los propietarios que no están dispuestos a contratar más personal”, dijo Sakaki Sirlin.

Sakaki Sirlin, enfermera y representante del consejo familiar de Kei-Ai, contó que desde que un promotor inmobiliario adquirió la antigua residencia sin fines de lucro en 2016, ha notado una mayor rotación de personal. Le preocupa que su padre, que debe usar una silla de ruedas y que no puede alimentarse por sí mismo, no reciba la atención que necesita. Los representantes de Kei-Ai no respondieron a una solicitud para comentar al respecto.

Hay casi 100,000 asistentes de enfermería certificados en California (CNAs), de acuerdo con datos laborales federales. Los defensores de los pacientes dicen que muchos CNAs optan por no trabajar en estas residencias porque el pago es bajo y el trabajo, duro.

“Si les pagaran mejor, tendrían mucho más personal”, incluso en lugares remotos de California, explicó Suzi Fregeau, gerente del programa de atención a largo plazo en los condados de Humboldt y Del Norte. El salario promedio por hora para los asistentes de enfermería certificados en California fue de $16.13 por hora en 2017, de acuerdo con los .

Algunas de las residencias de California que buscan exenciones han recibido repetidas llamadas de atención del Departamento de Salud Pública del estado por personal inadecuado que produjo daños a los pacientes. Entre ellas se encuentran las propiedades de Shlomo Rechnitz, que supuestamente controla 1 de cada 14 camas de residencias de personas mayores en California. Rechnitz ha enfrentado numerosos cuestionamientos federales y estatales por falta de personal y problemas de calidad en sus centros.

El CEO de una de las empresas que gestiona las residencias de Rechnitz dijo, en una declaración escrita, que varias de ellas presentaron solicitudes de exención de “necesidades de los pacientes” por su cuenta con datos proporcionados por la empresa. “Todas estas residencias priorizan las necesidades de sus pacientes por encima de todo y tienen un historial intachable de cumplimiento con los requisitos de personal”, aseguró David Silver, CEO de Rockport Administrative Services LLC.

“Lo que estamos viendo es que los centros que ya no cuentan con suficiente personal —las residencias por las que sí recibimos quejas— son las que piden exenciones”, dijo Joe Rodrigues, el ombudsman de atención a largo plazo del estado. “No apoyamos esas peticiones”.

Esta historia fue producida por Kaiser Health News, que publica , un servicio de la .

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The state’s new staffing requirements for nursing homes, quietly passed in last year’s budget bill, seem universally unpopular. Patient advocates say the new regulations don’t go far enough and that residents remain at risk in poorly staffed homes. Nursing home operators say they can’t hire enough staff to comply.

Under the new rules, which took effect in July but haven’t yet been enforced, skilled nursing facilities must provide at least 3.5 hours of direct care per resident per day, up from 3.2 hours of care previously. That care can range from inserting a feeding tube to changing an adult diaper or helping residents with eating and bathing.

The California Department of Public Health, which oversees nursing homes, is expected to announce in late January which — if any — facilities it will exempt from the new regulations. But some patient advocates don’t like the nursing homes’ balking.

“We’re appalled by the waiver system. It’s sending the worst possible message to California nursing homes that it’s OK to staff at levels that endanger residents,” said Mike Connors of California Advocates for Nursing Home Reform, a consumer advocacy group.

(Check to see which California nursing homes have applied for workforce shortage waivers and .)

Researchers have strongly linked more nursing staff with better care, with some experts recommending from 3.8 to 4.1 hours of care per patient per day as a bare minimum for quality nursing home care. Having enough staff helps prevent falls, pressure sores and other problems that can land fragile seniors in the hospital.

A recent Kaiser Health News found that for years nursing homes nationwide overstated staffing to the federal government. Now, nursing homes are required to report actual payroll records to remain eligible for Medicare and Medicaid payments.

During the first three months of 2018, 58 percent of California’s skilled nursing facilities averaged at least 3.5 hours of patient care a day, according to a Kaiser Health News analysis of payroll records submitted to the federal government. That rose to 76 percent when including nursing homes where administrators also were counted.

California is one of only a few states that set their own minimum requirements for nursing home staffing. Most states abide by federal government standards requiring skilled nursing facilities that receive money from Medicare or Medicaid to have enough staff to meet residents’ needs, said Robyn Grant, director of public policy and advocacy for National Consumer Voice for Quality Long-Term Care, an advocacy group.

Illinois requires nursing homes to provide a minimum of 3.8 hours of care per patient a day and the District of Columbia requires 4.1 hours, Grant said. Maine and Oklahoma take a different approach, establishing staff-to-patient ratios, rather than hours of care, for nursing homes.

Nursing home officials and their lobbyists say it’s tough to find qualified nurses and assistants in California’s robust economy, and they bemoan what they describe as inadequate reimbursement from Medicare and Medicaid. They also have criticized a provision of the new requirements that 2.4 of the 3.5 hours of patient care must be provided by a certified nursing assistant, rather than another nursing professional.

Nursing homes need flexibility because “not every patient is the same, not every diagnosis is the same,” said Matt Robinson, legislative affairs director for the California Association of Health Facilities, an industry group. “We’re not opposed to more staff. But we want quality staff. We want to make sure there’s a sustainable workforce to meet that mandate, otherwise it’s just an empty mandate.”

Robinson said facilities are applying for waivers on a “good-faith basis.” If waiver requests aren’t granted, he said, nursing homes may reduce their beds or even shut down.

In Los Angeles, the 300-bed Kei-Ai Los Angeles Healthcare Center has applied for an exemption citing a “workforce shortage.” But Cynthia Sakaki Sirlin, whose 86-year-old father, a veteran of the Korean War, lives there says, “I think it’s wrong.”

“I don’t know why they’re doing this. They need more nursing staff to improve patient care, not less, the research shows that. So why are they asking for a waiver? Why is the state allowing them? That just rewards owners who are not willing to staff the homes,” Sakaki Sirlin said.

Sakaki Sirlin, a nurse practitioner and a representative of Kei-Ai’s family council, said that since the formerly nonprofit nursing home was purchased by a real estate developer in 2016, she has noticed more staff turnover. She worries that her father, a wheelchair user who can’t feed himself, won’t get the care he needs. Representatives from Kei-Ai did not respond to a request for comment.

There are nearly 100,000 certified nursing assistants in California, according to federal labor data. Patient advocates say many CNAs choose not to work for nursing homes because of the comparatively low pay and tough workload.

“If they paid them better, they’d have plenty of staff,” even in remote parts of California, said Suzi Fregeau, long-term care program manager in Humboldt and Del Norte counties. The mean hourly wage for certified nursing assistants in California was $16.13 in 2017, according to .

Some of the California homes seeking exemptions have been repeatedly cited by the state’s Department of Public Health for inadequate staffing that led to patient harm. Among them are homes owned by Shlomo Rechnitz, who reportedly controls 1 in 14 nursing home beds in California. He has faced numerous federal and state probes of understaffing and quality problems at his homes.

The CEO of one of Rechnitz’s nursing home management companies said in a written statement that several homes submitted “patient needs” waiver requests on their own with data provided by the company. “All of these facilities prioritize the needs of their patients above all else and these facilities have a stellar history of complying with applicable staffing requirements,” said David Silver, CEO of Rockport Administrative Services LLC.

“What we’re seeing is that the facilities that already are understaffed — the facilities for which we do get complaints — are the ones asking for waivers,” said Joe Rodrigues, the state’s long-term care ombudsman. “We’re not supportive of those requests.”

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The twice-a-day medicine was a breakthrough because it supplanted an existing drug with debilitating side effects that had to be taken every six hours around the clock — a missed dose could permanently damage a child’s kidneys.

But the families’ elation dimmed when Raptor Pharmaceutical, which acquired the marketing rights and financed clinical trials for Procysbi, priced the drug: more than $300,000 a year for some patients.

“When I heard the number, I was like, holy … that’s incredible!” said Kevin Partington, the father of twins with cystinosis. “The first thought was, how do we pay for it and get this approved through insurance?”

Manufacturers selling precious, lifesaving medicines and patients share an uneasy alliance. They need each other but have clashing priorities, especially when it comes to drugs treating rare diseases such as cystinosis.

Cystinosis families say they are deeply grateful for Procysbi. The medicine would not be on the market without Raptor — which performed clinical trials — and Horizon Pharma, which acquired the rights two years ago, they acknowledge.

But they also feel they’ve been used. What began as a desperate search for life-extending medicine, cystinosis parents say, has become a story of corporations profiteering off their children.

What’s more, they say, even as they have tried to keep the companies at arm’s length, Horizon and Raptor have breached sensitive boundaries around their tightknit community to increase sales.

“I feel like it’s all about the bottom line,” said Denice Flerchinger, who has a daughter with cystinosis and helped raised some of the first research funds for the drug. “I don’t think any of us thought they could do this and get away with it.”

Procysbi is an “orphan drug,” under FDA rules, giving its makers an extended monopoly and the opportunity for big profits because it treats a disease affecting only about 500 Americans.

Its price has risen five times in the past five years, first by Raptor and later through its current owner, Horizon. For some patients, it now costs more than $1 million annually.

Procysbi’s high cost is needed to finance research for a range of medicines, said Matt Flesch, a spokesman for Horizon, which bought Raptor in late 2016 and has raised the drug’s list price 21 percent since then, according to data from Connecture, an information-technology firm. “If rare diseases don’t have a higher price, then companies aren’t going to bring new medicines forward,” he said.

After rebates and discounts, Horizon’s average annual revenue per patient is about $486,000, Flesch said. No patient needing Procysbi lacks it, due to a Horizon program that covers deductibles and copays or the entire cost if insurance is lacking, he added.

Cystinosis is an inherited disease that keeps children from processing a protein component called cystine, slowly weakening organs and tissues. Without treatment, patients can go blind and develop end-stage renal disease by their 10th birthday.

Treatment can be brutal. Before Procysbi, the only medicine prolonging life — typically into a patient’s 20s, when a kidney transplant is often needed — tasted like sulfur, induced vomiting, made an embarrassing odor and had to be taken every six hours. Even one missed 3 a.m. dose meant rising cystine levels for a baby or toddler and the possibility of permanent organ damage.

In 2003, Nancy Stack’s daughter Natalie made a wish on a napkin in purple crayon on her 12th birthday: “To make my disease go away forever.”

Years ago, “you were handed a pamphlet, ‘When Your Child Has a Terminal Illness,’ dot, dot, dot,” Stack said. “It never sat well with us. But when our own daughter at 12 understood that if there was no cure it meant she was doing to die, then we realized, wow, this is a big life event for us.”

Through friends, relatives and her husband’s contacts in real estate, the Stacks raised money for research and founded a nonprofit now called the Cystinosis Research Foundation. At the same time, they began building a network of families who were coping with a frightful illness.

“It was so encouraging to see this community doing all these amazing things,” said Nicole Manz, who learned about CRF in a hospital room where doctors diagnosed her son with cystinosis. “It was really empowering for our family.”

CRF’s ultimate goal was a cure, but one early, promising idea was to make the existing treatment easier to take. Scientists at the University of California-San Diego, a longtime center of cystinosis research, theorized that a time-release coating on the medicine could reduce side effects and the need for frequent doses.

With multiple CRF grants totaling $1.6 million funding early investigation, researchers found their hypothesis was correct. Now the foundation needed a corporate partner to finance more trials required for FDA approval.

Raptor, a company with about a dozen employees and no revenue, had acquired the rights to what would become Procysbi in 2007 and agreed to finance clinical trials to get it to market.

Raptor executives were quick to acknowledge how much they relied on the foundation and UC-San Diego, not only for the preliminary research but also for the patients’ contact information, which would save millions in marketing costs.

“We’re kind of coming in at the eleventh hour” in developing Procysbi, Raptor’s then-CEO, Christopher Starr, told the San Francisco Business Times in 2012, shortly before the drug’s launch.

Raptor’s research-and-development expense leading to Procysbi’s launch in June 2013 came to about $80 million, financial statements show. Most of that went toward getting Procysbi approved for cystinosis, although some was spent on investigating other drugs or diseases.

As Procysbi moved closer to approval, families knew it would be expensive. That’s not unusual for a rare-disease drug with a small market. Cystagon, the older, non-coated version of the medicine, cost about $9,000 a year at the time.

But nothing prepared those families for what happened. The $300,000 launch price was so high that Raptor CEO Starr objected, said Dr. Patrice Rioux, who was the company’s chief medical officer and also opposed the pricing.

Starr “was a scientist who wanted to provide the drug at a relatively good price,” Rioux said in an interview. “The board was not of this opinion” and overruled him, he said.

Starr did not respond to requests for an interview. In 2014, a year after Procysbi’s approval, the company announced his resignation as CEO, although he stayed on Raptor’s board. Rioux said he resigned at the same time.

“At the time of Procysbi’s approval, there was full agreement about its price within management,” Julie Anne Smith, who replaced Starr as CEO, said in an email. “I am proud to have been part of the Raptor team who worked with the cystinosis community” to get the drug approved, she said.

Stack wrote an angry note shortly after learning about the price.

“I feel awful about all this and personally accountable,” she said in an email to Raptor executives. “It is so disheartening — the community will suffer from the high cost of Procysbi.”

The drug quickly brought in far more money than Raptor spent developing it, a result of its acceptance by patients and insurers, plus the steep price increases.

Procysbi’s list price is 48 percent higher than it was in 2013. The drug has generated revenue totaling about $500 million through June 2018 for both Raptor and Horizon, financial statements show. At the time of the Horizon buyout, Raptor revenue of $1.5 billion from 2019 through 2026.

That cash stream prompted Horizon to buy Raptor for $800 million in 2016, enriching Starr, who still held shares and options worth $8.7 million, and Smith, his replacement CEO, who got $7.5 million in merger-related payments, according to an analysis of regulatory filings by Andy Restaino, CEO of Technical Compensation Advisors, an executive pay consultancy.

UC-San Diego, which licensed the rights to market Procysbi, has collected $20.9 million in royalties from Horizon and Raptor through 2017, according to data obtained under public information laws. The university “does not play a role in setting the market price for any drug,” UCSD said via a spokesman. Royalties help finance new research, it said.

Meanwhile the nonprofit organization started by families to develop the medicine, which held no business rights, got nothing.

“Those early grants were just [a] letter form and a handshake with the doctors,” Stack said. “We really didn’t understand how complex the system is, and how universities typically own all the … rights. We didn’t see dollar signs in our children.”

Procysbi proceeds have financed new research including continuing cystinosis studies as well as potential drugs for thyroid eye disease, rheumatological illness and other ailments, said Horizon spokesman Flesch. Last year, Horizon spent $225 million on research and development, according to financial statements.

“We’re really transforming ourselves into a research-focused company, and a primary focus there is orphan diseases,” Flesch said.

About three-fourths of U.S. cystinosis patients now take Procysbi, but Horizon continues to work aggressively to .

The company’s strategy for growth relies on building tight relationships with patients and advocacy groups, a typical marketing approach for companies selling orphan drugs, said Annabel Samimy, who follows pharma stocks for Stifel Financial.

But for some, the personal touch is intrusive. CRF had to ban Raptor reps from emotional family meetings that broached topics such as bed-wetting, Stack said. Tearful Raptor employees once joined patients in making posters about cystinosis hopes and fears, which seemed inappropriate, she said.

Horizon pays for travel and lodging to where intense discussions among patients and families can feel like therapy. The company makes audio recordings of the sessions to use in market research, which some patients find deceptive, said Shannon Keizer, a cystinosis patient in her 20s.

The company also hosts where families speak to physicians and one of eight Horizon “ambassadors” — patients and their family members who receive a $500 honorarium for each event

“Instead of wining and dining and paying out stipends, they need to lower the price of the drug,” said Flerchinger. “It’s our money they’re spending.”

Horizon’s Facebook page, branded as “Cystinosis United,” resembles the private Facebook groups where cystinosis families console and encourage one another, with pictures of smiling patients and slick, touching videos. The company added its logo to the banner photo after parents complained, according to a company email.

In conversations with Horizon, Stack refers to this hands-on approach as “overreach.” But she said she lies awake at night worried that her outspoken criticism could cut off access to her daughter’s life-sustaining drug. Horizon could stop manufacturing it.

“I think that’s the risk of doing all this. Speaking out so forcefully about the drug price,” Stack said. “What if they say this is really a pain so we’re just going to drop this drug? I would never forgive myself, because we discovered” it.

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Even as these patient groups grow in number and political influence, their funding and their relationships to drugmakers are little understood. Unlike payments to doctors and lobbying expenses, companies do not have to report payments to the groups.

The database, called “Pre$cription for Power,” shows that donations to patient advocacy groups tallied for 2015 — the most recent full year in which documents required by the Internal Revenue Service were available — dwarfed the total amount the companies spent on federal lobbying. The 14 companies that contributed $116 million to patient advocacy groups reported only about $63 million in that same year.

Though their primary missions are to focus attention on the needs of patients with a particular disease — such as arthritis, heart disease or various cancers — some groups effectively supplement the work lobbyists perform, providing patients to testify on Capitol Hill and organizing letter-writing and social media campaigns that are beneficial to pharmaceutical companies.

Six drugmakers, the data show, contributed a million dollars or more to individual groups that represent patients who rely on their drugs. The database identifies over 1,200 patient groups. Of those, 594 accepted money from the drugmakers in the database.

The financial ties are troubling if they cause even one patient group to act in a way that’s “not fully representing the interest of its constituents,” said Matthew McCoy, a medical ethics professor at the University of Pennsylvania who co-authored a 2017 study about patient advocacy groups’ influence and transparency.

Notably, such groups have been silent or slow to complain about high or escalating prices, a prime concern of patients.

“When so many patient organizations are being influenced in this way, it can shift our whole approach to health policy, taking away from the interests of patients and towards the interests of industry,” McCoy said. “That’s not just a problem for the patients and caregivers that particular patient organizations serve; that’s a problem for everyone.”

Bristol-Myers Squibb provides a stark example of how patient groups are valued. In 2015, it spent more than $20.5 million on patient groups, compared with on federal lobbying and less than on major trade associations, according to public records and company disclosures. The company said its decisions regarding lobbying and contributions to patient groups are “unrelated.”

“Bristol-Myers Squibb is focused on supporting a health care environment that rewards innovation and ensures access to medicines for patients,” said spokeswoman Laura Hortas. “The company supports patient organizations with this shared objective.”

The first-of-its-kind database, compiled by Kaiser Health News, tallies the money from Big Pharma to patient groups. KHN examined the 20 pharmaceutical firms included in the S&P 500, 14 of which were transparent — in varying degrees — about giving money to patient groups. Pre$cription for Power is based on information contained in charitable giving reports from company websites and federal 990 regulatory filings.

It spotlights donations pharma companies made to patient groups large and small. The recipients include well-known disease groups, like the American Diabetes Association, with revenues of hundreds of millions of dollars; high-profile foundations like Susan G. Komen, a patient group focused on breast cancer; and smaller, lesser-known groups, like the Caring Ambassadors Program, which focuses on lung cancer and hepatitis C.

The data show that 15 patient groups — with annual revenues as large as $3.6 million — relied on the pharmaceutical companies for at least 20 percent of their revenue, and some relied on them for more than half of their revenue. The database explores only a slice of the pharmaceutical industry’s giving overall and will be expanded with more companies and groups over time.

“It’s clear that more transparency in this space is vitally important,” said Sen. Claire McCaskill (D-Mo.), who has been investigating the links between patient advocates and opioid manufacturers and is considering legislation to track funding. “This database is one step forward in that effort, but we also need Congress to act.”

What Drives The Money Flow

The financial ties between drugmakers and the organizations that represent those who use or prescribe their blockbuster medicines have been of growing concern as drug prices escalate. The Senate investigated conflicts of interest in the run-up to the passage of the 2010 Physician Payments Sunshine Act — a law that required payments to physicians from makers of drugs and devices to be registered on a public website — but patient groups were not addressed in the bill.

Some of the patient groups with ties to trade groups echo industry talking points in media campaigns and letters to federal agencies, and do little else. And patients, supported by pharma, are dispatched to state capitals and Washington to support research funding. Some groups send patients updates on the newest drugs and industry products.

“It’s through groups like this that patients often learn about illnesses and treatments,” said Rick Claypool, a research director for Public Citizen, a consumer advocacy group that says it pharmaceutical funding.

For the patient group Caring Ambassadors Program, industry funds are needed to make up for a lack of public funding, said the group’s executive director, Lorren Sandt. According to IRS filings and published company reports, in 2015 the group received $413,000, the bulk of which came from one company, AbbVie, which makes a hepatitis C treatment and has been testing a new lung cancer drug, Rova-T, not yet approved. She said the money had no influence on the Caring Ambassadors Program’s priorities.

“There aren’t a lot of large pockets of funding outside of the pharmaceutical money,” Sandt said. “We take it where we can find it.”

Other patient groups such as The National Women’s Health Network, based in Washington, D.C., make sacrifices to avoid pharmaceutical funding. That includes operating with a small staff in a “modest” office building with few windows and outdated computers, according to executive director Cindy Pearson. “You can see the effect of our approach to funding as soon as you walk [in] the door.”

Pearson said it’s hard for patient groups not to be influenced by the funder, even if they proclaim independence. Patient groups “build relationships with their funders and feel in sync and have sympathy” for them. “It’s human nature. It’s not evil or weak, but it’s wrong.”

Charity As Marketing

Patients newly diagnosed with a disease often turn to patient advocacy groups for advice, but the money flow to such groups may distort patients’ knowledge and public debate over treatment options, said Dr. Adriane Fugh-Berman, the director of PharmedOut, a Georgetown University Medical Center program that is critical of some pharmaceutical marketing practices.

“[The money flow limits] their advocacy agenda to competing branded products when the best therapy might be generics, over-the-counter drugs or diet and exercise,” she said.

AbbVie — whose specialty drug Humira made up of the company’s net revenue in 2017 and is used to treat patients with autoimmune diseases, including Crohn’s disease and certain kinds of arthritis — gave $2.7 million to the Crohn’s & Colitis Foundation and $1.6 million to the Arthritis Foundation, according to the company’s public disclosures included in the database. The list price for a month’s supply of Humira, a biologic drug, is $4,872, according to Express Scripts, a pharmacy benefits manager.

Even though Humira will face competition from near-copycat drugs called biosimilars, it is expected to remain the highest-grossing drug in the United States through 2022, according to drug industry analysts at .

The Arthritis and Crohn’s foundations have been largely silent on the cost of Humira and vocal on safety concerns about biosimilars. The Arthritis Foundation has championed state laws that could add extra steps for consumers to receive biosimilars at the pharmacy counter, potentially keeping more patients on the brand-name drug. Experts say those laws could help protect Humira’s market share from generic competitors.

A coalition of patient groups, Patients for Biologics Safety & Access, opposes the automatic substitution of a cheaper biosimilar when doctors prescribe a biologic. In 2015, members of that coalition, including the Crohn’s & Colitis Foundation, the Arthritis Foundation and the Lupus Foundation of America, accepted about $9.1 million from pharmaceutical companies in the database, according to public disclosures. They include AbbVie and Johnson & Johnson, makers of blockbuster biologics.

The Arthritis Foundation did not deny receiving the money but said the foundation represents patients, not sponsors. It is “optimistic” about biosimilars’ ability to help patients and save them money, said Anna Hyde, vice president of advocacy and access. “The Foundation supports the Food and Drug Administration’s scientific standards in evaluating the safety and efficacy of biosimilars, and we support policies that encourage innovation and foster a competitive marketplace.”

(Story continues below.)

The Crohn’s & Colitis Foundation maintains “more than an arm’s-length distance” from its donors in the pharmaceutical industry, who have no say over the foundation’s strategic objectives, said president and CEO Michael Osso.

He added that the foundation’s position on biosimilars is “evolving.”

Lupus Foundation CEO Sandra Raymond said she could not explain how her group, also based in Washington, was involved in the coalition. She confirmed the Lupus Foundation received $444,000 from Pfizer in 2015 but said the money was not linked to any relationship with Patients for Biologics Safety & Access.

“I never went to a meeting,” Raymond said. “A former employee signed us up for a whole host of coalitions. I think we put our name on something or someone did.”

She said the Lupus Foundation was no longer a member of the coalition. Days after Kaiser Health News reached out to the coalition, its website was updated, excluding the Lupus Foundation.

For its part, AbbVie — which overall donated $24.7 million to patient groups in 2015, according to the new database — stipulates that its grants to nonprofits are “non-promotional” and provide no direct benefit to its business, according to a company statement. The company gives to patient groups because they serve as an “important, unbiased and independent resource for patients and caregivers.”

Insulin And Influence

The American Diabetes Association said in an email to KHN that it received $18.3 million in pharmaceutical funding in 2017, accounting for 12.3 percent of its revenue; that was down from $26.7 million in 2015. The money flowed in as insulin makers continued to hike prices in those years — up to four times per product — leading to hardships for patients.

The only “Big Three” insulin maker in the database, Eli Lilly, gave $2.9 million to the American Diabetes Association in 2015, according to disclosures from the company and its foundation. Sanofi and Novo Nordisk are the other two major insulin makers, but neither was in the S&P 500 and therefore not included in the database. Over the past 20 years, Eli Lilly has repeatedly raised prices on its bestselling insulins, Humalog and Humulin, even though the medicines have been around for decades. The drugmaker faced protests — by people demanding to know the cost of manufacturing a vial of insulin — at its Indianapolis headquarters last fall.

The ADA launched a campaign decrying “skyrocketing” insulin in late 2016 but did not call out any drugmaker in its literature. When legislators in Nevada passed a bill last year requiring insulin makers to disclose their profits to the public, the ADA did not take a public stance.

The American Diabetes Association said it doesn’t confront individual companies because it is seeking action from “all entities in the supply chain” — manufacturers, wholesalers, pharmacy benefit managers and insurers.

“As a public health organization, the ADA’s commitment and focus is on the needs of the more than 30 million people with diabetes,” said Dr. William Cefalu, its chief scientific and medical officer. “The ADA requires support from a diverse set of partners to achieve this objective.”

Eli Lilly said it contributes money to the American Diabetes Association because the two share a “common goal” of helping diabetes patients.

“We provide funding for a wide variety of educational programs and opportunities at ADA, and they design and implement those programs in ways that are aligned with their goals,” Eli Lilly said in a statement. “We’re proud to support the ADA on important work that helps millions of people living with diabetes.”

Most patient groups say that funders have little or no influence in shaping their programs and policies, but their agreements are private.

They Weren’t Always Backed By Pharma

Into the ’80s and early ’90s, patient lobbying was generally limited and self-funded with only one or two affluent patients from an organization traveling to Washington on a given day, said Diana Zuckerman, president of the nonprofit National Center for Health Research.

But the power of patient-lobbyists became apparent after a successful campaign by AIDS patients led to government action and a national push to find drugs to treat the then-terminal disease. Zuckerman said she will never forget when two women visited her office and asked how breast cancer patients could be as effective as the AIDS patients.

“At the time, there were no breast cancer patients advocating for money or anything else. It’s hard to believe,” she said. “I still remember that conversation, because it was really a turning point.”

Soon after, breast cancer patients started visiting the Hill more frequently. Patients with other diseases followed. Over time, patients’ voices became a potent force, often with industry support.

Even some wealthy, high-profile organizations take industry money: For example, $459,000 of Susan G. Komen’s $118 million in 2015 revenue came from drugmakers in the database, according to public disclosures. Asked about the pharma money, the foundation said it has institutional processes in place to ensure that “no corporate partner — pharma or otherwise — decides our mission priorities,” including a scientific advisory board — free of sponsor influence — that reviews its research program.

Today, patient advocacy groups flush with more industry dollars fly patients in for testimony and training about how to lobby for their drugs.

Some years ago, as the groups increased in number, Zuckerman said, she started getting email invitations from advocacy groups to attend so-called lobbying days explicitly sponsored by the pharmaceutical industry. The hosts often promised training and usually some kind of keynote speaker at a luncheon in Washington — plus a potential scholarship to cover travel. Now, lobbying days involving dozens of patients from a single group are part of the landscape.

Dan Boston, president of lobbying firm Health Policy Source, said, “It would be naive to think these people on a Tuesday afternoon just happen to turn up in XYZ places,” adding that the money isn’t necessarily a bad thing. Money tends to flow toward citizen groups that already have the same priorities as their funders, he said.

Marching Into The Future

Patient groups have been successful at campaigning for drug approvals, at times sparking controversy.

When scientists within the FDA advised against the approval of Exondys 51, a drug to treat Duchenne muscular dystrophy, parents of children with the rare genetic disorder and patients rallied to lobby for it in Washington. They were seen as pivotal to the FDA’s 2016 decision to grant approval for the drug, made by Sarepta Therapeutics. The decision was controversial in part because the FDA noted that clinical benefits of the drug — aimed at a subset of people with Duchenne muscular dystrophy — were not yet established.

Sarepta Therapeutics, which is not featured in the database, has taken measures to support its patient base. In March, it announced an annual scholarship program — 10 grants of up to $10,000 each for students with Duchenne muscular dystrophy to attend university or trade schools. Sarepta Therapeutics is also among the funders of Parent Project Muscular Dystrophy, a patient advocacy group at the forefront of the push for Exondys 51’s approval.

The Pre$cription for Power database will grow to include new disclosures. Not all drugmakers are willing to disclose their company giving. Eleven of the 20 companies examined — Allergan, Baxter International, Biogen, Celgene, Endo International, Gilead Sciences, Mallinckrodt, Mylan, Perrigo Co., Regeneron Pharmaceuticals and Vertex Pharmaceuticals — declined to disclose their company giving or did not respond to repeated calls.

Paul Thacker, a former investigator for Sen. Chuck Grassley (R-Iowa) who helped draft the Physician Payments Sunshine Act in 2010, said there is reason to question the flow of money to patient advocacy groups. The pharmaceutical industry has fostered relationships in every link of the drug supply chain, including payments to researchers, doctors and professional societies.

“There’s so much money out there, and they’ve created all of these allies, so nobody is clamoring for change,” Thacker said.

Since the Physician Payments Sunshine Act began requiring the industry to report its payments to physicians, the industry is more reluctant to co-opt them, so “pharma has to find other megaphones,” PharmedOut’s Fugh-Berman said.

And in times of public outrage over high drug prices and soaring insurance costs, patients are particularly sympathetic messengers, she said.

“Sick consumers make for good press,” Fugh-Berman said. “They make for good testimony before Congress. They can be very powerful spokespeople for pharmaceutical companies.”

To learn how Kaiser Health News created the Pre$cription for Power database, read the full methodology, here.

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A PhRMA , Scott LaGanga, for 10 years led the Partnership for Safe Medicines, a  that has recently emerged as a leading voice against Senate bills that would allow drug importation from Canada. LaGanga was responsible for PhRMA alliances with patient advocacy groups and served until recently as the nonprofit’s principal officer, according to the partnership’s tax forms.

In February, LaGanga moved to a senior role at PhRMA and stepped down as executive director of the Partnership for Safe Medicines — just as the group’s campaign to stop import legislation was revving up.

Both PhRMA and the partnership have gone to great lengths to show that drugmakers are not driving what they describe as a “grass-roots” effort to fight imports — including an expensive advertising blitz and an event last week that featured high-profile former FBI officers and a former Food and Drug Administration commissioner.

The U.S. drug industry has strongly opposed efforts to open the borders to drug imports, but PhRMA is not mentioned in the partnership’s recent advertising blitz. The nonprofit said its grass-roots effort is supported by 170 members, including professional organizations and trade groups.

The nonprofit describes PhRMA as a dues-paying member with no larger role in shaping the group’s activities. Partnership spokeswoman Clare Krusing would not say how much each member contributes. PhRMA spokeswoman Allyson Funk declined to say whether PhRMA funds the partnership.

“PhRMA engages with stakeholders across the health care system to hear their perspectives and priorities,” Funk said. “We work with many organizations with which we have both agreements and disagreements on public policy issues, and believe engagement and dialogue are critical.”

LaGanga is listed as the nonprofit’s executive director on each of the partnership’s annual tax filings since 2007, the earliest year for which they are .

LaGanga wrote a about the partnership’s origins. Published in the Journal of Commercial Biotechnology, it described “public-private partnerships in addressing counterfeit medicines.” His PhRMA job was not disclosed.

From 2010 to 2014, the organization hosted a conference called the Partnership for Safe Medicines Interchange. At a 2013 event , LaGanga thanks pharmaceutical companies for sponsoring the event, most of them PhRMA members.

The partnership recently launched its ambitious ad campaign — including television commercials, promoted search results on Google and a full-page print ad in The Washington Post and The Hill. The group’s shows recent commercials targeted to viewers in 13 states.

“We don’t disclose specific ad figures, but the campaign is in the high six figures,” Safdar said.

The campaign warns against the alleged dangers of legalizing Canadian drug imports. The commercials ask voters to urge their senators to “oppose dangerous drug importation legislation.”

The newspaper ad reads, “Keep the nation’s prescription drug supply safe. Urge the Senate to reject drug importation measures.” Its splash headline declares that “170 healthcare advocacy groups oppose drug importation,” touting a . It lists 160, and PhRMA’s name is not included.

“Having a big membership allows the coalition to present what looks like a unified show of grass-roots support … but it does raise questions about which members of the coalition are really driving and funding the group’s policymaking,” said Matthew McCoy, a postdoctoral fellow at the University of Pennsylvania who studies patient advocacy groups.

The list of “grass-roots groups” includes at least 64 trade organizations representing the biomedical industry, professional associations representing pharmacists, a private research company and two insurance companies.

One group that signed the letter, the “Citrus Council, National Kidney Foundation of Florida Inc,” represents a single volunteer, according to an email from the group. A spokesman for the National Kidney of Foundation of Florida said the volunteer’s views contradict the position of the umbrella group, and said the foundation supports “any sort of drug importation that allows our patients to have access to drugs at the best price.”

Two of the hepatitis advocacy groups listed — the National Association of Hepatitis Task Forces and the California Hepatitis C Task Force — are run by the same person: Bill Remak. Remak said the groups receive small amounts of PhRMA funding.

“I don’t enjoy having to take this extreme position of saying we shouldn’t import at all, but until we have some oversight regime, some way of protecting consumers, it’s a really tough call,” he said in an interview.

“Current drug importation proposals do not appear to have equal safety and chain-of-custody accountability laid out adequately for patient safety concerns,” said William Arnold, president of the Community Access National Network, an advocacy and support group for people living with HIV/AIDS or hepatitis in Washington, D.C. His group did not accept money from PhRMA from 2013 to 2015.

Last week, the partnership hosted a panel at the National Press Club featuring former FBI director Louis Freeh and former FDA commissioner Dr. Andrew von Eschenbach. The discussion focused on the health and legal dangers of online pharmacies.

“You can talk about lowering prices, but if a drug comes with a high probability of toxicity and death, that comes at a high cost to the patient,” von Eschenbach said. “That’s what’s at issue with drug importation.”

Each speaker argued that the bill co-sponsored by Sen. Bernie Sanders (I-Vt.) would be harmful to patients. That legislation would provide a mechanism for Canadian drug manufacturers to sell to U.S. consumers and pharmacies. Sanders introduced the bill in February. Around the same time, the partnership sent emails to member organizations seeking help to stop such a measure.

In the House, Rep. Elijah Cummings (D-Md.) introduced a similar bill to Sanders’, along with 23 other Democrats. In January, Sens. John McCain (R-Ariz.) and Amy Klobuchar (D-Minn.) also to allow drug imports from Canada.

Speakers at the partnership event claimed importation would lead to a flood of counterfeit medicines laced with arsenic, fentanyl and lead paint.

“These drugs are manufactured in jungles, in tin drums, in basements … those are the sort of sanitary conditions we’re talking about here,” said George Karavetsos, a former director of the FDA’s Office of Criminal Investigations.

Josh Miller-Lewis, Sanders’ deputy director of communications, refuted those arguments in an interview. He said Canadian drugmakers can apply for licenses, and all drugs would have to come from FDA-inspected plants.

Both von Eschenbach and Karavetsos have ties to the pharmaceutical industry: Von Eschenbach left the FDA in 2009 to join Greenleaf Health, which counsels pharmaceutical clients, before starting his own consulting company; and Karavetsos at DLA Piper, a Washington, D.C., law firm.

This isn’t Sanders’ first attempt to legalize importation. But Politico reported in October that by another $100 million a year, suggesting to many industry watchers that drugmakers are gearing up for a ferocious fight.

“I think it’s safe to say pharmaceutical corporations are prepared to spend some fraction of their multibillion-dollar profits to fight drug importation and any other policy that might end the plague of overpriced medicine,” said Rick Claypool, research director for Public Citizen, a watchdog group critical of the drug industry.

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the .

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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’s capital. The money was donated by the pharmaceutical industry.

“Obviously, we wanted to send as many patients to advocate on Capitol Hill as possible,” 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’ travel expenses.

The pemphigus/pemphigoid group, representing people with a rare autoimmune disease that often 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.

Among repeat attendees at this year’s 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 “patient voice” has helped pass laws, such as the 21st Century Cures Act that patients lobbied Congress for the previous year.

“Hearing personal stories makes things just hit home more,” she added.

But some conference first-timers were skeptical about the motivations: “What I’m seeing just doesn’t feel right,” 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 “involvement drafting, creating or discussing” the messaging delivered to Congress, adding: “It’s like marketing, I’m pushing an agenda by and through a sob story.”

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 patients and advocates attended, and nearly all took part in a subsequent “Lobby Day” to visit congressional staff and lawmakers. They permitted a reporter from Kaiser Health News to observe 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 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 “asks.”

The group’s president, Dr. Emil Kakkis, is a drug industry executive. He said the foundation doesn’t “tell patients what to do on the Hill. They are given options.”

During one session called “Tricks of the Trade: Preparing for a Successful Meeting,” 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’s prearranged meeting list. Checking a map, Kush looked at the audience and said: “If you see a little dot where you live, you may have a new member of Congress — or a green check on your state, that means you have a new senator.”

He asked the audience to raise their hands if they saw dots and checks.

“Oh, here you come,” Kush said. “It’s going to be like an army walking up the hallways [of Congress]. I just got chills.”

The foundation also enlists patient advocates to woo Congress members to join its rare disease caucus 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’s outreach and how to talk with regulators. Her goal is to develop federal billing codes that could help track the prevalence of her daughter’s 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.

“It’s great PR for the pharmaceutical company,” she said.

More than that, perhaps, such events allow parents and patients with rare disease to connect with each other — many are delighted to realize they’re not alone in their suffering.

“I’ve watched kids sometimes who felt so timid and afraid and shy to share their stories learn to embrace it as part of their life,” Muller said. “And I think that’s been the most incredible part for me.”

A Coordinated Strategy

One focus of this year’s conference was the OPEN Act, which provides a financial reward for drugmakers who do clinical trials to repurpose an existing drug to treat a rare disease. A white paper explained the bill to participants and gave tips for what to say on the Hill.

In what appeared to be a coordinated strategy, Rep. Gus Bilirakis (R-Fla.)  the OPEN (officially the Orphan Product Extensions Now, Accelerating Cures and Treatments) Act on the House floor 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 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’s Julia Jenkins. Jenkins said the bill is expected to be introduced in the Senate soon.

If 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 “double the number of treatments available” and generate lower-cost medicines for the rare disease community.

But in a , Harvard’s Dr. Aaron Kesselheim argued that the law offers unnecessary incentives for manufacturers that already have blockbuster drugs.

Mixed Motivations

The EveryLife Foundation’s Rare Giving program covered about 100 travel stipends for conference attendees. While that is largely paid for by the foundation’s board members, including Kakkis, Jenkins said the group is “actively trying to get industry to fund the program.”

Top donors to EveryLife include pharmaceutical companies AbbVie, Alexion, BioMarin, Raptor Pharmaceutical and the Kakkis family,Ìýaccording to the foundation’s most recent annual report. EveryLife  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 .

Kakkis is forthright about wearing multiple hats — posting his affiliations 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’ foundation — with his army of advocates — lobbied for the Cures Act. One of the patients, Max Schill, was the little boy who  congressional leaders as they signed the bill to send to President Barack Obama. Key backers included EveryLife’s 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’s process to win approval for Aldurazyme, an enzyme replacement therapy he developed to treat one form (MPS I) of a group of disorders known as mucopolysaccharidoses.

Eventually the drug was approved, he said, but with a three-year delay. And, during that time, “a number of MPS I patients passed away,” Kakkis said.

In Kakkis’ view, drug companies and patients have a shared interest in getting drugs developed and approved.

“I do think the company’s interest [and patients’ interests] are not misaligned,” he said.

‘That’s The Way Washington Works’

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  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 — including EveryLife — to speak at NIH during the agency’s annual rare disease day.

“I have no idea what their funding is, we just look at the work they do,” said Austin, director of the National Center for Advancing Translational Sciences.

Speaker Max Bronstein, EveryLife’s 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 “the legislation we’ve been working on for a few years.”

Austin said he was aware that Kakkis was both the head of EveryLife as well as Ultragenyx.

“You know, Washington is full of those people,” Austin said. “As I’ve learned being here, that’s the way Washington works, for better or worse. It’s a little stinky frankly, but there you are.”

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the .

ºÚÁϳԹÏÍø News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF—an independent source of health policy research, polling, and journalism. Learn more about .

USE OUR CONTENT

This story can be republished for free (details).