Do Neurology Patient Advocacy Groups Wield Too Much Power?

Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.

On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.

At the US Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).

Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.

“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of Medical Ethics and Health Policy at the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, told Medscape Medical News. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence,” he added.

Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, McCoy noted. “There is the potential for the rich to get richer,” he said.

A Seat at the Table

Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said McCoy.

Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.

The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.

As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.

Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.

Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. As reported by Medscape Medical News, the committee ultimately voted against approval. Six months later, the FDA approved Relyvrio anyway.

In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.

The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. As previously reported by Medscape Medical News, at a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.

In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.

In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.

Patient Voices the ‘Secret Sauce’

Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When The Michael J. Fox Foundation wanted to gather co-sponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.

Having those individuals “making the personal case for how this disease affects their families…was really the secret sauce,” in garnering a large number of co-sponsors and getting legislation signed into law within 2 years of its introduction, Thompson told Medscape Medical News.

ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.

Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.

But some said patient advocates are still coming far too late to the party.

“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told Medscape Medical News. There should be more focus on the patient perspective earlier in drug development and trial design, Rand added.

“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of Medical Ethics and Health Policy at the Perelman School of Medicine, University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter, Lynch told Medscape Medical News.

But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” She noted that individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”

If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Lynch.

Does Taking Industry Money Equal Conflicts of Interest (COIs)?

Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.

As reported by Medscape Medical News, the Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.

It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, McCoy and colleagues reported in JAMA Internal Medicine that between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had COIs, mostly from industry, and that they were not disclosed in approximately 20% of the instances.

In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.

McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference,” he said. “The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well,” McCoy said.

“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, he said, “the foundation has never specifically endorsed a product or device.”

When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Thompson.

Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag, she said. “You don’t want to bite the hand that feeds you, right?”

Both McCoy and Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.

Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said McCoy.

“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.

Lynch agreed.

Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices, get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.

The group may be at the table, “but they’re just one voice at the table,” she said.

McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Rand reported no relevant financial relationships. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.

Alicia Ault is a Saint Petersburg, Florida-based freelance journalist whose work has appeared in publications including JAMA and Smithsonian.com. You can find her on X: @aliciaault.