Patient Advocacy Groups: Help or Hindrance to FDA Drug Approval?

The battle over the FDA’s potential approval of eteplirsen, a drug to treat Duchenne muscular dystrophy, demonstrates the power of patient advocacy groups.

The FDA is subject to criticism on a weekly, if not daily, basis and April 25 was no exception. Late that day, an FDA advisory committee voted that an experimental drug to treat Duchenne muscular dystrophy (DMD), a debilitating degenerative disease, did not demonstrate efficacy and recommended against approval.

The vote was a devastating blow to the hundreds of parents, patient advocates and boys with DMD –- an unusually large attendance for such a meeting — who had packed a Maryland hotel conference room to persuade committee members to approve eteplirsen. Those who wanted to see a decision in favor of approving eteplirsen were outraged and channeled their anger toward the committee members.

Their only problem? The committee got it right, making its decision based on a lack of sound scientific evidence supporting the drug’s efficacy rather than listen to public pressure.

In the trial for eteplirsen, Sarepta Therapeutics enrolled only 12 young boys, a small number. And to make things even more difficult for Sarepta, the company didn’t include a placebo group in the trial, a protocol that is used in nearly all studies that lead to approval of a drug. Some members didn’t think the trial was designed in a way to properly show effectiveness.

But that didn’t matter, at least to some patient advocates in attendance.

“The worst thing you can do is deny access to a drug and then find out it works after we’ve lost a generation of boys,” Debra Miller, founder of the advocacy group, CureDuchenne, told The Boston Globe after the vote.

But if you thought this was the end of the road for eteplirsen, you are wrong. In fact, it was just the beginning.

“It’s not over,” Miller told the newspaper. “It wasn’t just the panel that heard the patient testimony. The FDA heard it, too, and their mandate is to listen to the patient perspective.”

The battle over eteplirsen is a prime example of how patient advocates are playing an increasing role in the FDA’s drug approval decision-making process. While patient advocacy has been a part of this process for decades, increasing pressure is being put on the agency to approve more drugs, and not necessarily to the benefit of patients.

The FDA may well appease these groups, but at the risk of discarding the necessary scientific proof that has been a hallmark of FDA drug approvals, potentially giving patients false hope of a medication’s efficacy, or worse, approving drugs that have serious side effects and risks. And it means succumbing to public pressure rather than listening to a group of medical experts.

After the vote, eteplirsen supporters sprang into action, lobbying their local congressmen and senators to do something to get the only drug out there that they claim has benefited boys with DMD, and penning op-eds in local newspapers to drum up support. A group of Republican senators even sent a letter to the FDA expressing their disappointment over the vote.

Time was of the essence, as the FDA was slated to make a decision on whether to approve eteplirsen by May 26. And on May 25, advocates caught a break. Sarepta said that the FDA was delaying its decision to review the eteplirsen application for a longer period of time. This would give the advocates even more time to lobby and put pressure on the FDA.

Earlier this month, Sarepta said it had received a request from the FDA to provide additional data from its clinical trial of 12 boys. Some observers saw the FDA’s request as an indication that the agency is looking for ways to approve the drug, over-riding the consensus of its advisory committee members.

No final decision has been made on eteplirsen’s fate, but patient advocates are still exerting pressure. There’s nothing wrong with wanting to have a drug approved that could save your child from a debilitating illness that claims the lives of boys while still in their 20s. Anyone can sympathize with these parents.

However, approving eteplirsen puts the FDA on a slippery slope where emotions, pressure and elected officials have a bigger influence on whether drugs get approved rather than medical experts who are in the best position to make such decisions.

Jonathan Block

Jonathan Block

Jonathan Block is an associate editor at BioCentury, which provides news and information about the biotechnology and pharmaceutical industries. Prior to joining BioCentury in 2019, Jonathan worked for MedShadow as content editor. He has been an editor and writer for multiple pharmaceutical, health and medical publications, including The Pink Sheet, Modern Healthcare, Health Plan Week and Psychiatry Advisor. He holds a BA from Tufts University and is earning an MPH with a focus on health policy from the CUNY Graduate School of Public Health & Health Policy.

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