Billy Ellsworth, a teenager with an inexorable and devastating degenerative muscle disease, will bring a football with him to a Maryland hotel conference center on Monday. For months, he has been brainstorming a way to prove to a panel of scientists and physicians that the experimental drug he has been taking for more than four years has kept him strong and well — and he’d like to punctuate his brief testimony in the clearest possible way: by throwing them the ball.
When he was 4¼, Billy was diagnosed with Duchenne muscular dystrophy, a rare and lethal disease that typically forces boys to use wheelchairs by their teens and kills them in their 20s or 30s. His mother, Terri, has always measured her son’s life in fractions; Billy is 15¼ now, and the reason he isn’t in a wheelchair yet, they believe, is the experimental drug eteplirsen.
On Monday, the panel of scientists and physicians who catch Billy’s pass — if he’s allowed to throw it — will vote on whether the drug is effective. It is more than just a major make-it-or-break-it milestone for the Massachusetts drug company Sarepta Therapeutics and the families who support approval. The meeting also provides a window into the growing sophistication of grass-roots patient groups, whose well-organized lobbying efforts are exerting intense pressure on the drug-approval process. The Duchenne parent groups are orchestrating webinars on how to participate in the meeting, providing travel grants to help families attend, gathering their own data on the risks families are willing to accept and lobbying Congress. […]
The FDA has been working to incorporate the patient perspective into the drug-approval process, especially over the past five years, under growing pressure from advocacy groups and Congress. The Duchenne parent groups have been especially active on this front, running studies of the benefits and risks patients and caregivers are willing to accept — to try to “quantify the tears” for regulators as Pat Furlong, president of the Parent Project Muscular Dystrophy, says.
The parent group led a coalition that drafted a paper to help guide companies on best practices for successfully creating drugs for the disease that the FDA credited and drew from when it published its own guidance last summer — a first that other disease groups are working to follow. This month, they have worked to get two dozen senators to sign a letter reminding the FDA to prioritize the patient perspective when considering drugs for diseases with no treatments.
The patient view can be extremely valuable: Patients know better than anyone what a meaningful benefit would be or what risks they would accept. But the patient’s view on whether the drug works may be influenced by hope. […]
More recently, Diana Zuckerman, president of the National Center for Health Research, pointed to last year’s controversial drug approval of a female libido drug, flibanserin, as a milestone that might have emboldened Sarepta.
“In that case, a campaign by patients persuaded an FDA Advisory Committee and the FDA to approve a drug they knew had little benefit and substantial risk,” Zuckerman wrote. “Sarepta probably assumed a campaign by desperate parents would be even more persuasive.” […]
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