The FDA's press release announcing the accelerated approval of Exondys -- the first drug for Duchenne muscular dystrophy -- sends a very important message.
The prose is PR-speak 101. The meaning is in the messenger:
"The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
Why is this release different from all other releases?
In most FDA releases about new drug approvals, the director of the division that reviewed the drug is quoted.
In this release, Janet Woodcock, who runs the whole show is quoted.
Woodcock has been publically involved in taking the FDA's good intentions about involving patients in the design of clinical trials and in defining study endpoints as well as encouraging scientifically rigorous alternatives to randomized clinical trials that can bring medicines to market faster as well as allow more patients to participate in a study without randomization.
After the FDA committee initially rejected Sarepta's application for accelerated approval, virtually attacking the patient community for not supporting a randomized trial.
So of course so-called patient advocacy organizations that attack and question new medicines at every turn hailed the first denial. The expertly and eternally ignorant Diane Zuckerman, president of the National Center for Health Research sums up the anti-innovation tunnel vision: "If this drug is approved — on the skimpiest, most pathetic data I’ve ever seen — then it is going to set a precedent that is very uncomfortable."
Woodcock stepped in at that meeting to note that too often the FDA focuses on not approving drugs because they are relatively safe and effective but that it often engages in "little consideration of another error, which is failing to approve a drug that actually works”. But most of this consequence is borne by patients who have little say."
Patients had a lot to say about the study design and basis for Exondys 51's approval. The FDA's bureaucrats and it's supporters among the handful of elites used the initial denial to derail such involvement. How ironic that these individuals, who pontificated about using data, not emotion, to determine drug approvals, made extreme and factually false statements (See, Zuckerman, Diane) before and after the denial.
Exondys 51 is now approved. So too is a pathway that allows patients who bear the risk of clinical trials and the burden of disease to have a say in how medicines should be measured and used. DMD advocate Christine McSherry, Executive Director of the Jett Foundation, stated that the DMD community’s involvement in drug approvals “will change the way people confront barriers to justice in society. This will be historic not just in Duchenne, not just in rare disease, but for every ordinary individual who has felt the need to change a broken system but never knew how to do it.”
Christine McSherry and her son Jett
Increasingly medical advances determine not only whether we live or die, but how we will live and die. Ordinary citizens, like Christine McSherry who are potential patients and the friends and relatives of such patients have as much to say on these innovations as any so-called expert.
That's what Janet Woodcock's announcement means.