Comment from Bower, Kaylin
AnonymousOpposeIndividual
Summary: The commenter, likely a former FDA official or a scientific professional, argues that patient-focused drug development can lead to political pressure and intimidation influencing FDA decisions. They express concern that public activism might be prioritized over scientific data, potentially undermining the integrity of the drug approval process for rare diseases.
This comment regards docket: FDA-2026-N-3947: "Impacts of Patient-Focused Drug Development Meetings; Establishments of a Public Docket; Request for Information and Comments":
Per the "Agency Scientific Dispute-Appeal" by Dr. Ellis F. Unger (initiator) dated July 18, 2016 Re: NDA #206488 (eteplirsen/Exondys 51), this is what patient-focused drug development should NOT be: "Approval of this NDA would send the signal that political pressure and even intimidation-not science-guides FDA decisions, with extremely negative consequences (See Grainger D., 11/30/15). 'DMD Drugs: an existential threat to FDA,' Forbes). The public is well aware of this development program: the meager size of the study population, the marginal (at best) effect size, the Division's dim view of the efficacy data, and the robust activism of some members of the DMD community. Many would be amazed at an approval action, because other DMD drugs, recently turned down for approval, appeared to provide stronger evidence of efficacy.
FDA and Congress were bombarded with correspondence-pleas urging approval of this NDA. More than 50 speakers registered to speak at the April Advisory Committee meeting. I received 2,792 emails urging approval. Here is an example of the body of an email I received last week:
'Dear Dr. califf: how is that everyone in and around DMD understands this simple Idea and the science geniuses at FDA don't? You stupid f _ _ _ ers are costing each and every DMD kids days of their lives with your Moronic Dystrophin dance. Time to get a f _ _ _ ing clue.'
The ramifications here are profound. The public will perceive that it was their unprecedented lobbying efforts that made the difference and earned eteplirsen its accelerated approval. For the future, this will have the effect of strongly encouraging public activism and intimidation as a substitute for data, which is one of the worse possible consequences for communities with rare diseases. This type of activism is not what was envisioned for patient-focused drug development" (Scientific Appeal-NDA 206488-Pages 23 and 24)