RARE Daily

Retiring Woodcock Reflects on FDA Controversies

January 30, 2024

Rare Daily Staff

U.S. Food and Drug Administration’s Principal Deputy Commission Janet Woodcock, who is retiring after 37 years with the agency, said rare disease advocates should start early in their advocacy to develop robust natural history data in an interview with the news site Endpoints.

“Don’t wait to advocate for companies to do the best trials possible. So don’t just lobby the FDA when you say it should get over the finish line because you’re so desperate,” she told Endpoints Senior Editor Zachary Brennan. “Preparation is very important.”

The comments came in a wide-ranging Q&A with Woodcock as she plans to retire next week.

Asked about the increase in accelerated approvals in recent years and whether the FDA had become more “lenient,” Woodcock thought “lenient” was not an apt descriptor. Instead, she pointed to changing science and noted that the majority of accelerated approvals had come in the area of cancer and that survival rates had steadily increased over the last couple of decades largely due to therapeutic advances.

“We see people quibbling over one or the other accelerated approval. If one accelerated approval doesn’t show it actually is that effective, people want to throw the baby out with the bathwater,” she said. “That’s a normal human instinct. But you have to look at the big picture.”

Asked about the controversial decision to grant accelerated approval of Sarepta’s Duchenne muscular dystrophy therapy eteplirsen despite the objection of staff, she said she could have handled the process better to avoid some of the controversy.

“When I said in my memo, these children become paralyzed, many of them by 18 years of age, but if they can still move a computer mouse around and communicate, it’s tremendously impactful,” she said. “I felt there was enough evidence that that would happen. But that was a judgment call.”

Photo: FDA Principal Deputy Commission Janet Woodcock

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