Janet Woodcock Pulled a Frances Kelsey and Everyone is Mad

In FDA’s long and illustrious history, there are very few watershed moments that can be attributed to one person at the Agency. FDA historians consider the decision by Frances Kelsey to not approve thalidomide in 1960, despite extensive pressure to do so, as one of pivotal moments in its history that defined how it regulates drugs. That decision is credited with leading to the Kefauver-Harris Amendment of 1962 whereby manufactures had to prove safety and efficacy of drugs prior to approval, one of the most impactful laws for the drug industry. In July of last year, something similar happened. FDA was under intense pressure to make a decision regarding eteplirsen, a drug to treat Duchenne Muscular Dystrophy (DMD). The review team at FDA’s division responsible to approve the drug had unanimously rejected the drug; the Advisory Committee had given a non-committal vote of 7-6 against approval, while the parents of children with DMD, and the company via a very aggressive social media campaign was pushing for approval despite the concerns about the drug’s efficacy. One person, Janet Woodcock, the well-known Director of the Center for Drug (CDER) decided almost single-handedly (based on publicly available internal memos and other sources) to overrule both the review team and the Advisory Committee to approve the drug, and succeeded in doing so. Say what you may about the decision, this episode will be remembered as a watershed moment for how FDA and patients can interact regarding important decisions that affect both. Prior to July, 2016, FDA had already rejected two other drugs for DMD due to safety concerns, and the manufacturers for those had announced suspension of further development. Eteplirsen was the only drug left with a pending FDA decision. The drug was safe but its effectiveness could not be clearly demonstrated in the clinical trials. Patients were desperate for hope; and hope is more powerful than any drug. The company hired consultants to coach the patients’ families to collect anecdotal data outside the clinical trials and trained them in FDA lingo. The patient families met Dr. Woodcock directly multiple times to plead the case in support of the drug. The review team was not moved, as it should not. The review team’s job is to make unemotional and unbiased decisions based the quality of data. But for the last few years, there has been increasing emphasis at the FDA to give higher weight to the voice of the patients. Someone at FDA had to make the tough call and Dr. Woodcock stepped up and “pulled a Kelsey” on everyone. Since the decision, there have been numerous articles deriding the decision. The same newspapers that used to run long editorials on why FDA’s rejection of eteplisen would be bad for the patients are now running long essays on how the company manipulated the system and specifically Dr. Woodcock. Her boss at the time, the FDA Commissioner Robert Califf, who refused to make a call at the time and left it to her “judgment”, has stated publicly how he did not support the decision. And this will continue for some time. But one thing is certain, this episode will be reviewed for a long time by FDA historians as the first major FDA decision made almost entirely based on the patients’ opinion of what they want from FDA. There are multiple facets to every episode but patients have an FDA-approved drug thanks to primarily one key figure, Janet Woodcock.