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medical review team and top drug evaluator Janet Woodcock drove the decision, not the commissioner. “The review always had to be based on science, on medicine.”
In an Endpoints News story published after the POGO report, an FDA official said Gottlieb “wasn’t involved in this matter and further, wasn’t aware of the letter.” The official added that FDA’s drug review decisions are “based on specific criteria applied to the product’s data and determined by appropriate FDA career staff.”
Crowley instead points to another reason: the December 2016 passage of the 21st Century Cures Act, a massive healthcare spending and reform bill that included a variety of new tools to help speed up drug reviews. Among other things, the law included mandates to incorporate patient perspectives into various stages of drug development and review, and to modernize clinical trial designs to make better use of biomarkers that could predict a drug’s benefit. The FDA has taken “patient-focused drug development” to heart, with the agency’s top drug evaluator, Janet Woodcock, being a key proponent. The act “really began to change the mindset at the FDA in a very clear way,” and Amicus was a direct beneficiary, Crowley says.
Crowley says Amicus went back to the agency in early 2017, and the questions the company got from medical reviewers were different. As expected, the agency wanted more data than Amicus had previously submitted, but it now also wanted to learn about patient experiences and asked for case studies and patient narratives. Crowley says Amicus put a book together incorporating that information, new data from patients on the drug commercially in Europe, as well as data on the effects of long-term treatment with the drug—in some cases a decade. In the meantime, more updated results from Amicus’s clinical trials that weren’t included in earlier briefing documents to the FDA were published in medical journals. And according to Crowley, Fabry patients stepped in, talking to the FDA about the need for new options, how the heart and kidney problems are deadly and migalastat might be able to help.
(Xconomy reached out to the National Fabry Disease Foundation and will update this story with any comments.)
Crowley says Amicus ultimately got a phone call in July 2017 asking how quickly the company could get a drug application together. The application was finished by the end of the year, and given a “priority review,” which cuts a few months off of the FDA’s typical review process.