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Meet the Regulators: FDA’s Top Officials Join BIO “Town Hall”

June 6, 2018
Top U.S. Food and Drug Administration officials outlined the agency’s modernization efforts and proposed reorganization, discussed the challenges and opportunities related to the emergence of new therapeutic modalities, and touched on subjects as diverse as the opioid crisis and the affordability of medicines during a wide-ranging Town Hall discussion with the Chair of BIO’s  Board of Directors John Maraganore, Ph.D on Tuesday.

FDA’s modernization plans are about “much more than restructuring,” said Janet Woodcock, director of FDA’s Center for Drug Evaluation and Review. The regulatory revamp includes a significant redesign of drug review processes, review documentation, standardization of regulatory procedures and project management, and an increase in support structure at FDA, she said. All of these proposed changes would help FDA “do a better job in overseeing drug development, providing advice and guidance, and interacting with the outside world,” said Woodcock.

The agency’s modernization in some ways mirrors the new science it’s tasked with regulating. “We’re at this inflection point where gene therapy and cell therapy are really ready to take off,” said Center for Biologics Evaluation and Research director Peter Marks. “I don’t think this is ‘fake news,’” he said, noting the 2017 approvals of the first gene and cell therapies in the US. “It’s a reality.”

Potentially hundreds or even thousands of rare diseases may become addressable by gene therapy, said Marks, but the challenge will be that these are all small populations. Honing clinical development strategies and manufacturing processes for treatments with so few patients will be critical. Older biopharmaceutical development paradigms - where pilot manufacturing stages yield to development and commercialization stages in a process that could take years - simply won’t do for modern medicines. “In the old paradigm that worked,” because trials were big and slow-moving,” he said. But after achieving promising results in trial of a gene therapy of patients with a fatal disease it would be “heart-wrenching” for patients to have to shut down production to scale up, he said. “We have to figure out how to bridge that gap. We have to start thinking creatively.”

Meanwhile these treatments are currently too expensive to make to be affordable in the context of common diseases, said Woodcock. Right now industry’s in a good spot as far as innovation, “but it isn’t sustainable,” she said. The cost of making enough product to supply larger clinical studies would be prohibitive, which will ultimately limit the therapies’ utility without manufacturing advances, the officials said. “A trial of thousands of individuals for a cardiac gene therapy would cost more than the GDP of some European countries,” said Marks.

Another challenge with new therapeutic modalities is understanding that in some cases, “we’re in new territory, and we’re not going to get it right the first time,” said Marks. How the FDA regulates gene and cell therapy will naturally evolve, and the challenge will be having to learn on the fly, he said.

The FDA officials also touched on some hot-button regulatory issues. They discussed patient-focused drug development, which Woodcock noted would “really become a force over the next decade. Patients are on fire about getting into the tent all the way and having a voice,” she said.

In a discussion about FDA’s role in combating the opioid crisis, Woodcock noted that new guidance for industry about pain treatments was forthcoming, covering different drug development scenarios, and how companies can design trials to be able to make claims that their treatments help avoid the use of opioids.

Finally, Maraganore asked the regulators about generic competition as the primary means of controlling the cost of medications. In 2017, the FDA approved more than 1,000 generic drugs, said Woodcock, fully recognizing that with each available generic, drug prices come down as competition increases.