FDA to issue guidance on cell and gene therapy in July
Scott Gottlieb has kept himself busy during his tenure as head of the Food and Drug Administration.
Scott Gottlieb has kept himself busy during his tenure as head of the Food and Drug Administration.
And the agency chief made clear in a fireside chat at the BIO International convention that the FDA has plenty coming down the pike in the months ahead — particularly in the areas of cell and gene therapy.
Gottlieb pointed to the hundreds of Investigational New Drug applications currently in place for cell and gene therapies, citing one study that suggests as many as 40 of these drugs could be approved over the next few years.
"There"s no question you can see multiple curative therapies in the near future," said Gottlieb about the space. "If I had come on this stage five years ago and said we can cure some inherited blood-borne illnesses like sickle cell, I think that would"ve been irresponsible. Now within the next ten years we"ll be able to do that."
While Gottlieb did acknowledge risks related to any curative drugs, he said "product innovation has led to [a] clinical revolution," pointing to the advent of viral vectors that can now act as a delivery mechanism for these therapies.
"A lot of the complexity with gene therapy is in product-related issues, not the clinical issues. Whereas with normal drug review, I"d say 80% is the clinical portion and 20% is the CMC and product portion of the review," he said, referring to the Chemistry, Manufacturing and Controls section of an application.
"I think with gene therapy and cell-based regenerative medicine it"s completely inverted. We"re having to think very differently about the regulatory issues with these."
The FDA intends to release six new sets of guidance in July that will lay out the "modern framework" for the agency"s approach to these therapies, many of which will be focused on product-related issues, whereas others will focus on accelerated clinical endpoints.
"We"re focusing, at least the initial guidance, on product areas where there"s a lot of activity going on because we want to objectively find what the clinical endpoints should be," he said.
Gottlieb also announced a new program within the Center for Biologics Evaluation and Research, called "Interact," for pre-clinical, early engagement for cell-based and regenerative therapies.
"We"re going to create a formalized structure for early engagement, before the IND stage, about how to get products into the IND stage, solving for the CMC issues and solving for some of the clinical development issues," Gottlieb said.
He also indicated the industry needs to move toward more continuous manufacturing for cell and gene therapies to create a more "seamless transitions between research-grade and commercial-grade manufacturing."
He noted there was a substantial budget request for 2019 for money to advance public-private partnerships related to continuous manufacturing.
"One of the principal advantages is going to be better cost efficiency and better scale up around manufacturing when it comes to cell-based regenerative medicine, gene therapies, and vaccines, for that matter — moving away from egg-based production of the flu vaccine," he added.
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