FDA Chief Calls for More Focus on Clinical Use of New Products

Thursday, September 7, 2017

The FDA's oversight of new drugs and devices would shift the emphasis from the investigational stage to the patient care setting under a plan to overhaul the agency's regulations.

Scott Gottlieb, commissioner of the Food and Drug Administration, said the increasing complexities of new medical technologies, such as Novartis's CAR-T cell therapy to treat blood cancers, challenge the agency to adjust regulatory policies. In remarks made at Research!America's 2017 National Health Research Forum Sept. 7, Gottlieb said these advanced technologies involve situations where new medical products are delivered transiently and sometimes in just a single use.

“But they have sustained effects,” Gottlieb said. “In these situations, determining safety and effectiveness doesn't boil down a judgment made at a single point in time.”

One of the concerns raised by some Democrats after President Donald Trump nominated Gottlieb for the job were his ties to drug companies and whether he would relax the FDA's standards for approving new drugs. Gottlieb said during his Senate confirmation hearing that choosing between speed and safety was a false dichotomy.

Ongoing Safety Evaluation

The proper evaluation of safety in these cases requires an ongoing process that, he said, crosses over the threshold of initial approval.

“This means more emphasis needs to be placed on how new technologies perform in clinical use during routine care, long-term risks and benefits need to be carefully monitored and this is similar to the way we evaluate many medical devices,” Gottlieb said. “Our questions related to these platforms would increasingly relate to their long-term performance. More of our performance will naturally shift to post-market tools.”

“Increasingly with personalized medicine therapies the FDA will have to shift its approval algorithm from relying on pre-approval data on safety/efficacy more to some pre-approval data supplemented with post-approval data,” Carol Pratt, an attorney with Lee & Hayes who specializes in FDA regulation, told Bloomberg BNA. Pratt is a Bloomberg BNA health care advisory board member.

The 21st Century Cures law (Pub. L. 115-244), the bipartisan biomedical innovation law to spur new drugs and devices, gave the FDA certain tools and resources to address the challenges that come with advancing science, Gottlieb said.

“But we know much work remains and our ability to fully capitalize on this science and maintain FDA's gold standard for product review means that FDA also needs to modernize itself along with the new platforms that we're evaluating,” he said.

The regulatory overhaul goes back to the 2004 FDA Critical Path Initiative to improve medical product development.

“Now at FDA we're going to be taking some fresh steps to continue to advance similar ambitions as it relates to the development, challenges associated with these very novel platforms that we're seeing now,” he said.

http://news.bna.com.mutex.gmu.edu/lsln/ (subscription only) 

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We have health challenges in this country that science will provide answers for if given the chance and we haven't given science that opportunity
Mary Woolley, President and CEO, Research!America