FDA Vows Modernization to Keep Up With Biotech Advances

Thursday, September 7, 2017

A week after its landmark approval of the first treatment that works by genetically altering a patient’s cells, the U.S. health regulator vowed to modernize to keep up with the fast-moving field of biotechnology research.

“We are at a point in the history of medicine that is similar to other great inflections in science where fundamental principles of science and medicine became firmly established as part of a leap in public health,” Food and Drug Administration Commissioner Scott Gottlieb said in a speech in Washington. “FDA’s goal is to make sure that our policies are as scientifically advanced as the products we’re being asked to evaluate.”

Gottllieb vowed to better adjust to the complexities of novel therapies and make sure the agency’s policies match the challenges faced by companies looking to follow in the footsteps of Novartis AG, whose Kymriah drug was just approved for pediatric patients with a hard-to-treat form of leukemia. The FDA is evaluating more than 550 applications to test gene therapies and 76 related to CAR-T, the same class of compounds as Kymriah, Gottlieb said in his address to Research America, a nonprofit public education and advocacy group dedicated to health and science research. 

The speech could be good news for the developers of therapies using the patient’s own immune system to attack tumors, including Kite Pharma Inc., which is awaiting approval for a CAR-T treatment. FDA’s faster-than-expected approval of Novartis’s Kymriah sent biotech shares higher last week. That optimism was tempered this week when one drugmaker halted two studies of an experimental blood cancer therapy after a patient died -- a reminder of the major risks and safety concerns in the nascent field.

Gottlieb focused his talk on the earliest stage of drug development, before potential new compounds make it into patients. The agency will engage earlier with companies and researchers working in these new areas, including technology platforms like gene therapy, cell therapy and regenerative medicine, he said.


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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