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September 7, 2017, 2:04 PM EDT

A week after its landmark approval of the first treatment thatworks by genetically altering a patients 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. FDAs goal is to make sure that our policies are as scientifically advanced as the products were being asked to evaluate.

Gottllieb vowed to betteradjust to the complexities of novel therapies and make sure the agencys 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 patients own immune system to attack tumors, including Kite Pharma Inc., which is awaiting approval for a CAR-T treatment. FDAs faster-than-expected approval of Novartiss 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.

Some academic and industry drug developers arent fully aware of what is is needed to get a new product approved, particularly smaller groups that often are working with the most innovative technology, he said. Others overestimate the amount of information the agency needs to start studies in patients. FDA staff will work with researchers to eliminate unnecessary steps and incorporate new testing approaches that may help cut costs and speed the approval process, he said.

Information that is gleaned from early work in CRISPR, a technology that allows researchers to easily manipulate genes in a way that many hope will one day be used to treat disease, may be used to hasten development of other products, the commissioner said.

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In many cases, the main challenges of novel medicines arent clinical questions about how well they work and their immediate safety, but newer issues related to how they are produced and delivered to the patient, the commissioner said. Manufacturing CAR-Ts like Kymriah, for instance, involves extracting infection-fighting cells from the patients blood; sending them to a centralized plant in New Jersey to get re-programmed; and shipping them back to be re-infused into the patient at medical centers.

The new therapies may also carry long-term risks that may not materialize for years -- if ever. Attention will be needed on how the therapies hold up during routine use, with risks and benefits that are closely monitored for years, he said.

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FDA Vows Modernization to Keep Up With Biotech Advances - Bloomberg

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