Commissioner Gottlieb, CAR T-Cells, & The Future Of Rare Disease Therapies

Pharmaceutical Online- 

What do Commissioner Gottlieb’s recent statements on orphan drug innovation—coupled with the FDA’s first gene therapy approval this summer (for the CAR T-Cell therapy, Kymriah)—mean for the industry? In her Sept. 27 article in Pharmaceutical Online, Catalyst’s Jill Hartzler-Warner explores some of the policy changes taking place in the rare disease space at FDA, and what could happen in the future, as new policies unfold.

Gottlieb has taken the helm of FDA at an exciting time, when insights from genomics are starting to come to fruition and promising new therapies based on new technologies are being developed. Given these advances, he plans to issue a new Medical Innovation Development Plan soon that will serve as a blueprint for the changes he will seek in order to foster innovation across FDA’s medical product centers. As Warner notes, Gottlieb has also emphasized the importance of incentivizing drug makers to develop treatments for rare diseases; creating and modifying incentive pathways will be a top priority for him.

Under Gottlieb, we are seeing changes such as recognizing the unique nature of platform technologies, modernizing how clinical information is collected and evaluated, and increasing opportunities to communicate and interact with review staff for new technologies. “The race is on, and the finish line is not yet in sight,” she says.