E18:  FDA's Rare Disease Conundrum
Mary Parker

E18: FDA's Rare Disease Conundrum

Genome mapping has made personalized medicine achievable, but how can the FDA balance safety requirements with the speed that is necessary for desperate patients?

For patients with rare genetic mutations, gene mapping and gene therapies are a ray of hope. However, even drugs that are intended for only one patient need to go through FDA approval. In cases where the patient has no other options, and when speed is essential to success, how do regulators navigate the pharmaceutical equivalent of triage medicine?

In this episode of Sounds of Science, I am joined by Charles River Distinguished Scientist Dr. Lauren Black. She has been instrumental in navigating several drugs through the FDA approval process, and in the past year two of those drugs have made it to their intended patients: Mila Makovec and Jaci Hermstad.

According to the FDA, there are currently four possible regulatory paths for faster approvals of essential drugs: Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track. Each workflow is designed for slightly different scenarios – for example, Accelerated Approvals can be granted for essential drugs that may a long time to show clinical benefit. But what they have in common are the patients they serve: people with dangerous diseases who need help fast.

Dr. Black and I discuss her background at the FDA during the AIDS crisis, her work on behalf of both Jaci and Mila, and how the new era of personalized medicine can be accommodated by the old methods of safety assessment.

If you would like to learn more about Jaci Hermstad, check out this Eureka blog article. And you can learn more about Mila and her impact on the future of drug development.