Seeking a new path for FDA regulation of revolutionary medical treatments


Researchers are developing therapies that could permanently alter a patient’s genes… . The early results are promising. Patients would live longer, better lives. …the potential savings for insurers—and for Medicaid—are enormous. But when?

Part of the answer depends on the Food and Drug Administration, where hundreds of applications involving gene therapy for various diseases are pending.

“It used to be that the model was to develop a drug that was going to be administered chronically over the life of a patient,” [FDA Commissioner Scott] Gottlieb says in a recent interview at the FDA’s headquarters. … And now, the model is to try to develop curative therapy,” usually a short course or a one-time treatment. “It’s a completely different therapeutic model. It’s a completely different payment model, and our payment system isn’t adapted to that.”


Is the regulatory system? The FDA is used to reviewing synthetic chemical drugs, but much of the innovation today is in “biologics,” treatments derived from living things, a category that includes genetic therapies. …

Related article:  Can we afford gene therapy’s million-dollar price tags?

The FDA already has a route known as “accelerated approval” for drugs whose trial results are believed “reasonably likely” to predict clinical benefit. The early approval is then intended to be cemented over time with long-term data. Dr. Gottlieb says this process is well-suited to the new challenge of genetic therapies.

Read full, original post: When Medical Innovation Meets Politics

Outbreak Daily Digest
Biotech Facts & Fallacies
GLP Podcasts
Infographic: Here’s where GM crops are grown around the world today

Infographic: Here’s where GM crops are grown around the world today

Do you know where biotech crops are grown in the world? This updated ISAAA infographics show where biotech crops were ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

* indicates required
Email Lists
Send this to a friend