US FDA unveils new pathway to approve personalized therapies

By Michael Erman

NEW YORK (Reuters) -The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the agency's top officials said on Wednesday.

FDA Commissioner Marty Makary and Chief Medical and Scientific Officer Vinay Prasad said in an essay published in the New England Journal of Medicine that for certain conditions, companies could rely on appropriately designed studies with small sample sizes rather than randomized trials. They will rely on biological plausibility and clinical improvements in those early patients.

"Current regulations are onerous and unnecessarily demanding," Makary and Prasad wrote. "For patients and families, there is no time to wait."

The new "plausible mechanism" pathway would allow the agency to grant marketing authorization after manufacturers demonstrate success with several consecutive patients.

Department of Health and Human Services spokesman Andrew Nixon said the FDA plans to issue joint guidance for the plausible mechanism pathway from its CBER and CDER divisions. CBER is the division that regulates biological products including cell and gene therapies, and CDER regulates most other drugs.

Companies that receive these approvals will be required to collect real-world evidence to confirm efficacy continues and to look for safety issues that might arise.

The new approach will prioritize treatments for rare diseases that are fatal or cause severe childhood disability. Common diseases with unmet medical needs may also qualify.

While makers of cell and gene therapies are likely to be significant beneficiaries of the new approval process, Makary and Prasad said that other types of treatments could also receive licensure this way.

"The FDA will work as a partner and guide in ushering these therapies to market," the officials wrote.

(Reporting by Michael ErmanEditing by Bill Berkrot)

---

Serious News for Serious Traders! Try StreetInsider.com Premium Free!

---

You May Also Be Interested In

• US Commodity Futures Trading Commission fines George Santos for alleged manipulative trading 
• Serbia secures US sanctions waiver for its Russian-owned NIS oil firm,  energy minister says
• Oil spill from shadow fleet tanker spreading off Oman, satellite images show