
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.
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)
LATEST POSTS
- 1
Insane Realities That Will Make You Reconsider How you might interpret History - 2
New science points to 4 distinct types of autism - 3
What did the gov’t approve for Israel’s 2026 state budget? - 4
Sanofi to acquire hepatitis B vaccine maker Dynavax for $2.2 billion - 5
Grasping Various Kinds of Local misdemeanors
Vote In favor of Your Favored Web-based Venture Stage
Instructions to Discuss Successfully with Your Auto Collision Lawyer
Baidu robotaxi outage in Wuhan caused by 'system failure', police say
the 6 Shrewd Beds for seniors: A Complete Survey
Japanese H3 rocket fails during launch of navigation satellite (video)
New portrait of the oldest-known supernova | Space photo of the day for March 27, 2026
Deadly attack on kindergarten reported in Sudan
Israel says it will keep control over part of southern Lebanon after war with Hezbollah ends
A coup too far: Why Benin's rebel soldiers failed where others in the region succeeded













