
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
Best Internet based Course for Learning Another Dialect: Which Stage Do You Like? - 2
Why are malnutrition deaths soaring in America? - 3
All that You Really want to Be aware of Dental Inserts Facilities - 4
Tesla Stock Hasn’t Looked This Cheap in a While - 5
When will the Epstein files be released — and will they reveal anything new?
Nuno Loureiro, MIT physicist, fatally shot at home; police investigate
Our favorite Space.com stories of 2025
Mont Blanc road tunnel reopens to traffic after 15 weeks of repairs
Most loved Occasion Dish: What Makes Your Merry Table?
West Bank man indicted for extortion, impersonation of IAF pilot, Mossad agent, illegal entry
A photographer finds thousands of dinosaur footprints near Italian Winter Olympic venue
Exploring the Difficulties of Co-Nurturing: Individual Bits of knowledge
Two reportedly killed as Israel attacks Hezbollah targets in Lebanon
The Specialty of Compromise: Examples from Reality












