
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
Insight: Pills, TikTok, weight-loss apps and the consumer-driven future of GLP-1s - 2
Becoming the best at Discussion: Individual Procedures - 3
South African radio presenter among five charged over Russia recruitment plot - 4
How a niche Catholic approach to infertility treatment became a new talking point for MAHA conservatives - 5
Figure out how to Guarantee Your Dental Embeds Endure forever
Rediscovering Experience Through Excursions: Individual Travel Stories
Vote in favor of the Web-based Work out schedule to Keep You Fit and Sound
Taco Bell debuts its Baja Blast pie, and the reactions may surprise you
Well known Travel Booking Locales: What's Your Pick?
How to watch the 2025 Macy's Thanksgiving Day Parade for free
Careful Connections: Building Association and Trust
How comfort foods trigger pleasure in our brains
Pick Your Favored pizza beating
Kate Hudson, 46, says she doesn't need long workouts to feel good













