
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
Figure out How to Utilize Your Web based Advertising Degree to Break into the Tech Business - 2
Kids with smartphones by age 12 are at higher risk of health issues, study finds - 3
Changing Negative Cash Mentalities: Enabling Your Monetary Excursion - 4
Book excerpt: "Eat Your Ice Cream" by Ezekiel J. Emanuel, M.D. - 5
The most effective method to Go with Informed Choices on Vehicle Leases
They want better health care — so they're turning to crypto startups
Rediscovering Experience Through Excursions: Individual Travel Stories
Journey Lines for Each Explorer: Track down Your Ideal Journey
Cygnus XL brings cargo to the ISS for 1st time | Space photo of the day for Dec. 1, 2025
Why haven’t humans been back to the moon in over 50 years?
Kids get diseases like lupus, too. As researchers hunt better treatments, this camp brings joy
Change Your Skincare: 10 Inventive Magnificence Gadgets
Vote In favor of Your Favored Pet Consideration Administration
IDF strikes terror infrastructure across Iran, attack reported on Kashan airport













