
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
PFAS in pregnant women’s drinking water puts their babies at higher risk, study finds - 2
Palestinian infant freezes to death in Gaza as Israel keeps blocking aid - 3
Must-See Attractions in Washington, D.C. - 4
Flourishing in a Cutthroat Work Market: Vocation Methodologies - 5
Top notch DSLR Cameras for Photography Devotees
AI is providing emotional support for employees – but is it a valuable tool or privacy threat?
Defense Minister Katz finally condemns Jewish extremist violence against Palestinians
Change Your Physical make-up: Compelling Activities for Muscle Building
The Best 20 Tunes that Characterized an Age
6 Fledgling Cameras for 2024: Ideal for New Photographic artists
Step by step instructions to Pick the Right Dental specialist for Your Dental Inserts Technique
Was This Driver Simply Having A great time Or Behaving Like An Ass?
Picking the Right Air Purifier for Your Home
An Extended time of Careful Nurturing: Individual Bits of knowledge on Bringing up Kids













