
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
Is 'Veronica Mars' about to be your new binge-watch? It's now streaming on Netflix.14.01.2026 - 2
Home Wellness Basics: Building Your Home Exercise center01.01.1 - 3
‘We are the alternative’: Anti-Hamas Gaza militia tells BBC group is receiving international support21.11.2025 - 4
What's your #1 tone06.06.2024 - 5
How C-reactive protein outpaced ‘bad’ cholesterol as leading heart disease risk marker19.12.2025
6 Fledgling Cameras for 2024: Ideal for New Photographic artists
Easy to understand Tech: Cell phones for Old in 2024
Novo Nordisk slashes prices of popular weight loss and diabetes drugs
Why do people get headaches and migraines? A child neurologist explains the science of head pain and how to treat it
Tech Devices 2023: The Most blazing Arrivals of the Year
Advocacy groups react after Mattel introduces 1st Barbie doll with autism
The Job of Attorneys: It is Important to Comprehend When Legitimate Help
The ‘Stranger Things’ finale, explained: What happens to Vecna? And why was a key character’s fate left unknown?
Sean Penn lights up, Kylie Jenner gets A-list approval and 7 other moments you didn’t see at the Golden Globes












