
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
The Best Portable Applications for Emotional wellness and Prosperity - 2
This Week In Space podcast: Episode 186 — Snow on the Moon? - 3
In Antarctica, photos show a remote area teeming with life amid growing risks from climate change - 4
Famous Rough terrain Vehicles for 2024 - 5
Top German court to rule on claims by Wirecard shareholders
Signature Scents: A Manual for Outstanding Fragrances
Bruno Mars tour 2026: How to get tickets for 'The Romantic Tour,' presale times, prices and more
The Specialty of Compromise: Examples from Reality
The pace of hiring just fell to the lowest since 2011, outside of the pandemic
US measles cases surpass 2,000, highest in 30 years: CDC
From Modesty to Administration: Self-improvement in Interactive abilities
A milestone for Artemis II: Astronauts enter the 'lunar sphere of influence'
The Best 10 Innovation Developments of the Year
The Best Internet based Courses for Expertise Improvement













