
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
Terminal cancer diagnosis announced by JFK's granddaughter22.11.2025 - 2
Lift Your Style: Famous Hairdos for Ladies06.06.2024 - 3
Euclid space telescope sees gorgeous cosmic cloud | Space photo of the day for Nov. 18, 202518.11.2025 - 4
Living Abroad: Social Inundation and Self-improvement22.09.2023 - 5
Liste des pr\u00eats qui ne n\u00e9cessitent pas de remboursement25.09.2023
Figure out how to Amplify Your Open Record Reward
Minneapolis ICE shooting: Woman dies after federal agent opens fire on her vehicle amid immigration crackdown
7 Straightforward Moves toward Move Information from Your Old Cell phone to Your New One: A Thorough Aide
The Main 10 Natural life Protection Associations
A hunger for new experiences Narratives: Motivating Travel and Experience
What is colostrum? And should you be taking it?
Top German court to rule on claims by Wirecard shareholders
Moving Pool Highlights for 2024
Astronomers detect rare 'free floating' exoplanet 10,000 light-years from Earth












