
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
Support Your Body: A Manual for Smart dieting and Sustenance - 2
Flu cases skyrocket in US. See cases, where people got sick. - 3
Two Endangered Bengal Tiger Cubs Die Days Apart at Zoo After Contracting Virus - 4
Chemical leak in Oklahoma forces evacuations and leaves many ill - 5
Zelensky warns of imminent massive Russian attack on Ukraine
5 Advancement Developments in Biotechnology
Vial marked 'Polonium 210' sparks scare during German Easter egg hunt
NASA satellite gazes into Medusa Pool | Space photo of the day for Dec. 24, 2025
France honors the victims of the Paris attacks' night of terror 10 years on
Vote in favor of the pasta that makes good dieting pleasant!
Am I a Summer, or is this a scam? What I learned from color analysis.
Figure out How to Keep up with and Clean Your Brilliant Bed for Ideal Execution
Phonetic Associations: A Survey of \Interfacing Worldwide People group\ Language Trade Application
Audits of 6 American Busssiness Class Flights













