
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)
NEUESTE BEITRÄGE
- 1
Tech Patterns 2023: 12 Advancements to Keep an eye Out For10.08.2023 - 2
Woman charged in unprovoked stabbing of tourist changing baby's diaper in Macy’s Herald Square store12.12.2025 - 3
James Webb Space Telescope's mysterious 'little red dots' may be black holes in disguise14.01.2026 - 4
Israel’s mixed messaging on Christmas draws controversy25.12.2025 - 5
Pat Finn, actor from 'The Middle,' dies at 60 after bladder cancer diagnosis24.12.2025
Ähnliche Artikel
How to get tickets to Jay-Z’s sold out Yankee Stadium shows25.03.2026
Which Startup's Innovation Could Reform Medical care?01.01.1
'It's doing badly': Fears grow for whale stuck off Germany's coast29.03.2026
Turkey's Erdogan denounces Israel-Greece-Cyprus trilateral summit, affirms support for Gaza24.12.2025
Hezbollah field commander killed in IDF strikes in Beirut01.04.2026
Why haven’t humans been back to the moon in over 50 years?30.03.2026
From Lounge chair to Money: Online Positions That Will Change Your Profession06.06.2024
2025 Arctic League telethon raises more than $39k07.12.2025
Grasping the Qualifications Among Separation and Dissolution30.06.2023
Why is Jerome Powell being investigated? Making sense of the DOJ's probe into the Federal Reserve chair.12.01.2026














