
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
Watch interstellar comet 3I/ATLAS make its closest approach to Earth in free livestream on Dec. 1817.12.2025 - 2
Kelsey Grammer on having a new baby at 70: 'You're just more available now'11.12.2025 - 3
France's Senate backs ban on social media platforms for under-15s31.03.2026 - 4
Productive CRM Programming for Client Relationship The executives06.06.2024 - 5
As Western heat wave ends, scientists try to make sense of its length and intensity28.03.2026 - 6
Exploring the School Application Cycle: Understudy Bits of knowledge22.09.2023 - 7
EU-funded BioSupPack project turns brewery waste into bioplastics30.03.2026 - 8
The Main 15 Powerful Business Heads of Today07.07.2023 - 9
At least 490 protesters killed in Iran, activists say11.01.2026 - 10
Figure out How to Track the Establishment of New 5G Pinnacles17.10.2023 - 11
Top 5 Top of the line Books of the Year11.08.2023 - 12
With Obamacare premium hikes, more people opting for no coverage or cheaper plans11.12.2025 - 13
Netflix Faces Wider Fallout After Italy Court Orders Refunds, Price Cuts06.04.2026 - 14
Doctored NXT Summit footage falsely portrays Modi as declaring war on Iran and Pakistan27.03.2026 - 15
The most effective method to Distinguish the Best Material Organization in Your Space17.10.2023
Ähnliche Artikel
Artemis 2 captures historic 'Earthset' photo | Space photo of the day for April 7, 202607.04.2026
Tesla Germany Registrations Quadruple to 9,252 Vehicles in Best March Ever07.04.2026
What's going on with Katseye? The Manon Bannerman hiatus drama, explained.07.04.2026
Two IDF officers, civilian face indictment in alleged Gaza aid-truck smuggling scheme07.04.2026
West Palm Beach Shorecrest, renderings of downtown waterfront condo07.04.2026
Artemis II astronauts make long-distance call to the space station as they head home from the moon07.04.2026
Tuesday, April 7. Russia’s War On Ukraine: News And Information From Ukraine07.04.2026
The Eastern Bongo, Kenya’s Rare Forest Antelope on the Brink07.04.2026
U.K. blocks Kanye West from entering Britain to headline now canceled festival: What led to the ban07.04.2026
Russia confirms 16 Cameroonian soldiers killed in Ukraine war07.04.2026
First Phosphate advances battery-grade phosphate project as analysts highlight strategic Federal support07.04.2026
After fleeing past Hezbollah fighting, some Israelis on northern border vow to stay07.04.2026
'Unreal' solar eclipse: Artemis 2 crew just saw one of the rarest sights in spaceflight history07.04.2026
Chinese construction workers in Israel: 'I’d rather be bombed than live in poverty'07.04.2026
Broken toilet, T-shirts on windows and collecting saliva: The weirdness of daily life aboard Orion07.04.2026
Czech Republic caps fuel prices amid Iran war energy crisis07.04.2026
NASA releases stunning new images captured by the Artemis II moon mission, including 'Earthset' and a solar eclipse from space07.04.2026
Amid Iran war, 53 of Israel's future scientists showcase projects in Jerusalem contest07.04.2026
Iranian-linked drone attack kills Kurdish couple in northern Iraq07.04.2026
Thousands of small fish defy gravity to climb Congo waterfall07.04.2026

































