
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
James Webb Space Telescope finds strongest evidence yet for atmosphere around rocky exoplanet: 'It's really like a wet lava ball'11.12.2025 - 2
The Solution to Individual budget: Dominating Cash The board07.07.2023 - 3
The Red Sea strategy: What does Israel stand to gain from recognizing Somaliland?27.12.2025 - 4
Manual for Individual accounting Rudiments for Fledglings01.01.1 - 5
Bruno Mars tour 2026: How to get tickets for 'The Romantic Tour,' presale times, prices and more09.01.2026 - 6
6 Vehicle Rental Administrations: Pick Your Ideal Ride06.06.2024 - 7
Gaza amputees struggle to rebuild lives as the enclave faces shortages of prosthetic limbs12.12.2025 - 8
Former Peruvian President Pedro Castillo sentenced for conspiracy27.11.2025 - 9
Climate engineering would alter the oceans, reshaping marine life – our new study examines each method’s risks14.01.2026 - 10
Vote In favor of Your Favored Kind Of Tea05.06.2024 - 11
NAFFIC and Aware to Launch First China-Europe Digital Product Passport30.03.2026 - 12
Find the Excellence of Old style Expressive dance: Encountering the Effortlessness and Polish of Dance14.07.2023 - 13
‘It’s Israeli policy’: Report reveals abuse of Palestinians in prisons19.11.2025 - 14
Songbirds swap colorful plumage genes across species lines among their evolutionary neighbors11.12.2025 - 15
From Novice to Master: Dominating a Side interest22.09.2023
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


































