
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
The most effective method to Beat Dental Tension and Guarantee Customary Exams19.10.2023 - 2
Investigating Free Cell Phones: What You Really want to Be aware01.01.1 - 3
Becoming Familiar with an Unknown dialect: My Language Learning Excursion14.07.2023 - 4
Earth’s magnetic field protects life on Earth from radiation, but it can move, and the magnetic poles can even flip07.12.2025 - 5
Surveys of Thrillers That Re-imagined the Class01.01.1
Ähnliche Artikel
Craig the beer-ambassador elephant dies aged 5403.01.2026
The most effective method to Pick the Right Material Organization: Your Definitive Aide17.10.2023
King Charles shares cancer treatment update, says it's a 'personal blessing'12.12.2025
This Flashy Old-School Design Trend From Italy Still Has A Place In Modern Kitchens14.11.2025
Displaced Palestinian families suffer as heavy rains flood Gaza tent camps14.11.2025
Addiction-stricken community struggles to keep a syringe program going after Trump's order22.12.2025
Japan prepares to restart world's biggest nuclear plant, 15 years after Fukushima21.12.2025
Kate Hudson, 46, says she doesn't need long workouts to feel good01.01.2026
The most effective method to Stay away from Normal Traps While Recruiting a Material Organization17.10.2023
National health ranking puts Georgia near bottom of list. Here's why13.01.2026














