
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
FBI arrests Brian Cole Jr. in Jan. 6 pipe bomb investigation, ending 5-year hunt - 2
Sustaining Public activity and Connections: Key Methodologies - 3
Golden satellite insulation sparkles during test | Space photo of the day for Dec. 30, 2025 - 4
4 Must-Visit bar-b-que Eateries This Year - 5
When preventable infections turn deadly behind bars | The Excerpt
The Response to Fake General Knowledge: Investigating the Eventual fate of artificial intelligence
Toddler given just 3 years to live after strange symptoms makes full recovery
See as Your #1: These Low-Sugar Food sources You Ought to Attempt
Brilliant and Gleaming: Excellence and Skincare Practices
Safeguarding Your Senior Protection Against Extortion and Tricks.
‘Wicked: For Good’ streaming release — How to watch the sequel starring Ariana Grande and Cynthia Erivo
Forum Dvorah demands clear support for women in combat as IDF gender debate escalates
Journeys That could only be described as epic: Delightful Voyage Lines All over the Planet
Surveys of Music Collections by Film Stars












