
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
Roman around the Christmas tree | Space photo of the day for Dec. 25, 2025 - 2
Share your pick for the riding area that characterizes your surf undertakings! - 3
Help Your Insusceptibility: Good dieting and Way of life Tips - 4
Ten Awesome Authentic Realities That Will Leave You Interested - 5
Starfront Observatories: A haven for distant stargazers
Geminid shooting stars: One of 2025's most exciting meteor showers begins tonight
Doctors thought he had cancer. An offhand suggestion led to a rare diagnosis.
Knesset sets special panel to fast-track Karhi’s communications reform
Mechanical Sidekick d: A Survey of \Elements and Execution d\ Cell phone
Ukrainian foreign minister appeals for funds for drones
Closets for Your Room: Plan and Utility Features
The most effective method to Explore Moral Situations in Brain research with Your Certification
Electric discovery on Mars! Scientists find tiny lightning bolts coming from Red Planet dust clouds
Criminal Guard Lawyer Expenses: What Would it be advisable for you to Hope to Pay?













