- CONTACT US
- AFS
- Business
- Bussiness
- Car
- Career
- Celebrity
- Digital Products
- Education
- Entertainment
- Fashion
- Film
- Food
- Fun
- Games
- General Health
- Health
- Health Awareness
- Healthy
- Healthy Lifestyle
- History Facts
- Household Appliances
- Internet
- Investment
- Law
- Lifestyle
- Loans&Mortgages
- Luxury Life Style
- movie
- Music
- Nature
- News
- Opinion
- Pet
- Plant
- Politics
- Recommends
- Science
- Self-care
- services
- Smart Phone
- Sports
- Style
- Technology
- tire
- Travel
- US
- World

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
How to watch 'A Charlie Brown Christmas' for free this weekend - 2
Dangerously cold tonight into Monday - 3
'Hero' who wrestled gun from Bondi shooter named as Ahmed al Ahmed - 4
Germany's Deutsche Welle broadcaster declared 'undesirable' in Russia - 5
9 African migrants died in freezing temperatures near Morocco-Algeria border
PFAS in pregnant women’s drinking water puts their babies at higher risk, study finds
Vote in favor of Your #1 Home Exercise Gear: Execution and Comfort Matter
US FDA unveils new pathway to approve personalized therapies
Dental, Vision, and Hearing Inclusion in Senior Protection.
Fiber is something most people could use more of. But experts advise caution with 'fibermaxxing'
6 Methods for further developing Rest Quality
Why do people get headaches and migraines? A child neurologist explains the science of head pain and how to treat it
The most effective method to Help a Friend or family member Determined to have Cellular breakdown in the lungs
Health Rounds: Regeneron drug wipes out residual multiple myeloma cells in small trial












