- 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
Let them eat (Taylor Swift) cake: The baker turning A-listers into life-size desserts - 2
King Charles shares cancer treatment update, says it's a 'personal blessing' - 3
Early diagnosis leads King Charles to scale back cancer treatment in the new year - 4
Mont Blanc road tunnel reopens to traffic after 15 weeks of repairs - 5
Bavarian leader questions Germany's Eurovision participation
Addressing sleep apnea early might decrease chances of developing Parkinson's disease
Step by step instructions to Safeguard Your Teeth During Sports Exercises
A Manual for Pick Viable Psychological well-being Backing Administrations In 2024
Instructions to Figure out the Various Phases of Cellular breakdown in the lungs
Kids get diseases like lupus, too. As researchers hunt better treatments, this camp brings joy
Health officials report 14 Legionnaires' disease cases in Florida, gym connection suspected
Guaranteeing Quality Medical care with Federal medical care Benefit Plans.
Vaccine committee votes to scrap universal hepatitis B shots for newborns despite outcry from children’s health experts
Your guide to how to safely thaw and cook your Thanksgiving turkey this year, according to experts













