- 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
Journalist reported killed in the Gaza Strip - 2
RFK Jr.’s vaccine advisers plan biggest change yet to childhood schedule - 3
UN torture cm'tee report flags Israel for allegedly mistreating journalists, detainees, ex-MAG - 4
'Inflaming tensions': Bedouin mayor slams Ben-Gvir's unauthorized visit after meeting cancellation - 5
International issues on the agenda as Frances's Macron visits China
Instructions to Floss Appropriately and Forestall Gum Sickness
Recent studies prove the ancient practice of nasal irrigation is effective at fighting the common cold
Cases of norovirus are on the rise just in time for the holiday season
Step by step instructions to Explore the Close to home Consequence of Cellular breakdown in the lungs
Insurance warning signs in doctors’ offices might discourage patients from speaking openly about their health
Get Cooking: 15 Speedy and Heavenly Recipes for Occupied Individuals
5 Wellbeing Applications Assist You With remaining Fit
Overhaul Your Rest: Tips for a Peaceful Evening
A definitive Manual for Good dieting on a Tight spending plan













