- 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
- Pet
- Plant
- Politics
- Recommends
- Science
- Self-care
- services
- Smart Phone
- Sports
- Style
- Technology
- tire
- Travel
- US
- World
- エンタメ
- スポーツ
- 科学
- 経済

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
All the ways Marjorie Taylor Greene has shifted her approach lately — and why Trump is 'surprised at her' - 2
Visiting This Japanese City Just Got A Little More Expensive (Here's What Travelers Should Know) - 3
BravoCon 2025: How to watch, full schedule and lineup, where to stream free and more - 4
Full SNAP benefits must be paid ‘promptly,’ USDA tells states as government reopens - 5
What to know about Jack Dorsey's new Vine revival, DiVine
What is Fusarium graminearum, the fungus a Chinese scientist pleaded guilty to smuggling into the US?
Instructions to Plan for Your Teeth Substitution Methodology
Experience Arranging: Planning for Epic Excursions
Step by step instructions to Guarantee the Life span of Your Dental Inserts: Support and Care Guide
The most effective method to Safeguard Your Teeth from Acidic Food varieties and Beverages
The most effective method to Help a Friend or family member Determined to have Cellular breakdown in the lungs
Tracking down the Right Equilibrium: Charges versus Personal Costs in Senior Protection.
5 Wellbeing Applications Assist You With remaining Fit
Step by step instructions to Prepare with Senior Protection for Inward feeling of harmony.











