WASHINGTON (dpa-AFX) - The U.S. Food and Drug Administration has issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.
The draft guidance, issued by the Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, specifically discusses genome editing and RNA-based therapies such as antisense oligonucleotides but leaves open the potential that this framework may apply to additional tailored therapeutics provided they directly address the underlying specific cause of the disease.
'President Trump promised to accelerate cures for American families - and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,' said Health and Human Services Secretary Robert F. Kennedy, Jr. 'We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.'
The draft guidance focuses on therapies that target a specific genetic, cellular or molecular abnormality and are designed to correct or modify the underlying cause of disease.
Key criteria include identifying the disease-causing abnormality, demonstrating the therapy targets the root cause or proximate biological pathway, relying on well-characterized natural history data in untreated patients, and confirming successful target drugging or editing.
For traditional approval, therapies should demonstrate improvement in clinical outcomes, disease course, or biomarkers if they are established to predict clinical benefit.
'Designing treatments unique to individual patients has always been the promised goal of personalized medicine,' said Chief Medical and Scientific Officer and Center for Biologics Evaluation and Research Director Vinay Prasad. 'After 25 years the FDA has, for the first time, outlined a framework to facilitate these approvals. The Plausible Mechanism Framework is a revolutionary advance in regulatory science.'
FDA said that it recognizes that an adequate and well-controlled clinical investigation will include a small sample size, therefore, investigation results should be sufficiently robust to exclude chance findings. When determining effectiveness, the FDA considers the specific disease, the strength of the evidence and the challenges of conducting clinical investigations for individualized therapies.
Copyright(c) 2026 RTTNews.com. All Rights Reserved
Copyright RTT News/dpa-AFX
© 2026 AFX News
