1. Pink Sheet
  2. >>R&D
  3. >>Clinical Trials
  4. >>Real-World Evidence

RWE-Only To Support A US FDA-Approved Indication? ‘Yes, Potentially’

 
• By Ramsey Baghdadi

CDER real world evidence policy head John Concato said the FDA could approve a new labeled use based only on real-world evidence, but made clear the approach is challenging. 

real world data
Using only RWE to gain a novel product approval will be challenging, FDA officials said. • Source: Shutterstock

A novel FDA-approved indication for an existing drug can be supported by real-world evidence alone, but the sponsor still must meet the traditional evidentiary standard, Office of Medical Policy Associate Director for RWE John Concato reaffirmed.

Key Takeaways

  • The FDA expects that a novel product could be approved based only on real-world evidence.

“The short answer is, ‘yes’ potentially,” Concato said in response to a direct question about whether the FDA would approve...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Real-World Evidence

AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
• By Sue Sutter

Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.

Post-Approval, Real-Time Monitoring Will Help Approval Standards, US FDA’s Makary Says

 
• By Sue Sutter

The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.

US FDA’s Prasad: ‘We Will Always Embrace Surrogate Endpoints’

 
• By Sarah Karlin-Smith

The new CBER director, once best known in the pharma world for criticizing accelerated approval, committed to expediting cancer drugs with surrogate endpoints.

UK And US Regulators In Sync On RWD External Control Arms

 
• By Vibha Sharma

The UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.

More from Clinical Trials