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.

  • But sponsors still will be...

“The short answer is, ‘yes’ potentially,” Concato said in response to a direct question about whether the FDA would approve an efficacy claim based solely on RWE. But he offered...

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

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.

ICH Targets RWE, Rare Diseases, Biosimilars, ATMPs In New Guideline Push

 
• By Vibha Sharma

The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.

UK MHRA’s Clinical Trial Lead On Combined Reviews, The Notification Scheme & Increasing Diversity

 
• By Eliza Slawther

In light of the UK’s MHRA announcing a major overhaul of its clinical trial legislation, the agency’s deputy director for clinical investigations and trials Andrea Manfrin tells the Pink Sheet what sponsors can expect from the new regulation.

More from Clinical Trials

Pink Sheet Podcast: US FDA’s Big Vaccine Policy Week

 
• By Derrick Gingery, Sarah Karlin-Smith, Sue Sutter, and Nielsen Hobbs

Pink Sheet reporter and editors discuss the new clinical trial requirements that the FDA announced for COVID-19 vaccines, the updated label for Novavax’s newly approved COVID-19 vaccine, and other vaccine-related events that were part of a busy week for policy in the sector.

ICH Targets RWE, Rare Diseases, Biosimilars, ATMPs In New Guideline Push

 
• By Vibha Sharma

The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.

ClinicalTrials.gov Or ISRCTN: Getting Transparency Right For UK Trials

 
• By Vibha Sharma

ISRCTN’s systems are being redesigned to facilitate compliance with upcoming requirements in the UK’s clinical trials legislation.