US FDA Invites Randomized Trial Designs With Real-World Elements

Agency is eager to engage with sponsors on ‘well thought-out proposals' to include real-world data sources in randomized studies, Office of New Drugs director Peter Stein says but adds that believability and robustness of the data are the agency’s primary concern.

Welcome mat outside the front door - Image
The welcome mat is out at the FDA for randomized studies that incorporate real-world data elements. • Source: Shutterstock

The US Food and Drug Administration’s door is open to drug developers interested in integrating real-world data elements into randomized trials, but some regulatory skepticism about the reliability and utility of such data may linger behind that door.

At a recent Duke-Margolis Center for Health Policy meeting on leveraging randomized clinical trials to generate real-world evidence for regulatory...

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

More from Real-World Evidence

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

 

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 

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 

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

 

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

US FDA Cell-Gene Therapy Head Says Agency Has Revived Stalled Programs

 

CBER's Nicole Verdun wants rare disease sponsors with stalled treatments to circle back because the FDA's evolved thinking on clinical trial designs may offer another opportunity for cast away products.

Canada Cuts Red Tape With Single Ethics Review For Trials

 

A new research ethics review system for multi-site clinical trials in Canada is expected to help improve study start-up times, avoid unnecessary duplication and boost the country’s competitiveness on a global stage.

EMA Official Clarifies Benefits And Limitations Of Innovation Pathways

 

A European Medicines Agency official provided an overview and update of the support the EMA offers developers of innovative products and discussed, among other things, its Innovation Task Force, Quality Innovation Group and a scientific advice pilot for clinical trials.