US FDA Is Hesitant About Using Observational Studies In Real-World Evidence Framework

Agency plans guidance on whether observational studies can provide real-world evidence to support regulatory decisions about drug effectiveness; however, RWE framework includes critical questions about retrospective studies, and reflects agency concerns about transparency.

New home construction. build with wooden truss, post and beam framework.

The US FDA’s real-world evidence (RWE) framework takes a cautious approach to the use of observational studies to support new efficacy claims for approved drugs.

The agency intends to issue guidance about observational study designs using real-world data (RWD), including how such studies might provide...

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 Approval Standards

More from Pathways & Standards

Don’t Look Back: US FDA AdComm Offers No Flexibility For Rexulti’s Post Hoc Bid In PTSD

 

The US FDA's Psychopharmacology Advisory Committee emphasized high standards over regulatory flexibility while turning down Otsuka's brexpiprazole plus serotonin for post-traumatic stress disorder based on conflicting Phase III trials and post-hoc Phase II analyses.

‘Unprecedented Story’: Revoked EU Marketing Authorization Fails To Thwart Translarna Sales

 

PTC Therapeutics is continuing to supply its Duchenne muscular dystrophy treatment, Translarna, in some European markets, despite EU revocation of the drug’s conditional marketing authorization earlier this year.

US FDA’s Second Half 2025 User Fee Goals Swing Toward Respiratory Disease

 

The 43 novel agents with user fee goal dates in the remainder of 2025 have fewer oncologics and infectious disease drugs than the first half of the year.