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

Real-World Data: FDA Expounds On Use Of Registries, Challenges In Standardization

 
• By Brenda Sandburg

Final guidance on assessing registries notes responsibility of sponsors to ensure reliability of data from other organizations and reduce loss of participants to follow-up, while final guidance on data standards for RWD acknowledges sponsors may have limited access to a study’s data sources.

Real World
FDA issues two final guidances on use of real-world data to generate real-world evidence for drugs and biologics • Source: Shutterstock

The US Food and Drug Administration closed out the year with the issuance of two final guidances on the use of real-world data, both of which provide minor changes to draft guidances issued in the fall of 2021.

The final guidance entitled “Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products,” specifies what...

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

Single Trial Success Stories: UCB’s Kygevvi Joins US FDA Approvals With Confirmatory Evidence

 
• By Bridget Silverman

The orphan drug share of US FDA’s 2025 novel approvals is holding steady, with examples of approvals based on a single trial with confirmatory evidence from UCB, Precigen, Jazz Pharmaceuticals and Stealth Biotherapeutics

Do US FDA Complete Response Letters Suggest Turn To Less Flexibility In Rare Disease?

 
• By Bridget Silverman

A cluster of CRLs for rare disease applications based on one trial plus confirmatory evidence may represent a shift away from the regulatory flexibility that had come to characterize ultra-rare drug development

Beyond The Hype: AI Use Cases In Drug Development

 
• By Bridget Silverman

AI is moving from blue-sky potential to specific applications in drug development, streamlining pharmacovigilance and clinical trial design, but strong governance remains essential to ensure transparency and reliability.

Biohaven’s Troriluzole And The Era Of Real-Time Complete Response Letters

 
• By Michael McCaughan

US FDA’s rejection of Biohaven’s troriluzole is a huge disappointment for patients with SCA and the company. But it is also a notable example of how communication strategies are changing now that FDA is making its ‘complete response’ letters public.

More from Clinical Trials

Pharma Needs Incentives To Optimize Cancer Drugs, EMA Oncology Chair Says

 
• By Eliza Slawther

Incentives are “probably the only solution” to encouraging companies to optimize cancer drugs, but this will require funding and systemic changes, the chair of the European Medicines Agency’s oncology working party says.

EMA’s Peter Arlett Says ‘We Will Do Better’ On EU Clinical Trials

 
• By Vibha Sharma

While the upcoming EU Biotech Act presents an opportunity to simplify the regulatory landscape for clinical trials, improving the current Clinical Trials Regulation remains the immediate priority.

Single Trial Success Stories: UCB’s Kygevvi Joins US FDA Approvals With Confirmatory Evidence

 
• By Bridget Silverman

The orphan drug share of US FDA’s 2025 novel approvals is holding steady, with examples of approvals based on a single trial with confirmatory evidence from UCB, Precigen, Jazz Pharmaceuticals and Stealth Biotherapeutics