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US FDA External Controls Guidance Does Little To Advance The Cause, Especially In Rare Diseases – Comments

 
• By Sue Sutter

Industry and patient groups take issue with draft guidance’s skeptical tone on utility and reliability of externally controlled studies, with some saying the guidance ignores special considerations inherent in studying rare diseases. Recommendations on access to patient-level data for external control arms also are problematic and likely infeasible, stakeholders say.

Road obstruction
Stakeholders would like the FDA to take a less obstructionist approach to use of externally controlled trials. • Source: Shutterstock

The US Food and Drug Administration recent draft guidance on externally controlled trials does little to advance the use of such approaches, ignores challenges inherent with studying rare disease therapies, and imposes potentially unrealistic expectations for data access, stakeholders say.

In comments to the docket, biopharma manufacturers, data companies and patient organizations find much to fault with the February draft guidance and ask the agency to provide more actual examples or case studies where the use of

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More from Real-World Evidence

EU Health Data Space May Speed Up R&D Through Access To Multi-Omics & Clinical Record Data

 
• By Eliza Slawther

The European Health Data Space framework will allow companies to accelerate R&D processes and identify new molecular targets faster by facilitating centralized access to certain types of high-quality data, Finland’s Orion Pharma says.

Why Successful Companies Are ‘Tough On Themselves’ When It Comes To HTA

 
• By Eliza Slawther

Pharmaceutical companies need to “pressure test” their clinical development strategies early for health technology assessment purposes, particularly in light of the new EU HTA Regulation, a life sciences consultant says.

Early Randomization Among Advice On How To Meet Both EMA & HTA Needs

 
• By Neena Brizmohun

Newly published insights from a series of European Medicines Agency workshops can guide drug developers in designing development plans that meet the needs of both regulators and health technology assessment bodies.

German, Dutch And Italian HTA Processes Not Supportive Of RWD, Say Companies

 
• By Eliza Slawther

Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.

More from Clinical Trials

What The EMA Can Teach HTA Bodies About Joint Clinical Assessments

 
• By Francesca Bruce

EU-level joint clinical assessments conducted under the Health Technology Assessment Regulation need to be more flexible when it comes to evidence requirements, according to experts speaking at a gene and cell therapy conference.

EU ‘Can’t Expect Industry To Give Up Proprietary Insights’ Under Health Data & Clinical Trials Regulations

 
• By Eliza Slawther

Both the EU Clinical Trials Regulation and the European Health Data Space Regulation have the potential to improve harmonization and be highly valuable for industry – but the importance of protecting company data will be paramount, a life sciences consultant says.

UK Mandates Unmodified Standardized Contracts To Speed Clinical Trial Set-Up

 
• By Vibha Sharma

The policy expectation that the model clinical trial agreements are used without modification is in line requests from industry and the NHS/HSC.