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EMA Tackles Hurdles To Using Single-Arm Trial Data For Pivotal Evidence In Filings

 
• By Neena Brizmohun

The European Medicines Agency has highlighted issues for drug developers to consider when seeking to submit clinical data from SATs as the pivotal evidence in their marketing authorization applications instead of randomized controlled trial data.

An EMA paper discusses specific characteristics of single-arm trials • Source: Shutterstock

The choice of endpoints and sources of bias and potential mitigation are among the topics covered in a new reflection paper from the European Medicines Agency on using single-arm trials (SATs) to provide confirmatory evidence on the efficacy of an investigational drug.

Key Takeaways

  • The European Medicines Agency has published a reflection paper on establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorization application.

  • The paper discusses general principles, choice of endpoints, target and trial populations, targets of estimation, statistical principles, and sources of bias and potential mitigation

The paper will interest drug developers that want to submit clinical data from SATs as the pivotal evidence in their marketing authorization applications (MAAs) instead of randomized

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