The use of single-arm trials (SATs) in oncology drug development has been a topic of ongoing discussion and debate. While SATs can facilitate faster patient access to novel therapies, they also present challenges in establishing robust evidence of efficacy and safety. In 2023, the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) released eagerly anticipated draft guidance documents addressing the role of SATs in regulatory submissions.

Key takeaways

  • Both the EMA and FDA express a clear preference for randomized controlled trials (RCTs) over SATs as the gold standard for demonstrating efficacy.
  • Regulatory acceptance of SATs will be evaluated on a case-by-case basis, underscoring the importance of early scientific advice discussions with the agencies.
  • Key limitations of SATs highlighted include inability to interpret time-to-event endpoints, challenges in assessing combination regimens, reliance on cross-trial comparisons, and limited safety data.
  • Both agencies caution against over-reliance on external controls due to risks of bias and lack of comparability across trials.

Read the full white paper to ensure your oncology development strategies align with the latest regulatory thinking on SATs.

Author

Adriana Andrić

Senior Consultant

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