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October 8, 2025Statistical Methods in Medical ResearchOpen Access

Implementing response-adaptive randomisation in stratified rare-disease trials: Design challenges and practical solutions

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Authors

RDRajenki DasUniversity of CambridgeNDNina DeliuMRC Biostatistics UnitMTMark ToshnerVascular Medicine

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Overview

This paper reports design challenges of implementing response-adaptive randomisation in rare-disease trials, suggesting solutions for allocation probabilities and missing data.

Key Points

  • Response-adaptive randomisation improves allocation probabilities in small samples, enhancing trial efficiency.
  • Mapping strategy was proposed to reduce frequentist errors through adjusted randomisation probabilities.
  • Interim analyses in trials help manage missing data, crucial for maintaining trial integrity and data usage.
  • Additional design considerations include data pooling across strata and the robustness of safety results.

Cite This Study

Das et al. (2025) studied this question.

synapsesocial.com/papers/68e5c1be6950a706b22b5958https://doi.org/10.1177/09622802251380625
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