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Statistical methods can combine information across similar rare cancers to reach an answer with fewer patients. Regulators need to say in advance when that is acceptable.
Hierarchical Bayesian models with borrowing across histologies or across related rare diseases can substantially reduce required sample size, and are used in basket trial analyses. Sponsors avoid them because acceptance is uncertain at review. A published position on acceptable borrowing structures, prior specification and pre-registration requirements would let sponsors design smaller trials with confidence.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, Basket, umbrella, and platform trials, Regulatory divergence between regions, Rare and paediatric cancers without markets.
Shares American Society of Clinical Oncology (ASCO), Basket, umbrella, and platform trials, Rare and paediatric cancers without markets.
Shares European Society for Medical Oncology (ESMO), Hazard ratio (HR), Trial design, endpoints and cost.
Shares European Society for Medical Oncology (ESMO), Hazard ratio (HR), American Society of Clinical Oncology (ASCO), Trial design, endpoints and cost.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, European Society for Medical Oncology (ESMO), Rare and paediatric cancers without markets.
Shares Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
Shares Basket, umbrella, and platform trials, Regulatory divergence between regions, Rare and paediatric cancers without markets, Trial design, endpoints and cost.