Agree in advance how to borrow evidence between similar rare cancers
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.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.
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