Extrapolating evidence for molecularly targeted therapies from common to rare cancers: a scoping review of methodological guidance.

ABSTRACT:

Objectives

Cancer is increasingly classified according to biomarkers that drive tumour growth and therapies developed to target them. In rare biomarker-defined cancers, randomised controlled trials to adequately assess targeted therapies may be infeasible. Extrapolating existing evidence of targeted therapy from common cancers to rare cancers sharing the same biomarker may reduce evidence requirements for regulatory approval in rare cancers. It is unclear whether guidelines exist for extrapolation. We sought to identify methodological guidance for extrapolating evidence from targeted therapies used for common cancers to rare biomarker-defined cancers.

Design

Scoping review.

Data sources

Websites of health technology assessment agencies, regulatory bodies, research gro

SUBMITTER: Cho D 

PROVIDER: S-EPMC9274540 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature