Criteria for assessing evidence for biomarker-targeted therapies in rare cancers-an extrapolation framework.

ABSTRACT:

Background

Advances in targeted therapy development and tumor sequencing technology are reclassifying cancers into smaller biomarker-defined diseases. Randomized controlled trials (RCTs) are often impractical in rare diseases, leading to calls for single-arm studies to be sufficient to inform clinical practice based on a strong biological rationale. However, without RCTs, favorable outcomes are often attributed to therapy but may be due to a more indolent disease course or other biases. When the clinical benefit of targeted therapy in a common cancer is established in RCTs, this benefit may extend to rarer cancers sharing the same biomarker. However, careful consideration of the appropriateness of extending the existing trial evidence beyond specific cancer types is required. A fra

SUBMITTER: Cho D 

PROVIDER: S-EPMC11369883 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature