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Extrapolating Evidence for Molecularly Targeted Therapies from Common to Rare Cancers: a Scoping Review of Methodological Guidance by Doah Cho; Saskia Cheyne; Sarah J Lord; John Simes; Chee Khoon Lee is a Medicine article available to read on EtoBox.

What is Extrapolating Evidence for Molecularly Targeted Therapies from Common to Rare Cancers: a Scoping Review of Methodological Guidance about?

## 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 groups, scientific societies and industry. EBM Reviews—Cochrane Methodology Register and Health Technology Assessment, Embase and MEDLINE databases (1946 to 11 May 2022). ## Eligibility criteria Papers proposing a framework or recommendations for extrapolating evidence for rare cancers, small populations and biomarker-defined cancers. ## Data extraction and synthesis We extracted framework details where available

Who reads Extrapolating Evidence for Molecularly Targeted Therapies from Common to Rare Cancers: a Scoping Review of Methodological Guidance?

Author

Doah Cho; Saskia Cheyne; Sarah J Lord; John Simes; Chee Khoon Lee

Published

2022

Language

EN

Field

Medicine (Health Sciences)