OBJECTIVES: The aim of this systemic scoping review was to explore the challenges associated with the economic evaluation of prognostic and predictive companion diagnostics in the delivery of personalized medicine. This study was part of the EU-funded RadioVal project.

METHODS: The review adhered to our published protocol and the PRISMA-ScR statement. We performed searches in MEDLINE and Scopus, from January 2008 to March 2023. A variety of key words and medical subject headings were categorized into three branch topics (economic evaluation, companion diagnostics, and modelling methods) separated by an “AND” operator. Screening and data extraction were conducted by two independent reviewers. A descriptive analysis of the extracted data was conducted to identify challenges most frequently found in the included studies.

RESULTS: The search resulted in 2,343 potentially eligible studies from which 109 studies were included in the final review. Most of the studies utilized static state transition cohort models or decision trees, although these may not be best suited to capture all the multi-faceted impacts of companion diagnostics. Only a few studies utilized more complex simulation methods. Reasoning for a specific modelling method was seldom provided. Sensitivity and specificity of the companion diagnostic was often considered, however only a few studies considered compliance to the test result or thresholds to act upon. Lack of randomized clinical trial data was the most prevalent challenge regarding evidence gaps. Increased uncertainty was identified in several studies, however methods for addressing uncertainty were limited.

CONCLUSIONS: The study highlights several challenges related to the economic evaluation of prognostic and predictive companion diagnostics. Most frequent challenges are related to the choice of modelling method, incorporating the clinical utility of the companion diagnostic into the model, and addressing greater uncertainty. Our future research will focus on developing guidelines on how to overcome these challenges.