Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine

Heleen Vellekoop (), Simone Huygens, Matthijs Versteegh, László Szilberhorn, Tamás Zelei, Balázs Nagy, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth and Maureen Rutten- van Mölken
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Heleen Vellekoop: Erasmus University Rotterdam
Simone Huygens: Erasmus University Rotterdam
Matthijs Versteegh: Erasmus University Rotterdam
László Szilberhorn: Syreon Research Institute
Tamás Zelei: Syreon Research Institute
Balázs Nagy: Syreon Research Institute
Rositsa Koleva-Kolarova: University of Oxford
Apostolos Tsiachristas: University of Oxford
Sarah Wordsworth: University of Oxford
Maureen Rutten- van Mölken: Erasmus University Rotterdam

PharmacoEconomics, 2021, vol. 39, issue 7, No 3, 788 pages

Abstract: Abstract Objective The objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM. Methods A targeted literature review of methodological papers was performed for an overview of modelling challenges in PM. Expert interviews were held to discuss best modelling practice. A systematic literature review of economic evaluations of PM was conducted to gain insight into current modelling practice. The findings were synthesised and used to develop a set of draft recommendations. The draft recommendations were discussed at a stakeholder workshop and subsequently finalised. Results Twenty-two methodological papers were identified. Some argued that the challenges in modelling PM can be addressed within existing methodological frameworks, others disagreed. Eighteen experts were interviewed. They believed large uncertainty to be a key concern. Out of 195 economic evaluations of PM identified, 56% addressed none of the identified modelling challenges. A set of 23 recommendations was developed. Eight recommendations focus on the modelling of test-treatment pathways. The use of non-randomised controlled trial data is discouraged but several recommendations are provided in case randomised controlled trial data are unavailable. The parameterisation of structural uncertainty is recommended. Other recommendations consider perspective and discounting; premature survival data; additional value elements; patient and clinician compliance; and managed entry agreements. Conclusions This study provides a comprehensive list of recommendations to modellers of PM and to evaluators and reviewers of PM models.

Date: 2021
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DOI: 10.1007/s40273-021-01010-z

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