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. 2021 Jul;39(7):771-788.
doi: 10.1007/s40273-021-01010-z. Epub 2021 Apr 16.

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

Heleen Vellekoop  1 , Simone Huygens  2 , Matthijs Versteegh  2 , László Szilberhorn  3 , Tamás Zelei  3 , Balázs Nagy  3 , Rositsa Koleva-Kolarova  4 , Apostolos Tsiachristas  4 , Sarah Wordsworth  4 , Maureen Rutten-van Mölken  2   5 ; HEcoPerMed Consortium
Affiliations

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

Heleen Vellekoop et al. Pharmacoeconomics. 2021 Jul.

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.

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Conflict of interest statement

Heleen Vellekoop, Simone Huygens, Matthijs Versteegh, László Szilberhorn, Tamás Zelei, Balázs Nagy, Rositsa Koleva-Kolarova, Apostolos Tsiachristas, Sarah Wordsworth and Maureen Rutten-van Mölken have no conflicts of interest that are directly relevant to the content of this article.

Figures

Fig. 1
Fig. 1
Flow chart of the systematic literature review. CE cost-effectiveness, ICER Institute for Clinical and Economic Review, NICE National Institute for Health and Care Excellence, PM personalised medicine
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Box 1 Considering interdependence between test results [29]
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Box 2 Incorporating waiting times [30]
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Box 3 Accounting for excess mortality among long-term survivors [50]
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Box 4 Incorporating compliance [56]
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Box 5 Incorporating the conditions of a managed entry agreement [64]
See this image and copyright information in PMC

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