TY - JOUR
T1 - Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine
AU - the HEcoPerMed Consortium
AU - Vellekoop, Heleen
AU - Huygens, Simone
AU - Versteegh, Matthijs
AU - Szilberhorn, László
AU - Zelei, Tamás
AU - Nagy, Balázs
AU - Koleva-Kolarova, Rositsa
AU - Tsiachristas, Apostolos
AU - Wordsworth, Sarah
AU - Rutten-van Mölken, Maureen
N1 - Funding Information:
The HEcoPerMed project has received funding from the European Union’s Horizon 2020 research and innovation programme under Grant Agreement no. 824997.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/4/16
Y1 - 2021/4/16
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85104961220&partnerID=8YFLogxK
U2 - 10.1007/s40273-021-01010-z
DO - 10.1007/s40273-021-01010-z
M3 - Review article
AN - SCOPUS:85104961220
SN - 1170-7690
VL - 39
SP - 771
EP - 788
JO - PharmacoEconomics
JF - PharmacoEconomics
IS - 7
ER -
