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Tools for the Economic Evaluation of Precision Medicine: A Sco** Review of Frameworks for Valuing Heterogeneity-Informed Decisions

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Abstract

Background and Objective

Precision medicine highlights the importance of exploring heterogeneity in the effectiveness and costs of interventions. Our objective was to identify and compare frameworks for valuing heterogeneity-informed decisions, and consider their strengths and weaknesses for application to precision medicine.

Methods

We conducted a sco** review to identify papers that proposed an analytical framework to place a value, in terms of costs and health benefits, on using heterogeneity to inform treatment selection. The search included English-language papers indexed in MEDLINE, Embase or EconLit, and a manual review of references and citations. We compared the frameworks qualitatively considering: the purpose and setting of the analysis; the types of precision medicine interventions where the framework could be applied; and the framework’s ability to address the methodological challenges of evaluating precision medicine.

Results

Four analytical frameworks were identified: value of stratification, value of heterogeneity, expected value of individualised care and loss with respect to efficient diffusion. Each framework is suited to slightly different settings and research questions. All focus on maximising net benefit, and quantify the opportunity cost of ignoring heterogeneity by comparing individualised or stratified decisions to a means-based population-wide decision. Where the frameworks differ is in their approaches to uncertainty, and in the additional metrics they consider.

Conclusions

Identifying and utilising heterogeneity is at the core of precision medicine, and the ability to quantify the value of heterogeneity-informed decisions is critical. Using an analytical framework to value heterogeneity will help provide evidence to inform investment in precision medicine interventions, appropriately capturing the value of targeted health interventions.

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Authors and Affiliations

  1. Canadian Centre for Applied Research in Cancer Control, Cancer Control Research, BC Cancer Research Centre, 675 W. 10th Ave, Vancouver, BC, V5Z 1L3, Canada

    Reka E. Pataky, Stuart Peacock & Dean A. Regier

  2. School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada

    Reka E. Pataky, Stirling Bryan & Dean A. Regier

  3. Centre for Clinical Epidemiology and Evaluation, Vancouver Coastal Health Research Institute, Vancouver, BC, Canada

    Stirling Bryan

  4. Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, BC, Canada

    Mohsen Sadatsafavi

  5. Faculty of Health Sciences, Simon Fraser University, Burnaby, BC, Canada

    Stuart Peacock

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  4. Stuart Peacock
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Correspondence to Reka E. Pataky.

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Funding

Financial support was provided by a Frederick Banting and Charles Best Canada Graduate Scholarships Doctoral Award from the Canadian Institutes of Health Research. The Canadian Centre for Applied Research in Cancer Control is funded by the Canadian Cancer Society.

Authors’ Contributions

All authors contributed to the study conception and design. Material preparation, data collection and analysis were performed by RP and DR. The first draft of the manuscript was written by RP. All authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

Conflicts of interest/competing interests

Reka E. Pataky, Stirling Bryan, Mohsen Sadatsafavi, Stuart Peacock and Dean A. Regier have no conflicts of interest that are directly relevant to the content of this article.

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Pataky, R.E., Bryan, S., Sadatsafavi, M. et al. Tools for the Economic Evaluation of Precision Medicine: A Sco** Review of Frameworks for Valuing Heterogeneity-Informed Decisions. PharmacoEconomics 40, 931–941 (2022). https://doi.org/10.1007/s40273-022-01176-0

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