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Concepts of ‘Personalization’ in Personalized Medicine: Implications for Economic Evaluation

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Abstract

Context

This study assesses if, and how, existing methods for economic evaluation are applicable to the evaluation of personalized medicine (PM) and, if not, where extension to methods may be required.

Methods

A structured workshop was held with a predefined group of experts (n = 47), and was run using a modified nominal group technique. Workshop findings were recorded using extensive note taking, and summarized using thematic data analysis. The workshop was complemented by structured literature searches.

Results

The key finding emerging from the workshop, using an economic perspective, was that two distinct, but linked, interpretations of the concept of PM exist (personalization by ‘physiology’ or ‘preferences’). These interpretations involve specific challenges for the design and conduct of economic evaluations. Existing evaluative (extra-welfarist) frameworks were generally considered appropriate for evaluating PM. When ‘personalization’ is viewed as using physiological biomarkers, challenges include representing complex care pathways; representing spillover effects; meeting data requirements such as evidence on heterogeneity; and choosing appropriate time horizons for the value of further research in uncertainty analysis. When viewed as tailoring medicine to patient preferences, further work is needed regarding revealed preferences, e.g. treatment (non)adherence; stated preferences, e.g. risk interpretation and attitude; consideration of heterogeneity in preferences; and the appropriate framework (welfarism vs. extra-welfarism) to incorporate non-health benefits.

Conclusions

Ideally, economic evaluations should take account of both interpretations of PM and consider physiology and preferences. It is important for decision makers to be cognizant of the issues involved with the economic evaluation of PM to appropriately interpret the evidence and target future research funding.

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Acknowledgments

The workshop organization and funding, as well as the contribution by Uwe Siebert, Petra Schnell-Inderst, Beate Jahn, and Ursula Rochau, was supported by the COMET Center ONCOTYROL, which is funded by the Austrian Federal Ministries for Transport, Innovation and Technology, and for Economy, Family and Youth (via the Austrian Research Promotion Agency) and the Tiroler Zukunftsstiftung/Standortagentur Tirol (SAT).

We would like to acknowledge the helpful comments from various colleagues and, in particular, insights from members of the International ONCOTYROL Expert Task Force. Special thanks are extended to Elske van den Akker-van Marle for helpful comments during the workshop, and to Sebastian Schleidgen for helpful comments regarding definitions of PM.

The work of Wolf Rogowski in the preparation of this article was supported by the grant ‘Individualized Health Care: Ethical, Economic and Legal Implications for the German Health Care System’ of the German Federal Ministry of Education and Research (BMBF; grant number 01GP1006B).

The contribution from Andrea Manca was made under the terms of a career development research training fellowship issued by the National Institute of Health Research (NIHR; grant CDF-2009-02-21). Oguzhan Alagoz is funded by grant CMII-0844423 from the National Science Foundation, and grant UL1TR000427 from the Clinical and Translational Science Award (CTSA) program, through the NIH National Center for Advancing Translational Sciences (NCATS). All other authors have no relevant funding sources to declare.

All of the authors have programmes of work, supported by various public funding bodies, on the economics of PMs and screening programmes which include the research topics addressed here. None of the authors have any direct financial conflicts of interest with regards to this study.

The views expressed in this publication are those of the author(s) and not necessarily those of the ONCOTYROL Center for Personalized Cancer Medicine, the Helmholtz Center Munich, the UK NHS, the NIHR, or the UK Department of Health.

Author contributions

Wolf Rogowski and Katherine Payne conceived the framework underpinning this study. Wolf Rogowski lead the writing of the manuscript, with large contributions from Katherine Payne. Uwe Siebert initiated and coordinated the ONCOTYROL workshops. Petra Schnell-Inderst, Beate Jahn, and Ursula Rochau conducted literature searches and documented the workshops. Oguzhan Alagoz updated the literature searches. All authors, in particular Andrea Manca and Reiner Leidl, provided substantial intellectual input. All authors were involved in writing of the manuscript, and read and approved the final version. All authors act as guarantors individually.

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Authors and Affiliations

  1. Helmholtz Zentrum München, Institute of Health Economics and Health Care Management, Ingolstädter Landstr. 1, 85764, Neuherberg, Germany

    Wolf Rogowski & Reiner Leidl

  2. Institute and Outpatient Clinic for Occupational, Social and Environmental Medicine, Ludwig-Maximilians-Universität München, Munich, Germany

    Wolf Rogowski

  3. Manchester Centre for Health Economics, Institute of Population Health, The University of Manchester, Manchester, UK

    Katherine Payne

  4. Department of Public Health and Health Technology Assessment, Institute of Public Health, Medical Decision Making and Health Technology Assessment, UMIT—University for Health Sciences, Medical Informatics and Technology, Hall in Tirol, Austria

    Petra Schnell-Inderst, Ursula Rochau, Beate Jahn & Uwe Siebert

  5. Division of Health Technology Assessment and Bioinformatics, ONCOTYROL—Center for Personalized Cancer Medicine, Innsbruck, Austria

    Petra Schnell-Inderst, Ursula Rochau, Beate Jahn & Uwe Siebert

  6. Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA

    Uwe Siebert

  7. Department of Health Policy and Management, Center for Health Decision Science, Harvard School of Public Health, Boston, MA, USA

    Uwe Siebert

  8. Centre for Health Economics, The University of York, York, UK

    Andrea Manca

  9. Munich Center of Health Sciences, Ludwig-Maximilians-Universität München, Munich, Germany

    Reiner Leidl

  10. Department of Industrial and Systems Engineering, Department of Population Health Sciences, University of Wisconsin-Madison, Madison, WI, USA

    Oguzhan Alagoz

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  1. Wolf Rogowski
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  2. Katherine Payne
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  3. Petra Schnell-Inderst
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  8. Reiner Leidl
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  9. Uwe Siebert
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Corresponding author

Correspondence to Wolf Rogowski.

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Rogowski, W., Payne, K., Schnell-Inderst, P. et al. Concepts of ‘Personalization’ in Personalized Medicine: Implications for Economic Evaluation. PharmacoEconomics 33, 49–59 (2015). https://doi.org/10.1007/s40273-014-0211-5

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