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Personalized Medicine in the Context of Comparative Effectiveness Research

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  • Basu Anirban

    (1959 NE Pacific St., Box 357660, Seattle 98195)

Abstract

The world of patient-centered outcomes research (PCOR) seems to bridge the previously disjointed worlds of comparative effectiveness research (CER) and personalized medicine (PM). Indeed, theoretical reasoning on how information on medical quality should inform decision making, both at the individual and the policy level, reveals that personalized information on the value of medical products is critical for improving decision making at all levels. However, challenges to generating, evaluating and translating evidence that might lead to personalization need to be critically assessed. In this paper, I discuss two different concepts of personalized medicine – passive personalization (PPM) and active personalization (APM) that are important to distinguish in order to invest efficiently in PCOR and develop objective evidence on the value of personalization that will aid in its translation. APM constitutes the process of actively seeking identifiers, which can be genotypical, phenotypical or even environmental, that can be used to differentiate between the marginal benefits of treatment across patients. In contrast, PPM involves a passive approach to personalization where, in the absence of explicit research to discover identifiers, patients and physicians “learn by doing” mostly due to the repeated use of similar products on similar patients. Benchmarking the current state of PPM sets the bar to which the expected value of any new APM agenda should be evaluated. Exploring processes that enable PPM in practice can help discover new APM agendas, such as those based on developing predictive algorithms based on clinical, phenotypical and preference data, which may be more efficient that trying to develop expensive genetic tests. It can also identify scenarios or subgroups of patients where genomic research would be most valuable since alternative prediction algorithms were difficult to develop in those settings. Two clinical scenarios are discussed where PPM was explored through novel econometric methods. Related discussions around exploring PPM processes, multi-dimensionality of outcomes, and a balanced agenda for future research on personalization follow.

Suggested Citation

  • Basu Anirban, 2013. "Personalized Medicine in the Context of Comparative Effectiveness Research," Forum for Health Economics & Policy, De Gruyter, vol. 16(2), pages 107-120, June.
    • Handle: RePEc:bpj:fhecpo:v:16:y:2013:i:2:p:107-120:n:1
    DOI: 10.1515/fhep-2013-0009
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    References listed on IDEAS

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    1. Basu, Anirban & Jena, Anupam B. & Philipson, Tomas J., 2011. "The impact of comparative effectiveness research on health and health care spending," Journal of Health Economics, Elsevier, vol. 30(4), pages 695-706, July.
    2. James J. Heckman & Edward Vytlacil, 2005. "Structural Equations, Treatment Effects, and Econometric Policy Evaluation," Econometrica, Econometric Society, vol. 73(3), pages 669-738, May.
    3. Heckman, James, 2001. "Accounting for Heterogeneity, Diversity and General Equilibrium in Evaluating Social Programmes," Economic Journal, Royal Economic Society, vol. 111(475), pages 654-699, November.
    4. Anirban Basu & James J. Heckman & Salvador Navarro-Lozano & Sergio Urzua, 2007. "Use of instrumental variables in the presence of heterogeneity and self-selection: an application to treatments of breast cancer patients," Health Economics, John Wiley & Sons, Ltd., vol. 16(11), pages 1133-1157.
    5. Anirban Basu, 2012. "Estimating Person-Centered Treatment (PeT) Effects Using Instrumental Variables," NBER Working Papers 18056, National Bureau of Economic Research, Inc.
    6. Hsiao,Cheng & Morimune,Kimio & Powell,James L. (ed.), 2001. "Nonlinear Statistical Modeling," Cambridge Books, Cambridge University Press, number 9780521662468.
    7. Anirban Basu, 2009. "Individualization at the Heart of Comparative Effectiveness Research: The Time for i-CER Has Come," Medical Decision Making, , vol. 29(6), pages 9-11, November.
    8. Anirban Basu & David Meltzer, 2007. "Value of Information on Preference Heterogeneity and Individualized Care," Medical Decision Making, , vol. 27(2), pages 112-127, March.
    9. Anirban Basu & James J. Heckman & Salvador Navarro-Lozano & Sergio Urzua, 2007. "Use of instrumental variables in the presence of heterogeneity and self-selection: An application in breast cancer patients," Health, Econometrics and Data Group (HEDG) Working Papers 07/07, HEDG, c/o Department of Economics, University of York.
    10. Basu, Anirban, 2011. "Economics of individualization in comparative effectiveness research and a basis for a patient-centered health care," Journal of Health Economics, Elsevier, vol. 30(3), pages 549-559, May.
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    Cited by:

    1. Anirban Basu & Josh J. Carlson & David L. Veenstra, 2016. "A Framework for Prioritizing Research Investments in Precision Medicine," Medical Decision Making, , vol. 36(5), pages 567-580, July.

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