Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group

Eric Faulkner*, Anke Peggy Holtorf, Surrey Walton, Christine Y. Liu, Hwee Lin, Eman Biltaj, Diana Brixner, Charles Barr, Jennifer Oberg, Gurmit Shandhu, Uwe Siebert, Susan R. Snyder, Simran Tiwana, John Watkins, Maarten J. IJzerman, Katherine Payne

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Precision medicine is a dynamic area embracing a diverse and increasing type of approaches that allow the targeting of new medicines, screening programs or preventive healthcare strategies, which include the use of biologic markers or complex tests driven by algorithms also potentially taking account of patient preferences. The International Society for Pharmacoeconomics and Outcome Research expanded its current work around precision medicine to (1) describe the evolving paradigm of precision medicine with examples of current and evolving applications, (2) describe key stakeholders perspectives on the value of precision medicine in their respective domains, and (3) define the core factors that should be considered in a value assessment framework for precision medicine. With the ultimate goal of improving health of well-defined patient groups, precision medicine will affect all stakeholders in the healthcare system at multiple levels spanning the individual perspective to the societal perspective. For an efficient, timely and practical precision medicine value assessment framework, it will be important to address these multiple perspectives through building consensus among the stakeholders for robust procedures and measures of value aspects, including performance of precision mechanism; aligned reimbursement processes of precision mechanism and subsequent treatment; transparent expectations for evidence requirements and study designs adequately matched to the intended use of the precision mechanism and to the smaller target patient populations; recognizing the potential range of value-generation such as ruling-in and ruling-out decisions.

Original languageEnglish
Pages (from-to)529-539
Number of pages11
JournalValue in health
Volume23
Issue number5
DOIs
Publication statusPublished - May 2020
Externally publishedYes

Keywords

  • UT-Hybrid-D
  • Precision medicine and diagnostics
  • Value frameworks
  • Health technology assessment (HTA)

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