Value in Health
May 2020 Volume 23, Issue 5, p527-676
https://www.valueinhealthjournal.com/issue/S1098-3015(20)X0006-4

THEMED SECTION: PRECISION MEDICINE
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
p529–539
Published online: April 1, 2020
Highlights
:: Various stakeholder groups (eg, developers, regulators, payers, providers, patients) view the value attributes associated with precision medicine in different ways, not all of which are reflected in value assessment models.
:: Value assessment of precision medicine is inconsistent and, in some cases, unclear across different geographies and health technology assessment, bodies, irrespective of some groups articulating approaches. Currently, there is not uniform standard, which has potential to result in variable patient access to precision medicine.
:: Rapid expansion of diagnostic testing, including evolution of next-generation testing, whole genome and exome testing and integration of evidence with machine learning are also key areas that health technology assessment, processes will need to adapt around.
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.