A Strategy to Support Efficient Development and Use of Innovations in Personalized Medicine and Precision Medicine

Abstract

This analysis addresses an important policy question: "What are potentially transformative strategies and methods to define and measure value at all levels of decision making that are aligned with personalized medicine/precision medicine (PM/PrM)?" To approach this question, we build on our previous work with PM and recent work as members of ISPOR's Special Task Force on Value Assessment Frameworks. We focused on 3 key challenges: (1) the need for a broader concept of value for PM/PrM; (2) the development of appropriate concepts and methods for measuring and splitting the innovation reward for PM/PrM; and (3) the application of this expanded concept to align value assessment in the hierarchy of related decision contexts, from formulary inclusion and value-based pricing to shared patient-provider decision making. Our working assumption was that the goal of policy affecting PM/PrM technologies should be to promote "dynamic efficiency"-the optimal development, adoption, and use of these technologies. For this purpose, we laid out 6 basic cross-cutting policy principles. First, rewards for innovation should be value-based and flexible over time and across indications as new evidence emerges. Second, the concept of value should be based on the microeconomic concept of willingness to pay and should include, at its core, the value of health gain-in terms of the length and quality of life- but also going beyond this to include other uncertainty-related elements. Third, innovation rewards should be modifiable given new real-world evidence and knowledge. Fourth, the scientific information embedded in PM and PrM technologies is a global public good that should be financed globally through differential pricing. Fifth, reward systems need to recognize that the diagnostic technologies that make PM and PrM possible are technically "economic complements." Sixth, splitting the rewards among complementary inputs is arbitrary in a static sense but matters dynamically. For PM/PrM value assessment, we argue for including the "value of knowing"; the need for indication-specific pricing; the importance of updating the economic evaluation and pricing over the product life cycle; addressing the new challenge posed by next-generation sequencing and high-cost combination therapies; and aligning value metrics across different decision contexts based on health benefit and patient-centered utility. DISCLOSURES: No previous outside funding supported this policy analysis. The authors have nothing to disclose. The authors are grateful to have received first place in the PhRMA Foundation-Personalized Medicine Coalition 2018 Value Assessment Challenge Award for this work.

FIGURE 1

The Elements of Value

FIGURE 2

Decision Contexts and Recent Value Frameworks: Moving from Plan Level to Patient Level