Adrian Towse, MA, MPhil

In its 2018 report, the ISPOR Special Task Force (STF) on U.S. Value Assessment Frameworks recommended using the quality-adjusted life year (QALY) as a starting point for health technology assessment of new medicines, and also recommended further work on the best way to incorporate it by exploring the use of “augmented cost-effectiveness analysis” (ACEA) and “multi-criteria decision analysis” (MCDA). In our proposal, we laid out a research agenda of nine potential topics to support further development of ACEA. Based on input from the reviewers and colleagues, we selected three topics for analysis. Our first policy analysis paper is titled: “Aligning Value Assessment Frameworks for Medicines across Decision Contexts: The Case for Using the Quality-Adjusted Life Year (QALY) as the Core Element.” The STF emphasized the importance of “decision context” in thinking about how the health system assesses value. We discuss four decision contexts that can be viewed as a cascade: regulatory approval, inclusion in the health plan benefit package, management of health benefits and utilization, and finally, clinical shared decision making. We argue that for the efficient use of new medicines at a system level, these decisions should be aligned in a manner that promotes their efficient interaction, and that the metric of the QALY can provide the backbone that connects and aligns these contexts. Our second paper is titled: “Reconciling ACEA and MCDA: Is There a Way Forward for Measuring Cost-Effectiveness in the U.S. Healthcare Setting?” We explore the idea of a “QALY-anchored” MCDA and compared with a form of ACEA where elements of value are either classified as health or consumption attributes—and ultimately monetized. We argue that trade-offs derived from an ACEA and a QALY-anchored MCDA model are similarly affected by budget changes; and they are aligned and would result in similar decisions. Our third paper is titled: “Applying ACEA to Innovative Medicines for Rare and Ultra-Rare Health-Catastrophic Conditions.” Here we argue that due to greater uncertainty and the potential for financial and health catastrophe, there would be greater insurance value in this situation. This would imply the need to add additional value beyond the QALY—in effect, applying a higher societal willingness-to-pay threshold.

Challenge Award Paper: A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine

Dr. Towse and Lou Garrison, OHE Senior Visiting Fellow and Emeritus Professor at the University of Washington School of Pharmacy, are joint recipients of the first prize of the 2018 Value Assessment Challenge Awards designed to encourage innovative approaches in defining and measuring value in health care. In their winning paper, Drs. Garrison and Towse call for a broadening of the concepts of value in personalized/ precision medicine, laying out six basic policy principles as pathways to help determine value. These range from the need for flexible, value-based pricing to real-world evidence generation in personalized/precision medicine and the challenging implications for assessing and rewarding value. Their proposed strategy weaves together various threads of our joint research over the past ten years—much of it in collaboration with others in their field. They recognize the new challenges for health technology assessment in precision medicine and call for a greater focus on ensuring genuine value creation is rewarded appropriately to support optimal rates of long-term innovation. There is much work to be done to implement this strategy, and they look forward to working together and with other colleagues to move this approach forward.