Peter J Neumann1, David D Kim1, Thomas A Trikalinos2, Mark J Sculpher3, Joshua A Salomon4, Lisa A Prosser5, Douglas K Owens6, David O Meltzer7, Karen M Kuntz8, Murray Krahn9, David Feeny10, Anirban Basu11, Louise B Russell12, Joanna E Siegel13, Theodore G Ganiats14, Gillian D Sanders15. 1. Center for the Evaluation of Value and Risk in Health (CEVR), Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA. 2. Department of Health Services, Policy & Practice, Brown University School of Public Health, Brown University, Providence, RI. 3. Centre for Health Economics, University of York, York, UK. 4. Center for Primary Care and Outcomes Research/Center for Health Policy, Stanford University, Stanford, CA. 5. Child Health Evaluation and Research Unit, Department of Pediatrics and Communicable Diseases, University of Michigan Medical School, and Department of Health Management and Policy, University of Michigan School of Public Health, Ann Arbor, MI. 6. VA Palo Alto Health Care System, Palo Alto, CA, and Center for Primary Care and Outcomes Research/Center for Health Policy, Stanford University, Stanford, CA. 7. Departments of Medicine and Economics, Harris School of Public Policy Studies, and Center for Health and the Social Sciences, University of Chicago, Chicago, IL. 8. Division of Health Policy and Management, School of Public Health, University of Minnesota, Minneapolis, MN. 9. Toronto Health Economics and Technology Assessment (THETA) Collaborative, Toronto General Hospital Research Institute (TGRI), University of Toronto, Toronto, Ontario, Canada. 10. Department of Economics, McMaster University, Hamilton, Ontario, Canada. 11. The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, Department of Pharmacy, Department of Health Services and Economics, University of Washington, Seattle, WA. 12. Department of Medical Ethics and Health Policy/Perelman School of Medicine, Center for Health Incentives and Behavioral Economics, and Leonard Davis Institute of Health Economics, University of Pennsylvania, Philadelphia, PA. 13. Patient-Centered Outcomes Research Institute, Washington, DC. 14. Department of Family Medicine and Public Health, University of California San Diego, La Jolla, CA. 15. Duke Clinical Research Institute, Duke University, Durham, NC.
Abstract
OBJECTIVES: In 2016, the Second Panel on Cost-effectiveness in Health and Medicine updated the seminal work of the original panel from 2 decades earlier. The Second Panel had an opportunity to reflect on the evolution of cost-effectiveness analysis (CEA) and to provide guidance for the next generation of practitioners and consumers. In this article, we present key topics for future research and policy. METHODS: During the course of its deliberations, the Second Panel discussed numerous topics for advancing methods and for improving the use of CEA in decision making. We identify and consider 7 areas for which the panel believes that future research would be particularly fruitful. In each of these areas, we highlight outstanding research needs. The list is not intended as an exhaustive inventory but rather a set of key items that surfaced repeatedly in the panel's discussions. In the online Appendix , we also list and expound briefly on 8 other important topics. RESULTS: We highlight 7 key areas: CEA and perspectives (determining, valuing, and summarizing elements for the analysis), modeling (comparative modeling and model transparency), health outcomes (valuing temporary health and path states, as well as health effects on caregivers), costing (a cost catalogue, valuing household production, and productivity effects), evidence synthesis (developing theory on learning across studies and combining data from clinical trials and observational studies), estimating and using cost-effectiveness thresholds (empirically representing 2 broad concepts: opportunity costs and public willingness to pay), and reporting and communicating CEAs (written protocols and a quality scoring system). CONCLUSIONS: Cost-effectiveness analysis remains a flourishing and evolving field with many opportunities for research. More work is needed on many fronts to understand how best to incorporate CEA into policy and practice.
OBJECTIVES: In 2016, the Second Panel on Cost-effectiveness in Health and Medicine updated the seminal work of the original panel from 2 decades earlier. The Second Panel had an opportunity to reflect on the evolution of cost-effectiveness analysis (CEA) and to provide guidance for the next generation of practitioners and consumers. In this article, we present key topics for future research and policy. METHODS: During the course of its deliberations, the Second Panel discussed numerous topics for advancing methods and for improving the use of CEA in decision making. We identify and consider 7 areas for which the panel believes that future research would be particularly fruitful. In each of these areas, we highlight outstanding research needs. The list is not intended as an exhaustive inventory but rather a set of key items that surfaced repeatedly in the panel's discussions. In the online Appendix , we also list and expound briefly on 8 other important topics. RESULTS: We highlight 7 key areas: CEA and perspectives (determining, valuing, and summarizing elements for the analysis), modeling (comparative modeling and model transparency), health outcomes (valuing temporary health and path states, as well as health effects on caregivers), costing (a cost catalogue, valuing household production, and productivity effects), evidence synthesis (developing theory on learning across studies and combining data from clinical trials and observational studies), estimating and using cost-effectiveness thresholds (empirically representing 2 broad concepts: opportunity costs and public willingness to pay), and reporting and communicating CEAs (written protocols and a quality scoring system). CONCLUSIONS: Cost-effectiveness analysis remains a flourishing and evolving field with many opportunities for research. More work is needed on many fronts to understand how best to incorporate CEA into policy and practice.
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