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Special Communication
September 13, 2016

Recommendations for Conduct, Methodological Practices, and Reporting of Cost-effectiveness Analyses: Second Panel on Cost-Effectiveness in Health and Medicine

Author Affiliations
  • 1Duke Clinical Research Institute, Duke University, Durham, North Carolina
  • 2Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts
  • 3Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy, and Departments of Health Services and Economics, University of Washington, Seattle
  • 4Division of Medical Ethics, Social Medicine, Harvard Medical School, Boston, Massachusetts
  • 5Department of Economics, McMaster University, Hamilton, Ontario, Canada
  • 6Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Ontario, Canada
  • 7Toronto Health Economics and Technology Assessment Collaborative, Toronto General Research Institute, University Health Network, Toronto General Hospital, Toronto, Ontario, Canada
  • 8Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada
  • 9Division of Health Policy and Management, School of Public Health, University of Minnesota, Minneapolis
  • 10Departments of Medicine and Economics, Harris School of Public Policy Studies, and Center for Health and the Social Sciences, University of Chicago, Chicago, Illinois
  • 11VA Palo Alto Health Care System, Palo Alto, California
  • 12Center for Health Policy/Center for Primary Care and Outcomes Research, Stanford University, Stanford, California
  • 13Child Health Evaluation and Research Unit, Department of Pediatrics and Communicable Diseases, University of Michigan Medical School, Ann Arbor
  • 14Department of Health Management and Policy, University of Michigan School of Public Health, Ann Arbor
  • 15Department of Global Health and Population, Harvard T. H. Chan School of Public Health, Boston, Massachusetts
  • 16Centre for Health Economics, University of York, York, England
  • 17Department of Health Services, Policy and Practice, School of Public Health, Brown University, Providence, Rhode Island
  • 18Department of Economics and Institute for Health, Rutgers University, New Brunswick, New Jersey
  • 19Patient-Centered Outcomes Research Institute, Washington, DC
  • 20Department of Family Medicine and Community Health, Miller School of Medicine, University of Miami, Miami, Florida
JAMA. 2016;316(10):1093-1103. doi:10.1001/jama.2016.12195

Importance  Since publication of the report by the Panel on Cost-Effectiveness in Health and Medicine in 1996, researchers have advanced the methods of cost-effectiveness analysis, and policy makers have experimented with its application. The need to deliver health care efficiently and the importance of using analytic techniques to understand the clinical and economic consequences of strategies to improve health have increased in recent years.

Objective  To review the state of the field and provide recommendations to improve the quality of cost-effectiveness analyses. The intended audiences include researchers, government policy makers, public health officials, health care administrators, payers, businesses, clinicians, patients, and consumers.

Design  In 2012, the Second Panel on Cost-Effectiveness in Health and Medicine was formed and included 2 co-chairs, 13 members, and 3 additional members of a leadership group. These members were selected on the basis of their experience in the field to provide broad expertise in the design, conduct, and use of cost-effectiveness analyses. Over the next 3.5 years, the panel developed recommendations by consensus. These recommendations were then reviewed by invited external reviewers and through a public posting process.

Findings  The concept of a “reference case” and a set of standard methodological practices that all cost-effectiveness analyses should follow to improve quality and comparability are recommended. All cost-effectiveness analyses should report 2 reference case analyses: one based on a health care sector perspective and another based on a societal perspective. The use of an “impact inventory,” which is a structured table that contains consequences (both inside and outside the formal health care sector), intended to clarify the scope and boundaries of the 2 reference case analyses is also recommended. This special communication reviews these recommendations and others concerning the estimation of the consequences of interventions, the valuation of health outcomes, and the reporting of cost-effectiveness analyses.

Conclusions and Relevance  The Second Panel reviewed the current status of the field of cost-effectiveness analysis and developed a new set of recommendations. Major changes include the recommendation to perform analyses from 2 reference case perspectives and to provide an impact inventory to clarify included consequences.

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