How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?

Objectives: To review assessments from the Institute for Clinical and Economic Review (ICER) and describe how cost-effectiveness, other benefits or disadvantages, and contextual considerations affect Council members’ assessments of value. Methods: Assessments published by the ICER between December 2014 and April 2019 were reviewed. Data on the assessment, intervention, results from cost-effectiveness analyses, and Council members’ votes were extracted. Voting data were examined using bar charts and radar plots. Spearman's correlations between the number of votes for other benefits and contextual considerations were estimated. Two case studies (tisagenlecleucel and voretigene neparvovec) explored the relationship between different aspects of value and the vote. Results: Thirty-one ICER assessments were reviewed, which included 51 value votes and 17 votes on other benefits and contextual considerations. On average, interventions with lower cost-effectiveness ratios received a higher proportion of high and intermediate value votes; however, there was heterogeneity across assessments. Of other benefits or disadvantages, having a novel mechanism of action received the most votes (n = 138), and reducing health disparities received the fewest (n = 24). Of contextual considerations, treating a condition that has a severe impact on length and quality of life received the most votes (n = 164). There was a strong positive correlation between votes for reduced caregiver/family burden and improving return to work/productivity (ρ = 0.88, P < .05). Two case studies highlighted that factors beyond cost-effectiveness can lead to lower (tisagenlecleucel) or higher (voretigene neparvovec) assessments of value. Conclusion: Council members’ judgments about the value of interventions are influenced by other benefits or disadvantages and contextual considerations but anchored by cost-effectiveness.