An empirical investigation into concerns over quality-adjusted life-years: A review of cost-effectiveness analyses in oncology

Abstract

Background

Health care payers often use cost-effectiveness analyses (CEA) using the quality-adjusted life-year (QALY), as the measure of benefit, to inform reimbursement decisions for new therapies. The QALY combines quantity of life and health-related quality of life into a single outcome measure and enables comparisons across diseases. Critics in the United States have attempted to ban the use of CEAs using QALYs based on the argument that these analyses identify subgroups of vulnerable patient populations for whom drugs are less cost-effective, thereby limiting access.

Materials and methods

We used the Tufts CEA Registry to identify QALY-based CEAs of cancer drugs conducted in the US from 1991 to 2023. We extracted the year of publication, cancer type, incremental cost-effectiveness ratio (including component incremental costs and QALYs), whether a subgroup analysis was performed, characteristics of that subgroup analysis, and how the subgroup affected cost-effectiveness.

Results

The final cohort included 322 full-text studies; 249 (77.3 %) analyzed treatments for solid tumors and the remainder treatments for blood cancers. Pembrolizumab was the most common therapy studied across all indications (10.2 %). Overall, 31 studies (9.6 %) included some form of subgroup analysis, all of which were age-related. Eleven (35.5 %) of the CEAs with age-related subgroup analyses were conducted following a pivotal clinical trial with the same subgroups.

Conclusions

QALY-based CEAs do not often include subgroups based on age, disease severity, chronic disease, or disability. In rare cases when these analyses are conducted, they are often motivated by clinically meaningful subgroup analyses performed in trials and not by payer budgetary considerations. Therefore, these results show concern about subgroup analyses does not justify efforts to exclude payers from using QALYs in CEA.

Policy summary

Concerns over CEAs identifying subgroups in their analyses do not appear to be justified and does not warrant precluding the use of QALYs for decision-making or price negotiation for drugs.