Summary: Researchers assessed various factors in this study, including trends over the past two decades in incremental quality-adjusted life years (QALYs) and incremental cost per incremental QALY (cost/QALY) for newly approved anti-cancer innovations, how innovations for hematologic cancers were different from those for other cancers in terms of cost/QALY, and how chimeric antigen receptor t-cell (CAR-T) therapy compares with the recent history of innovations for both hematologic and non-hematologic cancer. They concluded that CAR-T therapy improved health outcomes measured in QALYs to a greater extent than both treatments for non-hematologic cancers and non-CAR-T treatments for hematologic cancers, with no significant differences in cost/QALY. Researchers also noted that those improvements represented a break from a trend previously reported in the literature of negligible incremental effectiveness and rising costs per life-year gained.
- Researchers dentified all analyses of pharmaceutical treatments for cancer published since 2007 in the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, a comprehensive database focusing on a subset of CEAs called cost-utility analyses (CUAs), which quantify health benefits in terms of QALYs.
- CUA results on CAR-T therapies came from an analysis conducted by the Institute for Clinical and Economic Review (2018), and were limited to those conducted in the US context with at least one of the following measures of value: incremental QALYs or cost/QALY, and with intervention/indication approval year 1995 or later.
- Researchers measured linear trend in the value measures as a function of approval year and compared interventions for non-hematologic cancers with non-CAR-T interventions for hematologic cancers and, in turn, with CAR-T therapy.
- They performed regression analysis to control for the potential influence of characteristics of a CUA including discount rate chosen, time horizon, number of years between publication and approval of the intervention for the indication in question, and an indicator for rare diseases as defined by the Genetic and Rare Diseases Information Center of the NIH.
- Finally, they included indicators for CAR-T therapy and for non-CAR-T treatments for hematologic cancers were included to test for differences from innovations for other cancer types.
- Researchers included 103 incremental QALY and 108 cost/QALY measures.
- Upon regression analysis, they found that for innovations as a whole incremental QALYs gained have declined with approval year by 0.079 per year (95% CI: -0.128 to -0.030).
- They also found that over time, cost effectiveness has worsened somewhat, but not significantly so (cost/QALY increase of $36,147 per year; 95% CI: -$29,575 to $101,868).
- In addition, they observed CAR-T to be substantially more effective than both pharmaceutical cancer innovations outside of hematology (5.17 more incremental QALYs; 95% CI: 4.09 to 6.25) and other innovations within hematology (4.67 more incremental QALYs; 95% CI: 3.44 to 5.90).
- Innovations in hematology other than CAR-T are somewhat more effective than innovations outside of hematology (0.5 more incremental QALYs; 95% CI: -0.09 to 1.09), although the difference is not statistically significant.
- Finally, they found no statistically significant differences in cost/QALY between CAR-T and other pharmaceutical interventions.