David van Klaveren1,2, John B Wong2,3, David M Kent2, Ewout W Steyerberg1. 1. Department of Public Health, Erasmus University Medical Center, Rotterdam, The Netherlands (DVK, EWS). 2. Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA (DVK, JBW, DMK). 3. Division of Clinical Decision Making, Tufts Medical Center, Boston, MA, USA (JBW).
Abstract
BACKGROUND: The benefits and costs of a treatment are typically heterogeneous across individual patients. Randomized clinical trials permit the examination of individualized treatment benefits over the trial horizon but extrapolation to lifetime horizon usually involves combining trial-based individualized estimates of short-term risk reduction with less detailed (less granular) population life tables. However, the underlying assumption of equal post-trial life expectancy for low- and high-risk patients of the same sex and age is unrealistic. We aimed to study the influence of unequal granularity between models of short-term risk reduction and life expectancy on individualized estimates of cost-effectiveness of aggressive thrombolysis for patients with an acute myocardial infarction. METHODS: To estimate life years gained, we multiplied individualized estimates of short-term risk reduction either with less granular and with equally granular post-trial life expectancy estimates. Estimates of short-term risk reduction were obtained from GUSTO trial data (30,510 patients) using logistic regression analysis with treatment, sex, and age as predictor variables. Life expectancy estimates were derived from sex- and age-specific US life tables. RESULTS: Based on sex- and age-specific, short-term risk reductions but average population life expectancy (less granularity), we found that aggressive thrombolysis was cost-effective (incremental cost-effectiveness ratio below $50,000) for women above age 49 y and men above age 53 y (92% and 69% of the population, respectively). Considering sex- and age-specific short-term mortality risk reduction and correspondingly sex- and age-specific life expectancy (equal granularity), aggressive thrombolysis was cost-effective for men above age 45 y and women above age 50 y (95% and 76% of the population, respectively). CONCLUSIONS: Failure to model short-term risk reduction and life expectancy at an equal level of granularity may bias our estimates of individualized cost-effectiveness and misallocate resources.
BACKGROUND: The benefits and costs of a treatment are typically heterogeneous across individual patients. Randomized clinical trials permit the examination of individualized treatment benefits over the trial horizon but extrapolation to lifetime horizon usually involves combining trial-based individualized estimates of short-term risk reduction with less detailed (less granular) population life tables. However, the underlying assumption of equal post-trial life expectancy for low- and high-risk patients of the same sex and age is unrealistic. We aimed to study the influence of unequal granularity between models of short-term risk reduction and life expectancy on individualized estimates of cost-effectiveness of aggressive thrombolysis for patients with an acute myocardial infarction. METHODS: To estimate life years gained, we multiplied individualized estimates of short-term risk reduction either with less granular and with equally granular post-trial life expectancy estimates. Estimates of short-term risk reduction were obtained from GUSTO trial data (30,510 patients) using logistic regression analysis with treatment, sex, and age as predictor variables. Life expectancy estimates were derived from sex- and age-specific US life tables. RESULTS: Based on sex- and age-specific, short-term risk reductions but average population life expectancy (less granularity), we found that aggressive thrombolysis was cost-effective (incremental cost-effectiveness ratio below $50,000) for women above age 49 y and men above age 53 y (92% and 69% of the population, respectively). Considering sex- and age-specific short-term mortality risk reduction and correspondingly sex- and age-specific life expectancy (equal granularity), aggressive thrombolysis was cost-effective for men above age 45 y and women above age 50 y (95% and 76% of the population, respectively). CONCLUSIONS: Failure to model short-term risk reduction and life expectancy at an equal level of granularity may bias our estimates of individualized cost-effectiveness and misallocate resources.
Entities:
Keywords:
formulary decision making; outcomes research; translating research into practice
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