CURE MODELS- ACCOUNTING FOR CURED PATIENTS IN ECONOMIC EVALUATIONS
Author(s)
Othus M1, Bansal A2, Koepl L1, Wagner S3, Ramsey S1
1Fred Hutchinson Cancer Research Center, Seattle, WA, USA, 2University of Washington, Seattle, WA, USA, 3Bristol-Myers Squibb, Washington Crossing, PA, USA
OBJECTIVES: Economic evaluations of competing interventions often estimate mean overall survival (OS) as a measure of intervention effect. New treatments offer some patients the possibility of being “cured” of their disease, in that they become long-term survivors whose risk of death is the same as a disease-free person. Grouping cured and non-cured patients together and reporting one mean value for OS may provide a biased assessment of a therapy that cures a proportion of patients. In this study, we compared standard survival analysis versus an approach that accounts for the fraction of patients cured. METHODS: We used clinical trial data from advanced melanoma patients treated with ipilimumab (n=137) versus gp100 (n=136) and applied statistical methodology for mixture cure models. We used logistic regression to model the probability that a patient was cured and a Weibull regression model to estimate the excess mortality for non-cured patients. Both cured and non-cured patients were subject to background mortality not related to cancer; we calculated this using age- and gender-matched mortality data from US Social Security life tables. RESULTS: Ignoring a cured proportion, ipilimumab had an estimated mean OS that was 8 months longer than gp100. Cure model analysis showed that the proportion of cured patients drove this difference, with 20% cured on ipilimumab compared to 6% with gp100. The mean OS among non-cured patients was 5 months on ipilimumab versus 4 months on gp100. The mean OS among cured patients was 26 years on both arms. After adjusting for covariates, ipilimumab had an improved cure proportion compared to gp100 (OR=2.01, 95% CI (1.00, 4.06)), but there were no significant differences in survival among non-cured patients (HR=1.05, 95% CI (0.80, 1.38)). CONCLUSIONS: This analysis supports using cure modeling in health economic evaluation in advanced melanoma, since it may reduce bias in OS estimates.
Conference/Value in Health Info
2015-05, ISPOR 2015, Philadelphia, PA, USA
Value in Health, Vol. 18, No. 3 (May 2015)
Code
CS3
Topic
Clinical Outcomes, Economic Evaluation, Methodological & Statistical Research
Topic Subcategory
Clinical Outcomes Assessment, Confounding, Selection Bias Correction, Causal Inference, Cost/Cost of Illness/Resource Use Studies, Modeling and simulation
Disease
Oncology