Nonparametric inference for time-dependent incremental cost-effectiveness ratios

As the costs of medical care increase, more studies are evaluating cost in addition to effectiveness of treatments. Cost‐effectiveness analyses in randomized clinical trials have typically been conducted only at the end of follow‐up. However, cost‐effectiveness may change over time. We therefore pro...

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Bibliographic Details
Published in:Statistics in medicine 2015-12, Vol.34 (30), p.4057-4069
Main Authors: Yee, Laura M., Gary Chan, Kwun Chuen
Format: Article
Language:English
Subjects:
Biostatistics
Cardiac arrhythmia
Computer Simulation
Confidence Intervals
Cost-Benefit Analysis - statistics & numerical data
cost-effectiveness analysis
Death, Sudden, Cardiac - prevention & control
Defibrillators, Implantable
Estimating techniques
Fieller bands
Health care expenditures
Humans
induced informative censoring
Multicenter Studies as Topic - statistics & numerical data
nonparametric estimation
randomized clinical trials
Randomized Controlled Trials as Topic - economics
Randomized Controlled Trials as Topic - statistics & numerical data
Statistical inference
Statistics, Nonparametric
Survival Analysis
Variance analysis
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Summary:As the costs of medical care increase, more studies are evaluating cost in addition to effectiveness of treatments. Cost‐effectiveness analyses in randomized clinical trials have typically been conducted only at the end of follow‐up. However, cost‐effectiveness may change over time. We therefore propose a nonparametric estimator to assess the incremental cost‐effectiveness ratio over time. We also derive the asymptotic variance of our estimator and present formulation of Fieller‐based simultaneous confidence bands. Simulation studies demonstrate the performance of our point estimators, variance estimators, and confidence bands. We also illustrate our methods using data from a randomized clinical trial, the second Multicenter Automatic Defibrillator Implantation Trial. This trial studied the effects of implantable cardioverter‐defibrillators on patients at high risk for cardiac arrhythmia. Results show that our estimator performs well in large samples, indicating promising future directions in the field of cost‐effectiveness. Copyright © 2015 John Wiley & Sons, Ltd.
ISSN:0277-6715
1097-0258
DOI:10.1002/sim.6594