Assessing Treatment Effect Heterogeneity in Clinical Trials with Blocked Binary Outcomes
This paper addresses treatment effect heterogeneity (also referred to, more compactly, as treatment heterogeneity) in the context of a controlled clinical trial with binary endpoints. Treatment heterogeneity, variation in the true (causal) individual treatment effects, is explored using the concept of the potential outcome. This framework supposes the existance of latent responses for each subject corresponding to each possible treatment. In the context of a binary endpoint, treatment heterogeniety may be represented by the parameter, 2, the probability that an individual would have a failure on the experimental treatment, if received, and would have a success on control, if received. Previous research derived bounds for 2 based on matched pairs data. The present research extends this method to the blocked data context. Estimates (and their variances) and confidence intervals for the bounds are derived. We apply the new method to data from a renal disease clinical trial. In this example, bounds based on the blocked data are narrower than the corresponding bounds based only on the marginal success proportions. Some remaining challenges (including the possibility of further reducing bound widths) are discussed. (© 2005 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim)
J. M. Albert et al., "Assessing Treatment Effect Heterogeneity in Clinical Trials with Blocked Binary Outcomes," Biometrical Journal, John Wiley & Sons, Jan 2005.
The definitive version is available at https://doi.org/10.1002/bimj.200510157
Mathematics and Statistics
Keywords and Phrases
Bounds; Causal Effects; Randomized Block Design; Subject-Treatment Interaction; Counterfactuals Logic
Article - Journal
© 2005 John Wiley & Sons, All rights reserved.