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A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments

Krista Fischer, Els Goetghebeur UGent, Bernard Vrijens and Ian R White (2011) BIOSTATISTICS. 12(2). p.247-257
abstract
We propose a structural mean modeling approach to obtain compliance-adjusted estimates for treatment effects in a randomized-controlled trial comparing 2 active treatments. The model relates an individual's observed outcome to his or her counterfactual untreated outcome through the observed receipt of active treatments. Our proposed estimation procedure exploits baseline covariates that predict compliance levels on each arm. We give a closed-form estimator which allows for differential and unexplained selectivity (i.e. noncausal compliance-outcome association due to unobserved confounding) as well as a nonparametric error distribution. In a simple linear model for a 2-arm trial, we show that the distinct causal parameters are identified unless covariate-specific expected compliance levels are proportional on both treatment arms. In the latter case, only a linear contrast between the 2 treatment effects is estimable and may well be of key interest. We demonstrate the method in a clinical trial comparing 2 antidepressants.
Please use this url to cite or link to this publication:
author
organization
year
type
journalArticle (original)
publication status
published
subject
keyword
EFFICACY, CLINICAL-TRIALS, Causal inference, Randomized-controlled trials, Structural mean models
journal title
BIOSTATISTICS
Biostatistics
volume
12
issue
2
pages
247 - 257
Web of Science type
Article
Web of Science id
000288800600005
JCR category
STATISTICS & PROBABILITY
JCR impact factor
2.145 (2011)
JCR rank
8/116 (2011)
JCR quartile
1 (2011)
ISSN
1465-4644
DOI
10.1093/biostatistics/kxq053
language
English
UGent publication?
yes
classification
A1
copyright statement
I have transferred the copyright for this publication to the publisher
id
1894089
handle
http://hdl.handle.net/1854/LU-1894089
date created
2011-08-29 14:12:46
date last changed
2011-08-31 14:50:18
@article{1894089,
  abstract     = {We propose a structural mean modeling approach to obtain compliance-adjusted estimates for treatment effects in a randomized-controlled trial comparing 2 active treatments. The model relates an individual's observed outcome to his or her counterfactual untreated outcome through the observed receipt of active treatments. Our proposed estimation procedure exploits baseline covariates that predict compliance levels on each arm. We give a closed-form estimator which allows for differential and unexplained selectivity (i.e. noncausal compliance-outcome association due to unobserved confounding) as well as a nonparametric error distribution. In a simple linear model for a 2-arm trial, we show that the distinct causal parameters are identified unless covariate-specific expected compliance levels are proportional on both treatment arms. In the latter case, only a linear contrast between the 2 treatment effects is estimable and may well be of key interest. We demonstrate the method in a clinical trial comparing 2 antidepressants.},
  author       = {Fischer, Krista and Goetghebeur, Els and Vrijens, Bernard and White, Ian R},
  issn         = {1465-4644},
  journal      = {BIOSTATISTICS},
  keyword      = {EFFICACY,CLINICAL-TRIALS,Causal inference,Randomized-controlled trials,Structural mean models},
  language     = {eng},
  number       = {2},
  pages        = {247--257},
  title        = {A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments},
  url          = {http://dx.doi.org/10.1093/biostatistics/kxq053},
  volume       = {12},
  year         = {2011},
}

Chicago
Fischer, Krista, Els Goetghebeur, Bernard Vrijens, and Ian R White. 2011. “A Structural Mean Model to Allow for Noncompliance in a Randomized Trial Comparing 2 Active Treatments.” Biostatistics 12 (2): 247–257.
APA
Fischer, K., Goetghebeur, E., Vrijens, B., & White, I. R. (2011). A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments. BIOSTATISTICS, 12(2), 247–257.
Vancouver
1.
Fischer K, Goetghebeur E, Vrijens B, White IR. A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments. BIOSTATISTICS. 2011;12(2):247–57.
MLA
Fischer, Krista, Els Goetghebeur, Bernard Vrijens, et al. “A Structural Mean Model to Allow for Noncompliance in a Randomized Trial Comparing 2 Active Treatments.” BIOSTATISTICS 12.2 (2011): 247–257. Print.