TY - JOUR
T1 - Assessing the sensitivity of methods for estimating principal causal effects
AU - Stuart, Elizabeth A.
AU - Jo, Booil
N1 - Funding Information:
This study was supported by Award Number K25MH083846 from the National Institute of Mental Health (NIMH; PI: Stuart) as well as by Awards MH066247, MH066319 and MH086043 from NIMH (PI: Ialongo). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institute of Mental Health or the National Institutes of Health. We thank participants of the Prevention Science Methodology Group for useful feedback.
Publisher Copyright:
© SAGE Publications.
PY - 2015/12/1
Y1 - 2015/12/1
N2 - The framework of principal stratification provides a way to think about treatment effects conditional on post-randomization variables, such as level of compliance. In particular, the complier average causal effect (CACE) - the effect of the treatment for those individuals who would comply with their treatment assignment under either treatment condition - is often of substantive interest. However, estimation of the CACE is not always straightforward, with a variety of estimation procedures and underlying assumptions, but little advice to help researchers select between methods. In this article, we discuss and examine two methods that rely on very different assumptions to estimate the CACE: a maximum likelihood ('joint') method that assumes the 'exclusion restriction,' (ER) and a propensity score-based method that relies on 'principal ignorability.' We detail the assumptions underlying each approach, and assess each methods' sensitivity to both its own assumptions and those of the other method using both simulated data and a motivating example. We find that the ER-based joint approach appears somewhat less sensitive to its assumptions, and that the performance of both methods is significantly improved when there are strong predictors of compliance. Interestingly, we also find that each method performs particularly well when the assumptions of the other approach are violated. These results highlight the importance of carefully selecting an estimation procedure whose assumptions are likely to be satisfied in practice and of having strong predictors of principal stratum membership.
AB - The framework of principal stratification provides a way to think about treatment effects conditional on post-randomization variables, such as level of compliance. In particular, the complier average causal effect (CACE) - the effect of the treatment for those individuals who would comply with their treatment assignment under either treatment condition - is often of substantive interest. However, estimation of the CACE is not always straightforward, with a variety of estimation procedures and underlying assumptions, but little advice to help researchers select between methods. In this article, we discuss and examine two methods that rely on very different assumptions to estimate the CACE: a maximum likelihood ('joint') method that assumes the 'exclusion restriction,' (ER) and a propensity score-based method that relies on 'principal ignorability.' We detail the assumptions underlying each approach, and assess each methods' sensitivity to both its own assumptions and those of the other method using both simulated data and a motivating example. We find that the ER-based joint approach appears somewhat less sensitive to its assumptions, and that the performance of both methods is significantly improved when there are strong predictors of compliance. Interestingly, we also find that each method performs particularly well when the assumptions of the other approach are violated. These results highlight the importance of carefully selecting an estimation procedure whose assumptions are likely to be satisfied in practice and of having strong predictors of principal stratum membership.
KW - complier average causal effect
KW - intermediate outcomes
KW - non-compliance
KW - principal stratification
KW - propensity scores
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U2 - 10.1177/0962280211421840
DO - 10.1177/0962280211421840
M3 - Article
C2 - 21971481
AN - SCOPUS:84948395048
SN - 0962-2802
VL - 24
SP - 657
EP - 674
JO - Statistical Methods in Medical Research
JF - Statistical Methods in Medical Research
IS - 6
ER -