Adjusting for selection bias in assessing treatment effect estimates from multiple subgroups

2018 ◽  
Vol 61 (1) ◽  
pp. 216-229
Author(s):  
Ekkehard Glimm
Keyword(s):  
Selection Bias ◽  
Treatment Effect

Treatment Effect Deviation as an Alternative to Blinder–Oaxaca Decomposition for Studying Social Inequality

2019 ◽  
pp. 004912411985238 ◽  
Author(s):  
Weihua An ◽  
Adam N. Glynn
Keyword(s):  
Social Inequality ◽  
Selection Bias ◽  
Treatment Effect ◽  
Model Misspecification ◽  
Policy Preference ◽  
Omitted Variables ◽  
Oaxaca Decomposition ◽  
Explanatory Factors ◽  
First Case

The Blinder–Oaxaca decomposition (BOD) is a popular method for studying the contributions of explanatory factors to social inequality. The results have often been given causal interpretations. While recent work and this article both show that some types of BOD are equivalent to a counterfactual-based treatment effect/selection bias decomposition, this equivalence does not hold in general. Given this lack of general equivalence, in this article based on the counterfactual framework, we propose a method of treatment effect deviation (TED) to study social inequality. Essentially, the TED assesses to what extent the omission of particular covariates (i.e., selection bias in the omitted variables) can alter the estimated treatment effect. The TED has a better causal interpretation and can be estimated nonparametrically (and hence is more robust to model misspecification errors). Therefore, the TED may serve as an alternative to or may be used in tandem with the BOD. We illustrate the new method through two case studies. In the first case study, we show that the TED provides a more credible estimate of the treatment effect on the treated than does the BOD. However, both the TED and the BOD highlight the importance of accounting for prior earning in estimating the training effect. In the second case study, the results of the two methods differ notably, but both agree that normative factors play a significant role in generating the rural–urban disparity in social policy preference in China.

Impact of minimal sufficient balance, minimization, and stratified permuted blocks on bias and power in the estimation of treatment effect in sequential clinical trials with a binary endpoint

2021 ◽  
pp. 096228022110558
Author(s):  
Steven D Lauzon ◽  
Wenle Zhao ◽  
Paul J Nietert ◽  
Jody D Ciolino ◽  
Michael D Hill ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Logistic Regression ◽  
Selection Bias ◽  
Treatment Effect ◽  
Treatment Effects ◽  
Simulation Studies ◽  
Binary Outcome ◽  
Treatment Allocation ◽  
Analysis Phase ◽  
The Impact

Minimization is among the most common methods for controlling baseline covariate imbalance at the randomization phase of clinical trials. Previous studies have found that minimization does not preserve allocation randomness as well as other methods, such as minimal sufficient balance, making it more vulnerable to allocation predictability and selection bias. Additionally, minimization has been shown in simulation studies to inadequately control serious covariate imbalances when modest biased coin probabilities (≤0.65) are used. This current study extends the investigation of randomization methods to the analysis phase, comparing the impact of treatment allocation methods on power and bias in estimating treatment effects on a binary outcome using logistic regression. Power and bias in the estimation of treatment effect was found to be comparable across complete randomization, minimization, and minimal sufficient balance in unadjusted analyses. Further, minimal sufficient balance was found to have the most modest impact on power and the least bias in covariate-adjusted analyses. The minimal sufficient balance method is recommended for use in clinical trials as an alternative to minimization when covariate-adaptive subject randomization takes place.

scholarly journals Evaluating Glaucoma Treatment Effect on Intraocular Pressure Reduction Using Propensity Score Weighted Regression

2019 ◽  
Vol 9 (1) ◽  
Author(s):  
Mengfei Wu ◽  
Mengling Liu ◽  
Joel S. Schuman ◽  
Yuyan Wang ◽  
Katie A. Lucy ◽  
...  
Keyword(s):  
Intraocular Pressure ◽  
Propensity Score ◽  
Selection Bias ◽  
Treatment Effect ◽  
Laser Surgery ◽  
Observational Studies ◽  
Combination Therapies ◽  
Treatment Groups ◽  
Valid Assessment ◽  
Important Evidence

Abstract Observational studies in glaucoma patients can provide important evidence on treatment effects, especially for combination therapies which are often used in reality. But the success relies on the reduction of selection bias through methods such as propensity score (PS) weighting. The objective of this study was to assess the effects of five glaucoma treatments (medication, laser, non-laser surgery (NLS), laser + medication, and NLS + medication) on 1-year intraocular pressure (IOP) change. Data were collected from 90 glaucoma subjects who underwent a single laser, or NLS intervention, and/or took the same medication for at least 6 months, and had IOP measures before the treatment and 12-months after. Baseline IOP was significantly different across groups (p = 0.007) and this unbalance was successfully corrected by the PS weighting (p = 0.81). All groups showed statistically significant PS-weighted IOP reductions, with the largest reduction in NLS group (−6.78 mmHg). Baseline IOP significantly interacted with treatments (p = 0.03), and at high baseline IOP medication was less effective than other treatments. Our findings showed that the 1-year IOP reduction differed across treatment groups and was dependent on baseline IOP. The use of PS-weighted methods reduced treatment selection bias at baseline and allowed valid assessment of the treatment effect in an observational study.

scholarly journals Selection Bias When Using Instrumental Variable Methods to Compare Two Treatments But More Than Two Treatments Are Available

2016 ◽  
Vol 12 (1) ◽  
pp. 219-232 ◽  
Author(s):  
Ashkan Ertefaie ◽  
Dylan Small ◽  
James Flory ◽  
Sean Hennessy
Keyword(s):  
Selection Bias ◽  
Treatment Effect ◽  
Instrumental Variable ◽  
Treatment Effects ◽  
Health Improvement ◽  
Effect Estimate ◽  
Diabetic Patients ◽  
Data Set ◽  
The Health Improvement Network ◽  
Theoretical Evidence

Abstract Instrumental variable (IV) methods are widely used to adjust for the bias in estimating treatment effects caused by unmeasured confounders in observational studies. It is common that a comparison between two treatments is focused on and that only subjects receiving one of these two treatments are considered in the analysis even though more than two treatments are available. In this paper, we provide empirical and theoretical evidence that the IV methods may result in biased treatment effects if applied on a data set in which subjects are preselected based on their received treatments. We frame this as a selection bias problem and propose a procedure that identifies the treatment effect of interest as a function of a vector of sensitivity parameters. We also list assumptions under which analyzing the preselected data does not lead to a biased treatment effect estimate. The performance of the proposed method is examined using simulation studies. We applied our method on The Health Improvement Network (THIN) database to estimate the comparative effect of metformin and sulfonylureas on weight gain among diabetic patients.

Clarifying selection bias in cluster randomized trials

2021 ◽  
pp. 174077452110568
Author(s):  
Fan Li ◽  
Zizhong Tian ◽  
Jennifer Bobb ◽  
Georgia Papadogeorgou ◽  
Fan Li
Keyword(s):  
Selection Bias ◽  
Treatment Effect ◽  
Treatment Effects ◽  
Randomized Trials ◽  
Additional Data ◽  
Average Treatment Effect ◽  
Causal Effects ◽  
Cluster Randomized Trials ◽  
Average Treatment ◽  
Cluster Randomized

Background In cluster randomized trials, patients are typically recruited after clusters are randomized, and the recruiters and patients may not be blinded to the assignment. This often leads to differential recruitment and consequently systematic differences in baseline characteristics of the recruited patients between intervention and control arms, inducing post-randomization selection bias. We aim to rigorously define causal estimands in the presence of selection bias. We elucidate the conditions under which standard covariate adjustment methods can validly estimate these estimands. We further discuss the additional data and assumptions necessary for estimating causal effects when such conditions are not met. Methods Adopting the principal stratification framework in causal inference, we clarify there are two average treatment effect (ATE) estimands in cluster randomized trials: one for the overall population and one for the recruited population. We derive analytical formula of the two estimands in terms of principal-stratum-specific causal effects. Furthermore, using simulation studies, we assess the empirical performance of the multivariable regression adjustment method under different data generating processes leading to selection bias. Results When treatment effects are heterogeneous across principal strata, the average treatment effect on the overall population generally differs from the average treatment effect on the recruited population. A naïve intention-to-treat analysis of the recruited sample leads to biased estimates of both average treatment effects. In the presence of post-randomization selection and without additional data on the non-recruited subjects, the average treatment effect on the recruited population is estimable only when the treatment effects are homogeneous between principal strata, and the average treatment effect on the overall population is generally not estimable. The extent to which covariate adjustment can remove selection bias depends on the degree of effect heterogeneity across principal strata. Conclusion There is a need and opportunity to improve the analysis of cluster randomized trials that are subject to post-randomization selection bias. For studies prone to selection bias, it is important to explicitly specify the target population that the causal estimands are defined on and adopt design and estimation strategies accordingly. To draw valid inferences about treatment effects, investigators should (1) assess the possibility of heterogeneous treatment effects, and (2) consider collecting data on covariates that are predictive of the recruitment process, and on the non-recruited population from external sources such as electronic health records.

scholarly journals Resist the Temptation of Response-Adaptive Randomization

2020 ◽  
Vol 71 (11) ◽  
pp. 3002-3004 ◽  
Author(s):  
Michael Proschan ◽  
Scott Evans
Keyword(s):  
Infectious Disease ◽  
Clinical Trials ◽  
Selection Bias ◽  
Treatment Effect ◽  
Temporal Trends ◽  
Size Distributions ◽  
Adaptive Randomization ◽  
Long Duration ◽  
Treatment Effect Estimation ◽  
Effect Estimation

Abstract Response-adaptive randomization (RAR) has recently gained popularity in clinical trials. The intent is noble: minimize the number of participants randomized to inferior treatments and increase the amount of information about better treatments. Unfortunately, RAR causes many problems, including (1) bias from temporal trends, (2) inefficiency in treatment effect estimation, (3) volatility in sample-size distributions that can cause a nontrivial proportion of trials to assign more patients to an inferior arm, (4) difficulty of validly analyzing results, and (5) the potential for selection bias and other issues inherent to being unblinded to ongoing results. The problems of RAR are most acute in the very setting for which RAR has been proposed, namely long-duration “platform” trials and infectious disease settings where temporal trends are ubiquitous. Response-adaptive randomization can eliminate the benefits that randomization, the most powerful tool in clinical trials, provides. Use of RAR is discouraged.

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