Propensity score matching and stratification using multiparty data without pooling

Jixian Wang1, Roland Marion-Gallois1

  • 1Bristol Myers Squibb, Boudry, Switzerland.

Matching and stratification based on confounding factors or propensity scores (PS) are powerful approaches for reducing confounding bias in indirect treatment comparisons. However, implementing these approaches requires pooled individual patient data (IPD). The research presented here was motivated by an indirect comparison between a single-armed trial in acute myeloid leukemia (AML), and two external AML registries with current treatments for a control. For confidentiality reasons, IPD cannot be pooled. Common approaches to adjusting confounding bias, such as PS matching or stratification, cannot be applied as 1) a model for PS, for example, a logistic model, cannot be fitted without pooling covariate data; 2) pooling response data may be necessary for some statistical inference (e.g., estimating the SE of mean difference of matched pairs) after PS matching. We propose a set of approaches that do not require pooling IPD, using a combination of methods including a linear discriminant for matching and stratification, and secure multiparty computation for estimation of within-pair sample variance and for calculations involving multiple control sources. The approaches only need to share aggregated data offline, rather than real-time secure data transfer, as required by typical secure multiparty computation for model fitting. For survival analysis, we propose an approach using restricted mean survival time. A simulation study was conducted to evaluate this approach in several scenarios, in particular, with a mixture of continuous and binary covariates. The results confirmed the robustness and efficiency of the proposed approach. A real data example is also provided for illustration.

Related Concept Videos

Stratified Sampling Method01:16

Stratified Sampling Method

Sampling is a technique to select a portion (or subset) of the larger population and study that portion (the sample) to gain information about the population. The sampling method ensures that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
To choose a stratified sample, divide the population into groups called strata and then take a...
12.7K
Randomized Experiments01:13

Randomized Experiments

The randomization process involves assigning study participants randomly to experimental or control groups based on their probability of being equally assigned. Randomization is meant to eliminate selection bias and balance known and unknown confounding factors so that the control group is similar to the treatment group as much as possible. A computer program and a random number generator can be used to assign participants to groups in a way that minimizes bias.
Simple randomization
Simple...
7.2K
Wilcoxon Signed-Ranks Test for Matched Pairs01:09

Wilcoxon Signed-Ranks Test for Matched Pairs

The Wilcoxon signed-rank test for matched pairs evaluates the null hypothesis by combining the ranks of differences with their signs. It essentially tests whether the median of the differences in a population of matched pairs is zero. Since the test incorporates more information than the sign test, it generally yields more trustable conclusions. This test also does not require the data to follow a normal distribution, but two conditions must be met for it to be applicable: (1) the data must...
216
Friedman Two-way Analysis of Variance by Ranks01:21

Friedman Two-way Analysis of Variance by Ranks

Friedman's Two-Way Analysis of Variance by Ranks is a nonparametric test designed to identify differences across multiple test attempts when traditional assumptions of normality and equal variances do not apply. Unlike conventional ANOVA, which requires normally distributed data with equal variances, Friedman's test is ideal for ordinal or non-normally distributed data, making it particularly useful for analyzing dependent samples, such as matched subjects over time or repeated measures...
293
Strategies for Assessing and Addressing Confounding01:25

Strategies for Assessing and Addressing Confounding

Confounding is a critical issue in epidemiological studies, often leading to misleading conclusions about associations between exposures and outcomes. It occurs when the relationship between the exposure and the outcome is mixed with the effects of other factors that influence the outcome. Given that, addressing confounding is of high importance for drawing accurate inferences in research.
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
153
Cluster Sampling Method01:20

Cluster Sampling Method

Appropriate sampling methods ensure that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
To choose a cluster sample, divide the population into clusters (groups) and then randomly select some of the clusters. All the members from these clusters are in the cluster sample. For example, if you randomly sample four departments from your...
12.6K