Abstract Understanding treatment heterogeneity is essential to the development of precision medicine, which seeks to tailor medical treatments to subgroups of patients with similar characteristics. One of the challenges of achieving this goal is that we usually do not have a priori knowledge of the grouping information of patients with respect to treatment effect. To address this problem, we consider a heterogeneous regression model which allows the coefficients for treatment variables to be subject-dependent with unknown grouping information. We develop a concave fusion penalized method for estimating the grouping structure and the subgroup-specific treatment effects, and derive an alternating direction method of multipliers algorithm for its implementation. We also study the theoretical properties of the proposed method and show that under suitable conditions there exists a local minimizer that equals the oracle least squares estimator based on a priori knowledge of the true grouping information with high probability. This provides theoretical support for making statistical inference about the subgroup-specific treatment effects using the proposed method. The proposed method is illustrated in simulation studies and illustrated with real data from an AIDS Clinical Trials Group Study.
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Data Integration by Combining Big Data and Survey Sample Data for Finite Population Inference
The statistical challenges in using big data for making valid statistical inference in the finite population have been well documented in literature. These challenges are due primarily to statistical bias arising from under‐coverage in the big data source to represent the population of interest and measurement errors in the variables available in the data set. By stratifying the population into a big data stratum and a missing data stratum, we can estimate the missing data stratum by using a fully responding probability sample and hence the population as a whole by using a data integration estimator. By expressing the data integration estimator as a regression estimator, we can handle measurement errors in the variables in big data and also in the probability sample. We also propose a fully nonparametric classification method for identifying the overlapping units and develop a bias‐corrected data integration estimator under misclassification errors. Finally, we develop a two‐step regression data integration estimator to deal with measurement errors in the probability sample. An advantage of the approach advocated in this paper is that we do not have to make unrealistic missing‐at‐random assumptions for the methods to work. The proposed method is applied to the real data example using 2015–2016 Australian Agricultural Census data.
more » « less- Award ID(s):
- 1733572
- NSF-PAR ID:
- 10449561
- Publisher / Repository:
- Wiley-Blackwell
- Date Published:
- Journal Name:
- International Statistical Review
- Volume:
- 89
- Issue:
- 2
- ISSN:
- 0306-7734
- Page Range / eLocation ID:
- p. 382-401
- Format(s):
- Medium: X
- Sponsoring Org:
- National Science Foundation
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