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Improving trial generalizability using observational studies.

Publication ,  Journal Article
Lee, D; Yang, S; Dong, L; Wang, X; Zeng, D; Cai, J
Published in: Biometrics
June 2023

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces the covariate balance between the RCT and OS, therefore improving the trial-based estimator's generalizability. Exploiting semiparametric efficiency theory, we propose a doubly robust augmented calibration weighting estimator that achieves the efficiency bound derived under the identification assumptions. A nonparametric sieve method is provided as an alternative to the parametric approach, which enables the robust approximation of the nuisance functions and data-adaptive selection of outcome predictors for calibration. We establish asymptotic results and confirm the finite sample performances of the proposed estimators by simulation experiments and an application on the estimation of the treatment effect of adjuvant chemotherapy for early-stage non-small-cell lung patients after surgery.

Duke Scholars

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Published In

Biometrics

DOI

EISSN

1541-0420

Publication Date

June 2023

Volume

79

Issue

2

Start / End Page

1213 / 1225

Location

England

Related Subject Headings

  • Statistics & Probability
  • Models, Statistical
  • Humans
  • Computer Simulation
  • 4905 Statistics
  • 0199 Other Mathematical Sciences
  • 0104 Statistics
 

Citation

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Lee, D., Yang, S., Dong, L., Wang, X., Zeng, D., & Cai, J. (2023). Improving trial generalizability using observational studies. Biometrics, 79(2), 1213–1225. https://doi.org/10.1111/biom.13609
Lee, Dasom, Shu Yang, Lin Dong, Xiaofei Wang, Donglin Zeng, and Jianwen Cai. “Improving trial generalizability using observational studies.Biometrics 79, no. 2 (June 2023): 1213–25. https://doi.org/10.1111/biom.13609.
Lee D, Yang S, Dong L, Wang X, Zeng D, Cai J. Improving trial generalizability using observational studies. Biometrics. 2023 Jun;79(2):1213–25.
Lee, Dasom, et al. “Improving trial generalizability using observational studies.Biometrics, vol. 79, no. 2, June 2023, pp. 1213–25. Pubmed, doi:10.1111/biom.13609.
Lee D, Yang S, Dong L, Wang X, Zeng D, Cai J. Improving trial generalizability using observational studies. Biometrics. 2023 Jun;79(2):1213–1225.
Journal cover image

Published In

Biometrics

DOI

EISSN

1541-0420

Publication Date

June 2023

Volume

79

Issue

2

Start / End Page

1213 / 1225

Location

England

Related Subject Headings

  • Statistics & Probability
  • Models, Statistical
  • Humans
  • Computer Simulation
  • 4905 Statistics
  • 0199 Other Mathematical Sciences
  • 0104 Statistics