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Partial Identification of Personalized Treatment Response with Trial-reported Analyses of Binary Subgroups

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  • Sheyu Li
  • Valentyn Litvin
  • Charles F. Manski

Abstract

Medical journals have adhered to a reporting practice that seriously limits the usefulness of published trial findings. Medical decision makers commonly observe many patient covariates and seek to use this information to personalize treatment choices. Yet standard summaries of trial findings only partition subjects into broad subgroups, typically into binary categories. Given this reporting practice, we study the problem of inference on long mean treatment outcomes E[y(t)|x], where t is a treatment, y(t) is a treatment outcome, and the covariate vector x has length K, each component being a binary variable. The available data are estimates of {E[y(t)|xk = 0], E[y(t)|xk = 1], P(xk)}, k = 1, . . . , K reported in journal articles. We show that reported trial findings partially identify {E[y(t)|x], P(x)}. Illustrative computations demonstrate that the summaries of trial findings in journal articles may imply only wide bounds on long mean outcomes. One can realistically tighten inferences if one can combine reported trial findings with credible assumptions having identifying power, such as bounded-variation assumptions.

Suggested Citation

  • Sheyu Li & Valentyn Litvin & Charles F. Manski, 2022. "Partial Identification of Personalized Treatment Response with Trial-reported Analyses of Binary Subgroups," NBER Working Papers 30461, National Bureau of Economic Research, Inc.
  • Handle: RePEc:nbr:nberwo:30461
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    References listed on IDEAS

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    1. Kenneth F Schulz & Douglas G Altman & David Moher & for the CONSORT Group, 2010. "CONSORT 2010 Statement: Updated Guidelines for Reporting Parallel Group Randomised Trials," PLOS Medicine, Public Library of Science, vol. 7(3), pages 1-7, March.
    2. Charles F. Manski & Anat R. Tambur & Michael Gmeiner, 2019. "Predicting kidney transplant outcomes with partial knowledge of HLA mismatch," Proceedings of the National Academy of Sciences, Proceedings of the National Academy of Sciences, vol. 116(41), pages 20339-20345, October.
    3. Charles F. Manski & John V. Pepper, 2018. "How Do Right-to-Carry Laws Affect Crime Rates? Coping with Ambiguity Using Bounded-Variation Assumptions," The Review of Economics and Statistics, MIT Press, vol. 100(2), pages 232-244, May.
    4. Shareen A Iqbal & Joshua D Wallach & Muin J Khoury & Sheri D Schully & John P A Ioannidis, 2016. "Reproducible Research Practices and Transparency across the Biomedical Literature," PLOS Biology, Public Library of Science, vol. 14(1), pages 1-13, January.
    5. Charles F. Manski, 2018. "Credible ecological inference for medical decisions with personalized risk assessment," Quantitative Economics, Econometric Society, vol. 9(2), pages 541-569, July.
    Full references (including those not matched with items on IDEAS)

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    JEL classification:

    • C13 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods and Methodology: General - - - Estimation: General
    • I10 - Health, Education, and Welfare - - Health - - - General

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