IDEAS home Printed from https://ideas.repec.org/a/bla/jorssc/v71y2022i5p1065-1084.html
   My bibliography  Save this article

A principal stratification approach to estimating the effect of continuing treatment after observing early outcomes

Author

Listed:
  • Patrick M. Schnell
  • Richard Baumgartner
  • Shahrul Mt‐Isa
  • Vladimir Svetnik

Abstract

Chronic diseases often require continuing care, and early response to treatment can be an important predictor of long‐term efficacy. Often, an apparent lack of early efficacy may lead to discontinuation of treatment, with the decision made either by clinicians or by the patients themselves. Thus, it is important to determine whether or not a desired early outcome corresponds to a beneficial long‐term effect of continuing treatment, and conversely, whether or not the absence of such an outcome corresponds to a lack of long‐term benefit. However, primary clinical trials of such treatments are not commonly designed to answer such questions, for example by randomizing subjects to continue or discontinue treatment after observing early outcomes. We propose an approach to estimating the effect of continuing treatment after observing early outcomes using data from randomized controlled trials in which treatment discontinuation was not part of the design. Our approach estimates average causal effects of continuing treatment on long‐term outcomes in principal strata defined by the potential early outcomes under treatment. For illustration, we estimate the effects of continuing to take gaboxadol to treat insomnia conditional on early improvement in subjective sleep quality after two nights, based on a standard parallel‐arm randomized controlled trial.

Suggested Citation

  • Patrick M. Schnell & Richard Baumgartner & Shahrul Mt‐Isa & Vladimir Svetnik, 2022. "A principal stratification approach to estimating the effect of continuing treatment after observing early outcomes," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 71(5), pages 1065-1084, November.
  • Handle: RePEc:bla:jorssc:v:71:y:2022:i:5:p:1065-1084
    DOI: 10.1111/rssc.12552
    as

    Download full text from publisher

    File URL: https://doi.org/10.1111/rssc.12552
    Download Restriction: no

    File URL: https://libkey.io/10.1111/rssc.12552?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    References listed on IDEAS

    as
    1. Constantine E. Frangakis & Donald B. Rubin, 2002. "Principal Stratification in Causal Inference," Biometrics, The International Biometric Society, vol. 58(1), pages 21-29, March.
    2. Victor Chernozhukov & Denis Chetverikov & Mert Demirer & Esther Duflo & Christian Hansen & Whitney Newey & James Robins, 2018. "Double/debiased machine learning for treatment and structural parameters," Econometrics Journal, Royal Economic Society, vol. 21(1), pages 1-68, February.
    3. Gustafson Paul, 2010. "Bayesian Inference for Partially Identified Models," The International Journal of Biostatistics, De Gruyter, vol. 6(2), pages 1-20, March.
    4. Peng Ding & Jiannan Lu, 2017. "Principal stratification analysis using principal scores," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 79(3), pages 757-777, June.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Zhichao Jiang & Shu Yang & Peng Ding, 2022. "Multiply robust estimation of causal effects under principal ignorability," Journal of the Royal Statistical Society Series B, Royal Statistical Society, vol. 84(4), pages 1423-1445, September.
    2. Michael R. Elliott & Anna Conlon & Yun Li, 2013. "Discussion on “Surrogate Measures and Consistent Surrogates”," Biometrics, The International Biometric Society, vol. 69(3), pages 565-569, September.
    3. Laura Forastiere & Patrizia Lattarulo & Marco Mariani & Fabrizia Mealli & Laura Razzolini, 2021. "Exploring Encouragement, Treatment, and Spillover Effects Using Principal Stratification, With Application to a Field Experiment on Teens’ Museum Attendance," Journal of Business & Economic Statistics, Taylor & Francis Journals, vol. 39(1), pages 244-258, January.
    4. Silvia Noirjean & Mario Biggeri & Laura Forastiere & Fabrizia Mealli & Maria Nannini, 2023. "Estimating causal effects of community health financing via principal stratification," Statistical Methods & Applications, Springer;Società Italiana di Statistica, vol. 32(4), pages 1317-1350, October.
    5. Rahul Singh, 2022. "Generalized Kernel Ridge Regression for Long Term Causal Inference: Treatment Effects, Dose Responses, and Counterfactual Distributions," Papers 2201.05139, arXiv.org.
    6. Gilbert Peter B. & Blette Bryan S. & Hudgens Michael G. & Shepherd Bryan E., 2020. "Post-randomization Biomarker Effect Modification Analysis in an HIV Vaccine Clinical Trial," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 54-69, January.
    7. Huber, Martin & Meier, Jonas & Wallimann, Hannes, 2022. "Business analytics meets artificial intelligence: Assessing the demand effects of discounts on Swiss train tickets," Transportation Research Part B: Methodological, Elsevier, vol. 163(C), pages 22-39.
    8. Wenlong Ji & Lihua Lei & Asher Spector, 2023. "Model-Agnostic Covariate-Assisted Inference on Partially Identified Causal Effects," Papers 2310.08115, arXiv.org.
    9. Park Soojin & Kürüm Esra, 2020. "A Two-Stage Joint Modeling Method for Causal Mediation Analysis in the Presence of Treatment Noncompliance," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 131-149, January.
    10. Simon Calmar Andersen & Louise Beuchert & Phillip Heiler & Helena Skyt Nielsen, 2023. "A Guide to Impact Evaluation under Sample Selection and Missing Data: Teacher's Aides and Adolescent Mental Health," Papers 2308.04963, arXiv.org.
    11. Vira Semenova, 2020. "Generalized Lee Bounds," Papers 2008.12720, arXiv.org, revised Feb 2023.
    12. Shanshan Luo & Wei Li & Yangbo He, 2023. "Causal inference with outcomes truncated by death in multiarm studies," Biometrics, The International Biometric Society, vol. 79(1), pages 502-513, March.
    13. Chanmin Kim & Lucas R. F. Henneman & Christine Choirat & Corwin M. Zigler, 2020. "Health effects of power plant emissions through ambient air quality," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 183(4), pages 1677-1703, October.
    14. Park Soojin & Kürüm Esra, 2020. "A Two-Stage Joint Modeling Method for Causal Mediation Analysis in the Presence of Treatment Noncompliance," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 131-149, January.
    15. Soojin Park & Gregory J. Palardy, 2020. "Sensitivity Evaluation of Methods for Estimating Complier Average Causal Mediation Effects to Assumptions," Journal of Educational and Behavioral Statistics, , vol. 45(4), pages 475-506, August.
    16. Choi, Jin-young & Lee, Goeun & Lee, Myoung-jae, 2023. "Endogenous treatment effect for any response conditional on control propensity score," Statistics & Probability Letters, Elsevier, vol. 196(C).
    17. Isaac Meza & Rahul Singh, 2021. "Nested Nonparametric Instrumental Variable Regression: Long Term, Mediated, and Time Varying Treatment Effects," Papers 2112.14249, arXiv.org, revised Mar 2024.
    18. Avi Feller & Fabrizia Mealli & Luke Miratrix, 2017. "Principal Score Methods: Assumptions, Extensions, and Practical Considerations," Journal of Educational and Behavioral Statistics, , vol. 42(6), pages 726-758, December.
    19. Gilbert Peter B. & Blette Bryan S. & Shepherd Bryan E. & Hudgens Michael G., 2020. "Post-randomization Biomarker Effect Modification Analysis in an HIV Vaccine Clinical Trial," Journal of Causal Inference, De Gruyter, vol. 8(1), pages 54-69, January.
    20. Andrey Fradkin & Elena Grewal & David Holtz, 2021. "Reciprocity and Unveiling in Two-Sided Reputation Systems: Evidence from an Experiment on Airbnb," Marketing Science, INFORMS, vol. 40(6), pages 1013-1029, November.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:bla:jorssc:v:71:y:2022:i:5:p:1065-1084. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Wiley Content Delivery (email available below). General contact details of provider: https://edirc.repec.org/data/rssssea.html .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.