IDEAS home Printed from https://ideas.repec.org/a/bpj/ijbist/v8y2012i1n14.html
   My bibliography  Save this article

Targeted Maximum Likelihood Estimation for Dynamic Treatment Regimes in Sequentially Randomized Controlled Trials

Author

Listed:
  • Chaffee Paul H.

    (University of California, Berkeley)

  • van der Laan Mark J.

    (University of California, Berkeley)

Abstract

Sequential Randomized Controlled Trials (SRCTs) are rapidly becoming essential tools in the search for optimized treatment regimes in ongoing treatment settings. Analyzing data for multiple time-point treatments with a view toward optimal treatment regimes is of interest in many types of afflictions: HIV infection, Attention Deficit Hyperactivity Disorder in children, leukemia, prostate cancer, renal failure, and many others. Methods for analyzing data from SRCTs exist but they are either inefficient or suffer from the drawbacks of estimating equation methodology. We describe an estimation procedure, targeted maximum likelihood estimation (TMLE), which has been fully developed and implemented in point treatment settings, including time to event outcomes, binary outcomes and continuous outcomes. Here we develop and implement TMLE in the SRCT setting. As in the former settings, the TMLE procedure is targeted toward a pre-specified parameter of the distribution of the observed data, and thereby achieves important bias reduction in estimation of that parameter. As with the so-called Augmented Inverse Probability of Censoring Weight (A-IPCW) estimator, TMLE is double-robust and locally efficient. We report simulation results corresponding to two data-generating distributions from a longitudinal data structure.

Suggested Citation

  • Chaffee Paul H. & van der Laan Mark J., 2012. "Targeted Maximum Likelihood Estimation for Dynamic Treatment Regimes in Sequentially Randomized Controlled Trials," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-32, June.
    • Handle: RePEc:bpj:ijbist:v:8:y:2012:i:1:n:14
    DOI: 10.1515/1557-4679.1406
    as

    Download full text from publisher

    File URL: https://doi.org/10.1515/1557-4679.1406
    Download Restriction: For access to full text, subscription to the journal or payment for the individual article is required.
    File URL: https://libkey.io/10.1515/1557-4679.1406?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
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Stitelman Ori M & van der Laan Mark J., 2010. "Collaborative Targeted Maximum Likelihood for Time to Event Data," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-46, June.
    2. Guo Xiang & Tsiatis Anastasios, 2005. "A Weighted Risk Set Estimator for Survival Distributions in Two-Stage Randomization Designs with Censored Survival Data," The International Journal of Biostatistics, De Gruyter, vol. 1(1), pages 1-17, May.
    3. Jared K. Lunceford & Marie Davidian & Anastasios A. Tsiatis, 2002. "Estimation of Survival Distributions of Treatment Policies in Two-Stage Randomization Designs in Clinical Trials," Biometrics, The International Biometric Society, vol. 58(1), pages 48-57, March.
    4. Murphy S.A. & van der Laan M.J. & Robins J.M., 2001. "Marginal Mean Models for Dynamic Regimes," Journal of the American Statistical Association, American Statistical Association, vol. 96, pages 1410-1423, December.
    5. Porter Kristin E. & Gruber Susan & van der Laan Mark J. & Sekhon Jasjeet S., 2011. "The Relative Performance of Targeted Maximum Likelihood Estimators," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-34, August.
    6. Lu Wang & Andrea Rotnitzky & Xihong Lin & Randall E. Millikan & Peter F. Thall, 2012. "Evaluation of Viable Dynamic Treatment Regimes in a Sequentially Randomized Trial of Advanced Prostate Cancer," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 107(498), pages 493-508, June.
    7. Gruber Susan & van der Laan Mark J., 2010. "A Targeted Maximum Likelihood Estimator of a Causal Effect on a Bounded Continuous Outcome," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-18, August.
    8. Moodie, Erica E. M. & Platt, Robert W. & Kramer, Michael S., 2009. "Estimating Response-Maximized Decision Rules With Applications to Breastfeeding," Journal of the American Statistical Association, American Statistical Association, vol. 104(485), pages 155-165.
    9. Orellana Liliana & Rotnitzky Andrea & Robins James M., 2010. "Dynamic Regime Marginal Structural Mean Models for Estimation of Optimal Dynamic Treatment Regimes, Part I: Main Content," The International Journal of Biostatistics, De Gruyter, vol. 6(2), pages 1-49, March.
    10. Abdus S. Wahed & Anastasios A. Tsiatis, 2004. "Optimal Estimator for the Survival Distribution and Related Quantities for Treatment Policies in Two-Stage Randomization Designs in Clinical Trials," Biometrics, The International Biometric Society, vol. 60(1), pages 124-133, March.
    11. van der Laan Mark J. & Petersen Maya L, 2007. "Causal Effect Models for Realistic Individualized Treatment and Intention to Treat Rules," The International Journal of Biostatistics, De Gruyter, vol. 3(1), pages 1-55, March.
    12. van der Laan Mark J. & Rubin Daniel, 2006. "Targeted Maximum Likelihood Learning," The International Journal of Biostatistics, De Gruyter, vol. 2(1), pages 1-40, December.
    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. Yasuhiro Hagiwara & Tomohiro Shinozaki & Hirofumi Mukai & Yutaka Matsuyama, 2021. "Sensitivity analysis for subsequent treatments in confirmatory oncology clinical trials: A two‐stage stochastic dynamic treatment regime approach," Biometrics, The International Biometric Society, vol. 77(2), pages 702-714, June.
    2. Stitelman Ori M & Wester C. William & De Gruttola Victor & van der Laan Mark J., 2011. "Targeted Maximum Likelihood Estimation of Effect Modification Parameters in Survival Analysis," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-34, March.
    3. Rose Sherri & van der Laan Mark J., 2011. "A Targeted Maximum Likelihood Estimator for Two-Stage Designs," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-21, March.
    4. Susan Gruber & Mark J. van der Laan, 2013. "An Application of Targeted Maximum Likelihood Estimation to the Meta-Analysis of Safety Data," Biometrics, The International Biometric Society, vol. 69(1), pages 254-262, March.
    5. Xinyu Tang & Abdus S. Wahed, 2011. "Comparison of treatment regimes with adjustment for auxiliary variables," Journal of Applied Statistics, Taylor & Francis Journals, vol. 38(12), pages 2925-2938, March.
    6. Stitelman Ori M. & De Gruttola Victor & van der Laan Mark J., 2012. "A General Implementation of TMLE for Longitudinal Data Applied to Causal Inference in Survival Analysis," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-39, September.
    7. Zhen Li & Jie Chen & Eric Laber & Fang Liu & Richard Baumgartner, 2023. "Optimal Treatment Regimes: A Review and Empirical Comparison," International Statistical Review, International Statistical Institute, vol. 91(3), pages 427-463, December.
    8. van der Laan Mark J., 2014. "Targeted Estimation of Nuisance Parameters to Obtain Valid Statistical Inference," The International Journal of Biostatistics, De Gruyter, vol. 10(1), pages 1-29, May.
    9. Tang, Xinyu & Melguizo, Maria, 2015. "DTR: An R Package for Estimation and Comparison of Survival Outcomes of Dynamic Treatment," Journal of Statistical Software, Foundation for Open Access Statistics, vol. 65(i07).
    10. Lucia Babino & Andrea Rotnitzky & James Robins, 2019. "Multiple robust estimation of marginal structural mean models for unconstrained outcomes," Biometrics, The International Biometric Society, vol. 75(1), pages 90-99, March.
    11. Gruber Susan & van der Laan Mark J., 2010. "A Targeted Maximum Likelihood Estimator of a Causal Effect on a Bounded Continuous Outcome," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-18, August.
    12. Gruber Susan & van der Laan Mark J., 2010. "An Application of Collaborative Targeted Maximum Likelihood Estimation in Causal Inference and Genomics," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-31, May.
    13. Rich Benjamin & Moodie Erica E. M. & A. Stephens David, 2016. "Influence Re-weighted G-Estimation," The International Journal of Biostatistics, De Gruyter, vol. 12(1), pages 157-177, May.
    14. Mireille E. Schnitzer & Erica E.M. Moodie & Mark J. van der Laan & Robert W. Platt & Marina B. Klein, 2014. "Modeling the impact of hepatitis C viral clearance on end-stage liver disease in an HIV co-infected cohort with targeted maximum likelihood estimation," Biometrics, The International Biometric Society, vol. 70(1), pages 144-152, March.
    15. Frölich, Markus & Huber, Martin & Wiesenfarth, Manuel, 2017. "The finite sample performance of semi- and non-parametric estimators for treatment effects and policy evaluation," Computational Statistics & Data Analysis, Elsevier, vol. 115(C), pages 91-102.
    16. van der Laan Mark J. & Petersen Maya & Zheng Wenjing, 2013. "Estimating the Effect of a Community-Based Intervention with Two Communities," Journal of Causal Inference, De Gruyter, vol. 1(1), pages 83-106, June.
    17. van der Laan Mark J., 2010. "Targeted Maximum Likelihood Based Causal Inference: Part I," The International Journal of Biostatistics, De Gruyter, vol. 6(2), pages 1-45, February.
    18. Brooks Jordan & van der Laan Mark J. & Go Alan S., 2012. "Targeted Maximum Likelihood Estimation for Prediction Calibration," The International Journal of Biostatistics, De Gruyter, vol. 8(1), pages 1-35, October.
    19. Markus Frölich & Martin Huber, 2014. "Treatment Evaluation With Multiple Outcome Periods Under Endogeneity and Attrition," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 109(508), pages 1697-1711, December.
    20. Yuliya Lokhnygina & Jeffrey D. Helterbrand, 2007. "Cox Regression Methods for Two-Stage Randomization Designs," Biometrics, The International Biometric Society, vol. 63(2), pages 422-428, June.

    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:bpj:ijbist:v:8:y:2012:i:1:n:14. 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: Peter Golla (email available below). General contact details of provider: https://www.degruyter.com .

    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.