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Causal Effect Models for Realistic Individualized Treatment and Intention to Treat Rules

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  • van der Laan Mark J.

    (Division of Biostatistics, School of Public Health, University of California, Berkeley)

  • Petersen Maya L

    (University of California, Berkeley)

Abstract

Marginal structural models (MSM) are an important class of models in causal inference. Given a longitudinal data structure observed on a sample of n independent and identically distributed experimental units, MSM model the counterfactual outcome distribution corresponding with a static treatment intervention, conditional on user-supplied baseline covariates. Identification of a static treatment regimen-specific outcome distribution based on observational data requires, beyond the standard sequential randomization assumption, the assumption that each experimental unit has positive probability of following the static treatment regimen. The latter assumption is called the experimental treatment assignment (ETA) assumption, and is parameter-specific. In many studies the ETA is violated because some of the static treatment interventions to be compared cannot be followed by all experimental units, due either to baseline characteristics or to the occurrence of certain events over time. For example, the development of adverse effects or contraindications can force a subject to stop an assigned treatment regimen.In this article we propose causal effect models for a user-supplied set of realistic individualized treatment rules. Realistic individualized treatment rules are defined as treatment rules which always map into the set of possible treatment options. Thus, causal effect models for realistic treatment rules do not rely on the ETA assumption and are fully identifiable from the data. Further, these models can be chosen to generalize marginal structural models for static treatment interventions. The estimating function methodology of Robins and Rotnitzky (1992) (analogue to its application in Murphy, et. al. (2001) for a single treatment rule) provides us with the corresponding locally efficient double robust inverse probability of treatment weighted estimator.In addition, we define causal effect models for "intention-to-treat" regimens. The proposed intention-to-treat interventions enforce a static intervention until the time point at which the next treatment does not belong to the set of possible treatment options, at which point the intervention is stopped. We provide locally efficient estimators of such intention-to-treat causal effects.

Suggested Citation

  • 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.
  • Handle: RePEc:bpj:ijbist:v:3:y:2007:i:1:n:3
    DOI: 10.2202/1557-4679.1022
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    Citations

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    Cited by:

    1. 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.
    2. Kristin A. Linn & Eric B. Laber & Leonard A. Stefanski, 2017. "Interactive -Learning for Quantiles," Journal of the American Statistical Association, Taylor & Francis Journals, vol. 112(518), pages 638-649, April.
    3. 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.
    4. 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.
    5. van der Laan Mark J. & Gruber Susan, 2010. "Collaborative Double Robust Targeted Maximum Likelihood Estimation," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-71, May.
    6. Adel Daoud & Anders Herlitz & SV Subramanian, 2020. "Combining distributive ethics and causal Inference to make trade-offs between austerity and population health," Papers 2007.15550, arXiv.org, revised Aug 2020.
    7. van der Laan Mark J., 2010. "Targeted Maximum Likelihood Based Causal Inference: Part II," The International Journal of Biostatistics, De Gruyter, vol. 6(2), pages 1-33, February.
    8. Kim Kwangho & Kennedy Edward H. & Naimi Ashley I., 2021. "Incremental intervention effects in studies with dropout and many timepoints#," Journal of Causal Inference, De Gruyter, vol. 9(1), pages 302-344, January.
    9. Cain Lauren E. & Robins James M. & Lanoy Emilie & Logan Roger & Costagliola Dominique & Hernán Miguel A., 2010. "When to Start Treatment? A Systematic Approach to the Comparison of Dynamic Regimes Using Observational Data," The International Journal of Biostatistics, De Gruyter, vol. 6(2), pages 1-26, April.
    10. Biernot Peter & Moodie Erica E. M., 2010. "A Comparison of Variable Selection Approaches for Dynamic Treatment Regimes," The International Journal of Biostatistics, De Gruyter, vol. 6(1), pages 1-20, January.
    11. Jacqueline A. Mauro & Edward H. Kennedy & Daniel Nagin, 2020. "Instrumental variable methods using dynamic interventions," Journal of the Royal Statistical Society Series A, Royal Statistical Society, vol. 183(4), pages 1523-1551, October.
    12. Daoud, Adel & Herlitz, Anders & Subramanian, S.V., 2022. "IMF fairness: Calibrating the policies of the International Monetary Fund based on distributive justice," World Development, Elsevier, vol. 157(C).
    13. Cole Manschot & Eric Laber & Marie Davidian, 2023. "Interim monitoring of sequential multiple assignment randomized trials using partial information," Biometrics, The International Biometric Society, vol. 79(4), pages 2881-2894, December.
    14. Lina M. Montoya & Michael R. Kosorok & Elvin H. Geng & Joshua Schwab & Thomas A. Odeny & Maya L. Petersen, 2023. "Efficient and robust approaches for analysis of sequential multiple assignment randomized trials: Illustration using the ADAPT‐R trial," Biometrics, The International Biometric Society, vol. 79(3), pages 2577-2591, September.
    15. Jiacheng Wu & Nina Galanter & Susan M. Shortreed & Erica E.M. Moodie, 2022. "Ranking tailoring variables for constructing individualized treatment rules: An application to schizophrenia," Journal of the Royal Statistical Society Series C, Royal Statistical Society, vol. 71(2), pages 309-330, March.
    16. 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.
    17. Noémi Kreif & Oleg Sofrygin & Julie A. Schmittdiel & Alyce S. Adams & Richard W. Grant & Zheng Zhu & Mark J. van der Laan & Romain Neugebauer, 2021. "Exploiting nonsystematic covariate monitoring to broaden the scope of evidence about the causal effects of adaptive treatment strategies," Biometrics, The International Biometric Society, vol. 77(1), pages 329-342, March.

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