Screening Experiments for Developing Dynamic Treatment Regimes
Open accessAbstract
Dynamic treatment regimes are time-varying treatments that individualize sequences of treatments to the patient. The construction of dynamic treatment regimes is challenging because a patient will be eligible for some treatment components only if he has not responded (or has responded) to other treatment components. In addition there are usually a number of potentially useful treatment components and combinations thereof. In this article, we propose new methodology for identifying promising components and screening out negligible ones. First, we define causal factorial effects for treatment components that may be applied sequentially to a patient. Second we propose experimental designs that can be used to study the treatment components. Surprisingly, modifications can be made to (fractional) factorial designs - more commonly found in the engineering statistics literature -for screening in this setting. Furthermore we provide an analysis model that can be used to screen the factorial effects. We demonstrate the proposed methodology using examples motivated in the literature and also via a simulation study.
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A SMART is a form of factorial experimental design.
“a SMART is a form of a factorial experimental design [17].”
From Introduction to SMART designs for the development of adaptive interventions: with application to weight loss research (2014) · cites this paper as [17]
SMART is a type of factorial design.
“SMART is a type of factorial design [17]”
From Introduction to SMART designs for the development of adaptive interventions: with application to weight loss research (2014) · cites this paper as [17]
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Cites
Reinforcement Learning: An Introduction
Sutton, R. S., & Barto, A. (2005) · IEEE Transactions on Neural Networks · doi:10.1109/tnn.2004.842673 · Cited by 25,263 · see citing works
Estimating causal effects of treatments in randomized and nonrandomized studies
Rubin, D. B. (1974) · Journal of Educational Psychology · doi:10.1037/h0037350 · Cited by 9,436 · see citing works
Statistics for experimenters. An introduction to design, data analysis, and model building.
Box, G. E. P., Hunter, W. G., & Hunter, J. S. (1978) · Cited by 4,021 · see citing works
Bayesian inference for causal effects: The role of randomization
Rubin, D. B. (1978) · The Annals of Statistics · doi:10.1214/aos/1176344064 · Cited by 2,503 · see citing works
Robins, J. (1986) · Mathematical Modelling · doi:10.1016/0270-0255(86)90088-6 · Cited by 2,451 · see citing works
Experiments - Planning, Analysis and Parameter Design Optimization
(2001) · Journal of Tropical Pediatrics · doi:10.1093/tropej/47.2.126 · Cited by 1,132 · see citing works
Collins, L. M., Murphy, S. A., & Strecher, V. (2007) · American Journal of Preventive Medicine · doi:10.1016/j.amepre.2007.01.022 · Cited by 1,082 · see citing works
Self-citationOptimal Dynamic Treatment Regimes
Murphy, S. A. (2003) · Journal of the Royal Statistical Society Series B (Statistical Methodology) · doi:10.1111/1467-9868.00389 · Cited by 988 · see citing works
Self-citationSemiparametric Theory and Missing Data
Tsiatis, A. A. (2006) · Springer eBooks · doi:10.1007/0-387-37345-4 · Cited by 859 · see citing works
Stroup, T., Mcevoy, J., Swartz, M., Byerly, M., Glick, I., Cañive, J., McGee, M., Simpson, G., Stevens, M., & Lieberman, J. (2003) · Schizophrenia bulletin · doi:10.1093/oxfordjournals.schbul.a006986 · Cited by 647 · see citing works
An experimental design for the development of adaptive treatment strategies
Murphy, S. A. (2005) · Statistics in Medicine · doi:10.1002/sim.2022 · Cited by 595 · see citing works
Self-citationOptimal Structural Nested Models for Optimal Sequential Decisions
Robins, J. M. (2004) · Lecture notes in statistics · doi:10.1007/978-1-4419-9076-1_11 · Cited by 547 · see citing works
Semiparametric efficiency bounds
Newey, W. K. (1990) · Journal of Applied Econometrics · doi:10.1002/jae.3950050202 · Cited by 533 · see citing works
D, M. F. M., Rush, A. J., Trivedi, M. H., Nierenberg, A. A., Thase, M. E., Sackeïm, H. A., Quitkin, F. M., Wisniewski, S. R., Lavori, P. W., Rosenbaum, J. F., et al. (2003) · Psychiatric Clinics of North America · doi:10.1016/s0193-953x(02)00107-7 · Cited by 520 · see citing works
Causal Inference from Complex Longitudinal Data
Robins, J. M. (1997) · Lecture Notes in Statistics · doi:10.1007/978-1-4612-1842-5_4 · Cited by 510 · see citing works
Marginal Mean Models for Dynamic Regimes
Murphy, S. A., van der Laan, M. J., Robins, J. M., & Conduct Problems Prevention Research Group (2001) · Journal of the American Statistical Association · doi:10.1198/016214501753382327 · Cited by 393 · see citing works
Self-citationMinimum Aberration 2k–pDesigns
Fries, A., & Hunter, W. G. (1980) · Technometrics · doi:10.1080/00401706.1980.10486210 · Cited by 360 · see citing works
Asymptotic efficiency in semi-parametric models with censoring
Chamberlain, G. (1986) · Journal of Econometrics · doi:10.1016/0304-4076(86)90038-2 · Cited by 271 · see citing works
Robins, J. M. (1987) · Computers & Mathematics with Applications · doi:10.1016/0898-1221(87)90238-0 · Cited by 207 · see citing works
The Theory of Response-Adaptive Randomization in Clinical Trials
Hu, F., & Rosenberger, W. F. (2006) · Cited by 201 · see citing works
Robins, J. M., & Greenland, S. (1994) · Journal of the American Statistical Association · doi:10.1080/01621459.1994.10476807 · Cited by 181 · see citing works
Robins, J. M. (1992) · Biometrika · doi:10.1093/biomet/79.2.321 · Cited by 180 · see citing works
Flexible treatment strategies in chronic disease: clinical and research implications
Lavori, P. W., Dawson, R., & Rush, A. J. (2000) · Biological Psychiatry · doi:10.1016/s0006-3223(00)00946-x · Cited by 138 · see citing works
Selecting Therapeutic Strategies Based on Efficacy and Death in Multicourse Clinical Trials
Thall, P. F., Sung, H., & Estey, E. H. (2002) · Journal of the American Statistical Association · doi:10.1198/016214502753479202 · Cited by 117 · see citing works
Latent Variable Modeling and Applications to Causality
Berkane, M. (1997) · Lecture notes in statistics · doi:10.1007/978-1-4612-1842-5 · Cited by 110 · see citing works
Texas Medication Algorithm Project, Phase 3 (TMAP-3)
Rush, A. J., Crismon, M. L., Kashner, T. M., Toprac, M. G., Carmody, T. J., Trivedi, M. H., Suppes, T. M., Miller, A. L., Biggs, M. M., Shores‐Wilson, K., et al. (2003) · The Journal of Clinical Psychiatry · doi:10.4088/jcp.v64n0402 · Cited by 108 · see citing works
Strategies for comparing treatments on a binary response with multi-centre data
Agresti, A., & Hartzel, J. S. (2000) · Statistics in Medicine · doi:10.1002/(sici)1097-0258(20000430)19:8 · Cited by 102 · see citing works
Modeling in Medical Decision Making: A Bayesian Approach
Parmigiani, G. (2002) · Cited by 97 · see citing works
Robins, J. M. (1987) · Computers & Mathematics with Applications · doi:10.1016/0898-1221(87)90237-9 · Cited by 91 · see citing works
Factorial designs for randomized clinical trials.
Byar, D. P., & Piantadosi, S. (1985) · PubMed · Cited by 85 · see citing works
Group-Screening with More Than Two Stages
Patel, M. S. (1962) · Technometrics · doi:10.1080/00401706.1962.10490005 · Cited by 68 · see citing works
Estimation of Effects of Sequential Treatments by Reparameterizing Directed Acyclic GraphsPreprint
Robins, J. M., & Wasserman, L. (2013) · arXiv (Cornell University) · doi:10.48550/arxiv.1302.1566 · Cited by 60 · see citing works
Using Behavioral Reinforcement To Improve Methadone Treatment Participation
Brooner, R. K., & KIDORF, M. S. (2002) · Science & Practice Perspectives · doi:10.1151/spp021138 · Cited by 60 · see citing works
Structured treatment interruptions in HIV/AIDS therapy
Lisziewicz, J., & Lori, F. (2002) · Microbes and Infection · doi:10.1016/s1286-4579(01)01529-5 · Cited by 36 · see citing works
Group-Screening with More Than Two Stages
Patel, M. S. (1962) · Technometrics · doi:10.2307/1266619 · Cited by 30 · see citing works
Robins, J. M., & Greenland, S. (1994) · Journal of the American Statistical Association · doi:10.2307/2290899 · Cited by 25 · see citing works
5 Adaptive designs for parametric models
Zacks, S. (1996) · Handbook of statistics · doi:10.1016/s0169-7161(96)13007-8 · Cited by 12 · see citing works
An Overview of Industrial Screening Experiments
Montgomery, D. C., & Jennings, C. L. (2006) · Screening · doi:10.1007/0-387-28014-6_1 · Cited by 5 · see citing works
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Almirall, D., Nahum-Shani, I., Sherwood, N. E., & Murphy, S. A. (2014) · Translational Behavioral Medicine · doi:10.1007/s13142-014-0265-0 · Cited by 453 · see citing works
Self-citationAlmirall, D., Compton, S. N., Gunlicks‐Stoessel, M., Duan, N., & Murphy, S. A. (2012) · Statistics in Medicine · doi:10.1002/sim.4512 · Cited by 223 · see citing works
Self-citationWang, L., Rotnitzky, A., Lin, X., Millikan, R. E., & Thall, P. F. (2012) · Journal of the American Statistical Association · doi:10.1080/01621459.2011.641416 · Cited by 108 · see citing works
Almirall, D., Lizotte, D. J., & Murphy, S. A. (2012) · Journal of the American Statistical Association · doi:10.1080/01621459.2012.665615 · Cited by 19 · see citing works
Self-citationBuyze, J., Van Rompaye, B., & Goetghebeur, E. (2010) · Statistics in Medicine · doi:10.1002/sim.3856 · Cited by 9 · see citing works
- Authors
- Murphy, S. A., & Bingham, D.
- Affiliations
- University of Michigan (US)
- Simon Fraser University (CA)
- Venue
- Journal of the American Statistical Association
- Publisher
- Informa UK Limited
- Published
- 2009-03
- Volume
- 104
- Issue
- 485
- Pages
- 391-408
- ISSN
- 0162-1459 (ISSN-L)
- 1537-274X
- PMC
- PMC2892819
- OpenAlex
- W2078249808
- Cited by
- 41
- References
- 39
- Citations by year
- 2025: 4 · 2024: 2 · 2023: 1 · 2022: 4 · 2021: 1 · 2020: 1 · 2019: 1 · 2018: 6 · 2017: 2 · 2016: 1 · 2014: 5 · 2013: 5 · 2012: 4
- FWCI
- 2.21
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Journal of the American Statistical Association (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- RePEc: Research Papers in Economics (submittedVersion)
- Funders
- National Institute of Mental Health (R01MH080015)
- National Institute on Drug Abuse (P50DA010075)
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Advanced Causal Inference Techniques
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Social Sciences › Decision Sciences › Management Science and Operations Research › Optimal Experimental Design Methods
- Keywords
- dynamic treatment regimes, sequential therapy, fractional factorial design, personalized treatment, treatment eligibility, causal inference
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