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Bayesian methods to compare dose levels with placebo in a small n, sequential, multiple assignment, randomized trial

dc.contributor.authorFang, Fang
dc.contributor.authorHochstedler, Kimberly A.
dc.contributor.authorTamura, Roy N.
dc.contributor.authorBraun, Thomas M.
dc.contributor.authorKidwell, Kelley M.
dc.date.accessioned2021-02-04T21:53:16Z
dc.date.available2022-03-04 16:53:15en
dc.date.available2021-02-04T21:53:16Z
dc.date.issued2021-02-20
dc.identifier.citationFang, Fang; Hochstedler, Kimberly A.; Tamura, Roy N.; Braun, Thomas M.; Kidwell, Kelley M. (2021). "Bayesian methods to compare dose levels with placebo in a small n, sequential, multiple assignment, randomized trial." Statistics in Medicine 40(4): 963-977.
dc.identifier.issn0277-6715
dc.identifier.issn1097-0258
dc.identifier.urihttps://hdl.handle.net/2027.42/166252
dc.publisherWiley Periodicals, Inc.
dc.subject.otheradaptive randomization
dc.subject.otherrepeated measures
dc.subject.otherclinical trial
dc.titleBayesian methods to compare dose levels with placebo in a small n, sequential, multiple assignment, randomized trial
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelPublic Health
dc.subject.hlbsecondlevelMedicine (General)
dc.subject.hlbsecondlevelStatistics and Numeric Data
dc.subject.hlbtoplevelHealth Sciences
dc.subject.hlbtoplevelScience
dc.subject.hlbtoplevelSocial Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/166252/1/sim8813.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/166252/2/sim8813_am.pdf
dc.identifier.doi10.1002/sim.8813
dc.identifier.doihttps://dx.doi.org/10.7302/175
dc.identifier.sourceStatistics in Medicine
dc.identifier.citedreferenceFDA guidance for industry (Draft). Rare diseases: common issues in drug development; 2015.
dc.identifier.citedreferenceWilliamson T, Eliasziw M, Fick GH. Log‐binomial models: exploring failed convergence. Emerg Themes Epidemiol. 2013; 10 ( 1 ): 14.
dc.identifier.citedreferenceWei B, Braun TM, Tamura RN, Kidwell KM. A Bayesian analysis of small n sequential multiple assignment randomized trials (snSMARTs). Stat Medic. 2018; 37 ( 26 ): 3723 ‐ 3732.
dc.identifier.citedreferenceHonkanen VEA, Siegel AF, Szalai JP, Berger V, Feldman BM, Siegel JN. A three‐stage clinical trial design for rare disorders. Stat Medic. 2001; 20 ( 20 ): 3009 ‐ 3021.
dc.identifier.citedreferenceGriggs RC, Batshaw M, Dunkle M, et al. Clinical research for rare disease: opportunities, challenges and solutions. Mol Genet Metab. 2009; 96 ( 1 ): 20 ‐ 26.
dc.identifier.citedreferenceLevin KA. Study design VII. Randomised controlled trials. Evidence‐Based Dentistry. 2007; 8 ( 1 ): 22.
dc.identifier.citedreferenceNason M, Follmann D. Design and analysis of crossover trials for absorbing binary endpoints. Biometrics. 2010; 66 ( 3 ): 958 ‐ 965.
dc.identifier.citedreferenceMakubate B, Senn S. Planning and analysis of cross‐over trials in infertility. Stat Medic. 2010; 29 ( 30 ): 3203 ‐ 3210.
dc.identifier.citedreferenceAlmirall D, Compton SN, Rynn MA, Walkup JT, Murphy SA. SMARTer discontinuation trial designs for developing an adaptive treatment strategy. J Child Adolescent Psychopharmacol. 2012; 22 ( 5 ): 364 ‐ 374.
dc.identifier.citedreferenceRosner GL, Stadler W, Ratain MJ. Randomized discontinuation design: application to cytostatic antineoplastic agents. J Clin Oncol. 2002; 20 ( 22 ): 4478 ‐ 4484.
dc.identifier.citedreferenceMurphy SA. Optimal dynamic treatment regimes. J Royal Stat Soc Ser B (Stat Methodol). 2003; 65 ( 2 ): 331 ‐ 355.
dc.identifier.citedreferenceRobins J. A new approach to causal inference in mortality studies with a sustained exposure period–application to control of the healthy worker survivor effect. Math Modell. 1986; 7 ( 9‐12 ): 1393 ‐ 1512.
dc.identifier.citedreferenceMurphy SA. An experimental design for the development of adaptive treatment strategies. Stat Medic. 2005; 24 ( 10 ): 1455 ‐ 1481.
dc.identifier.citedreferenceLavori PW, Dawson R. A design for testing clinical strategies: biased adaptive within‐subject randomization. J Royal Stat Soc Ser A (Stat Soc). 2000; 163 ( 1 ): 29 ‐ 38.
dc.identifier.citedreferenceTamura RN, Krischer JP, Pagnoux C, et al. A small n sequential multiple assignment randomized trial design for use in rare disease research. Contemp Clin Trials. 2016; 46: 48 ‐ 51.
dc.identifier.citedreferenceGupta S, Faughnan ME, Tomlinson GA, Bayoumi AM. A framework for applying unfamiliar trial designs in studies of rare diseases. J Clin Epidemiol. 2011; 64 ( 10 ): 1085 ‐ 1094.
dc.identifier.citedreference107th Congress rare diseases act of 2002. Pub Law; 2002:107‐280.
dc.working.doi10.7302/175en
dc.owningcollnameInterdisciplinary and Peer-Reviewed


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