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Volumn 14, Issue 5, 2017, Pages 462-469

Adaptive designs from a Data Safety Monitoring Board perspective: Some controversies and some case studies

Author keywords

Adaptive design; enrichment design; flexible design; group sequential test; multi arm multi stage trial; optimal design; promising zone; sample size re estimation; seamless Phase II III design

Indexed keywords

BAYES THEOREM; CASE STUDY; COMPUTER ASSISTED TOMOGRAPHY; CONFERENCE PAPER; DECISION MAKING; DOSE RESPONSE; HOSPITALIZATION; HUMAN; INFORMATION PROCESSING; INTERVENTION STUDY; OVERALL SURVIVAL; PATIENT MONITORING; PRIORITY JOURNAL; REPRODUCIBILITY; RISK BENEFIT ANALYSIS; SAMPLE SIZE; BIOASSAY; CLINICAL TRIAL (TOPIC); DRUG DESIGN; ETHICS; METHODOLOGY; PROCEDURES; PROFESSIONAL STANDARD; STANDARDS; STATISTICAL ANALYSIS;

EID: 85021087924     PISSN: 17407745     EISSN: 17407753     Source Type: Journal    
DOI: 10.1177/1740774516689261     Document Type: Conference Paper
Times cited : (6)

References (40)
  • 1
    • 84954167098 scopus 로고    scopus 로고
    • Twenty-five years of confirmatory adaptive designs: Opportunities and pitfalls
    • Bauer P, Bretz F, Dragalin V, et al. Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Stat Med 2016; 30: 325-347.
    • (2016) Stat Med , vol.30 , pp. 325-347
    • Bauer, P.1    Bretz, F.2    Dragalin, V.3
  • 3
    • 33748533820 scopus 로고    scopus 로고
    • Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: Applications and practical considerations
    • Schmidli H, Bretz F, Racine A, et al. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: applications and practical considerations. Biometrical J 2006; 48: 635-643.
    • (2006) Biometrical J , vol.48 , pp. 635-643
    • Schmidli, H.1    Bretz, F.2    Racine, A.3
  • 4
    • 33749333699 scopus 로고    scopus 로고
    • Standard versus adaptive monitoring procedures: A commentary
    • Fleming TR,. Standard versus adaptive monitoring procedures: a commentary. Stat Med 2006; 25: 3305-3312.
    • (2006) Stat Med , vol.25 , pp. 3305-3312
    • Fleming, T.R.1
  • 5
    • 1642625262 scopus 로고    scopus 로고
    • Effects of selenium supplementation for cancer prevention in patients with carcinoma of the skin: A randomized clinical trial
    • (Editorial: 1984 - 1985.)
    • Clark LC, Combs GF, Turnbull BW, et al. Effects of selenium supplementation for cancer prevention in patients with carcinoma of the skin: a randomized clinical trial. JAMA 1996; 276: 1957-1963 (Editorial: pp. 1984-1985.)
    • (1996) JAMA , vol.276 , pp. 1957-1963
    • Clark, L.C.1    Combs, G.F.2    Turnbull, B.W.3
  • 6
    • 21444457563 scopus 로고    scopus 로고
    • Reporting conflicts of interest, financial aspects of research, and role of sponsors in funded studies
    • Fontanarosa PB, Flanagan A, DeAngelis CD,. Reporting conflicts of interest, financial aspects of research, and role of sponsors in funded studies. JAMA 2005; 296: 110-111.
    • (2005) JAMA , vol.296 , pp. 110-111
    • Fontanarosa, P.B.1    Flanagan, A.2    DeAngelis, C.D.3
  • 7
    • 84875655969 scopus 로고    scopus 로고
    • Effect of an investigational vaccine for preventing staphylococcus aureus infections after cardiothoracic surgery: A randomized trial
    • Fowler VG, Allen KB, Moreira ED, et al. Effect of an investigational vaccine for preventing staphylococcus aureus infections after cardiothoracic surgery: a randomized trial. JAMA 2013; 309: 1368-1378.
    • (2013) JAMA , vol.309 , pp. 1368-1378
    • Fowler, V.G.1    Allen, K.B.2    Moreira, E.D.3
  • 8
    • 0001333062 scopus 로고    scopus 로고
    • Likelihood ratio tests for a changepoint with survival data
    • Luo X, Turnbull BW, Clark LC,. Likelihood ratio tests for a changepoint with survival data. Biometrika 1997; 84: 555-565.
    • (1997) Biometrika , vol.84 , pp. 555-565
    • Luo, X.1    Turnbull, B.W.2    Clark, L.C.3
  • 9
    • 79961108629 scopus 로고    scopus 로고
    • Reduced lung-cancer mortality with low-dose computed tomographic screening
    • The National Lung Screening Trial Research Team
    • The National Lung Screening Trial Research Team. Reduced lung-cancer mortality with low-dose computed tomographic screening. N Engl J Med 2011; 365: 395-409.
    • (2011) N Engl J Med , vol.365 , pp. 395-409
  • 10
    • 24944501851 scopus 로고    scopus 로고
    • ASTIN: A Bayesian adaptive dose-response trial in acute stroke
    • Grieve AP, Krams M,. ASTIN: a Bayesian adaptive dose-response trial in acute stroke. Clin Trials 2005; 2: 340-351.
    • (2005) Clin Trials , vol.2 , pp. 340-351
    • Grieve, A.P.1    Krams, M.2
  • 11
    • 78249285421 scopus 로고    scopus 로고
    • US Food and Drug Administration. Rockville, MD: Center for Drug Evaluation and Research (CDER); Center for Biologics Evaluation and Research (CBER), Sec.VIID
    • US Food and Drug Administration. Draft guidance for industry adaptive design clinical trials for drugs and biologics. Rockville, MD: Center for Drug Evaluation and Research (CDER); Center for Biologics Evaluation and Research (CBER), 2010, Sec.VIID.
    • (2010) Draft Guidance for Industry Adaptive Design Clinical Trials for Drugs and Biologics
  • 12
    • 85008365775 scopus 로고    scopus 로고
    • US Food and Drug Administration. Rockville, MD: Center for Devices and Radiological Health; Center for Biologics Evaluation and Research (CBER)
    • US Food and Drug Administration. Adaptive designs for medical device clinical studies, draft guidance. Rockville, MD: Center for Devices and Radiological Health; Center for Biologics Evaluation and Research (CBER), 2015, p. 26.
    • (2015) Adaptive Designs for Medical Device Clinical Studies, Draft Guidance , pp. 26
  • 13
    • 84966533874 scopus 로고    scopus 로고
    • Efficiencies of platform clinical trials: A vision of the future
    • Saville BR, Berry SM,. Efficiencies of platform clinical trials: a vision of the future. Clin Trials 2016; 13: 358-366.
    • (2016) Clin Trials , vol.13 , pp. 358-366
    • Saville, B.R.1    Berry, S.M.2
  • 14
    • 0035810547 scopus 로고    scopus 로고
    • Effect of carvedilol on outcome after myocardial infarction in patients with left-ventricular dysfunction: The CAPRICORN randomised trial
    • CAPRICORN Investigators
    • CAPRICORN Investigators. Effect of carvedilol on outcome after myocardial infarction in patients with left-ventricular dysfunction: the CAPRICORN randomised trial. Lancet 2001; 357: 1385-1390.
    • (2001) Lancet , vol.357 , pp. 1385-1390
  • 15
    • 0035029225 scopus 로고    scopus 로고
    • CAPRICORN: A story of alpha allocation and beta-blockers in left ventricular dysfunction post-MI
    • Coats AJ,. CAPRICORN: a story of alpha allocation and beta-blockers in left ventricular dysfunction post-MI. Int J Cardiol 2001; 78: 109-113.
    • (2001) Int J Cardiol , vol.78 , pp. 109-113
    • Coats, A.J.1
  • 16
    • 0032886394 scopus 로고    scopus 로고
    • Modification of sample size in group sequential clinical trials
    • Cui L, Hung HMJ, Wang SJ,. Modification of sample size in group sequential clinical trials. Biometrics 1999; 55: 853-857.
    • (1999) Biometrics , vol.55 , pp. 853-857
    • Cui, L.1    Hung, H.M.J.2    Wang, S.J.3
  • 17
    • 82055200248 scopus 로고    scopus 로고
    • Adaptive increase in sample size when interim results are promising: A practical guide with examples
    • Mehta CR, Pocock SJ,. Adaptive increase in sample size when interim results are promising: a practical guide with examples. Stat Med 2011; 30: 3267-3284.
    • (2011) Stat Med , vol.30 , pp. 3267-3284
    • Mehta, C.R.1    Pocock, S.J.2
  • 18
    • 84945978676 scopus 로고    scopus 로고
    • Adaptive sample size modification in clinical trials: Start small then ask for more?
    • Jennison C, Turnbull BW,. Adaptive sample size modification in clinical trials: start small then ask for more? Stat Med 2015; 34: 3793-3810.
    • (2015) Stat Med , vol.34 , pp. 3793-3810
    • Jennison, C.1    Turnbull, B.W.2
  • 19
    • 82055196645 scopus 로고    scopus 로고
    • Comments on "adaptive increase in sample size when interim results are promising: A practical guide with examples
    • Emerson SS, Levin GP, Emerson SC,. Comments on "adaptive increase in sample size when interim results are promising: a practical guide with examples." Stat Med 2011; 30: 3285-3301.
    • (2011) Stat Med , vol.30 , pp. 3285-3301
    • Emerson, S.S.1    Levin, G.P.2    Emerson, S.C.3
  • 20
    • 33644770078 scopus 로고    scopus 로고
    • Efficient group sequential designs when there are several effect sizes under consideration
    • Jennison C, Turnbull BW,. Efficient group sequential designs when there are several effect sizes under consideration. Stat Med 2006; 25: 917-932.
    • (2006) Stat Med , vol.25 , pp. 917-932
    • Jennison, C.1    Turnbull, B.W.2
  • 21
    • 33644973601 scopus 로고    scopus 로고
    • Adaptive and non-adaptive group sequential tests
    • Jennison C, Turnbull BW,. Adaptive and non-adaptive group sequential tests. Biometrika 2006; 93: 1-21.
    • (2006) Biometrika , vol.93 , pp. 1-21
    • Jennison, C.1    Turnbull, B.W.2
  • 22
    • 84855243901 scopus 로고    scopus 로고
    • Comments on "adaptive increase in sample size when interim results are promising: A practical guide with examples" by C. R. Mehta and S. J. Pocock
    • Glimm E,. Comments on "Adaptive increase in sample size when interim results are promising: a practical guide with examples" by C. R. Mehta and S. J. Pocock. Stat Med 2012; 31: 98-99.
    • (2012) Stat Med , vol.31 , pp. 98-99
    • Glimm, E.1
  • 24
    • 0037108040 scopus 로고    scopus 로고
    • On changing a long-term clinical trial midstream
    • Wittes JT,. On changing a long-term clinical trial midstream. Stat Med 2002; 27: 2789-2795.
    • (2002) Stat Med , vol.27 , pp. 2789-2795
    • Wittes, J.T.1
  • 25
    • 33644618943 scopus 로고    scopus 로고
    • Liability issues for data monitoring committee members
    • DeMets DL, Fleming TR, Rockhold F, et al. Liability issues for data monitoring committee members. Clin Trials 2004; 1: 525-531.
    • (2004) Clin Trials , vol.1 , pp. 525-531
    • DeMets, D.L.1    Fleming, T.R.2    Rockhold, F.3
  • 26
    • 84990043625 scopus 로고    scopus 로고
    • Data monitoring committees - Expecting the unexpected
    • DeMets DL, Ellenberg SS,. Data monitoring committees-expecting the unexpected. N Engl J Med 2016; 375: 1365-1371.
    • (2016) N Engl J Med , vol.375 , pp. 1365-1371
    • DeMets, D.L.1    Ellenberg, S.S.2
  • 27
    • 84925493847 scopus 로고    scopus 로고
    • Odanacatib for the treatment of postmenopausal osteoporosis: Development history and design and participant characteristics of LOFT, the Long-Term Odanacatib Fracture Trial
    • Bone HG, Dempster DW, Eisman JA, et al. Odanacatib for the treatment of postmenopausal osteoporosis: development history and design and participant characteristics of LOFT, the Long-Term Odanacatib Fracture Trial. Osteoporos Int 2015; 26: 699-712.
    • (2015) Osteoporos Int , vol.26 , pp. 699-712
    • Bone, H.G.1    Dempster, D.W.2    Eisman, J.A.3
  • 28
    • 84910130068 scopus 로고    scopus 로고
    • Building a more connected DSMB: Better integrating ethics review and safety monitoring
    • Eckstein L,. Building a more connected DSMB: better integrating ethics review and safety monitoring. Account Res 2015; 22: 81-105.
    • (2015) Account Res , vol.22 , pp. 81-105
    • Eckstein, L.1
  • 30
    • 84886896973 scopus 로고    scopus 로고
    • Bayesian hierarchical modeling of patient subpopulations: Efficient designs of Phase II oncology clinical trials
    • Berry SM, Broglio KR, Groshen S, et al. Bayesian hierarchical modeling of patient subpopulations: efficient designs of Phase II oncology clinical trials. Clin Trials 2013; 10: 720-734.
    • (2013) Clin Trials , vol.10 , pp. 720-734
    • Berry, S.M.1    Broglio, K.R.2    Groshen, S.3
  • 31
    • 77952301687 scopus 로고    scopus 로고
    • Robot-assisted therapy for long-term upper-limb impairment after stroke
    • Lo AC, Guarino PD, Richards LG, et al. Robot-assisted therapy for long-term upper-limb impairment after stroke. N Engl J Med 2010; 362: 1772-1783.
    • (2010) N Engl J Med , vol.362 , pp. 1772-1783
    • Lo, A.C.1    Guarino, P.D.2    Richards, L.G.3
  • 32
    • 0002857793 scopus 로고    scopus 로고
    • Estimation of parameters and sample size re-estimation
    • Lan KKG, Trost DC,. Estimation of parameters and sample size re-estimation. In: Proc Am Stat Assoc Biopharm Sect 1997; 32: 48-51.
    • (1997) Proc Am Stat Assoc Biopharm Sect , vol.32 , pp. 48-51
    • Lan, K.K.G.1    Trost, D.C.2
  • 33
    • 0035739842 scopus 로고    scopus 로고
    • Flexible sample size considerations using information based interim monitoring
    • Mehta C, Tsiatis A,. Flexible sample size considerations using information based interim monitoring. Drug Inf J 2001; 35: 1095-112.
    • (2001) Drug Inf J , vol.35 , pp. 1095-1112
    • Mehta, C.1    Tsiatis, A.2
  • 34
    • 33749322274 scopus 로고    scopus 로고
    • Information-based monitoring of clinical trials
    • Tsiatis AA,. Information-based monitoring of clinical trials. Stat Med 2006; 25: 3236-3244.
    • (2006) Stat Med , vol.25 , pp. 3236-3244
    • Tsiatis, A.A.1
  • 35
    • 34247113737 scopus 로고    scopus 로고
    • Optimal medical therapy with or without PCI for stable coronary disease
    • Boden WE, O'Rourke RA, Teo KK, et al. Optimal medical therapy with or without PCI for stable coronary disease. N Engl J Med 2007; 356: 1503-1516.
    • (2007) N Engl J Med , vol.356 , pp. 1503-1516
    • Boden, W.E.1    O'Rourke, R.A.2    Teo, K.K.3
  • 36
    • 0035879641 scopus 로고    scopus 로고
    • Group sequential test strategies for superiority and non-inferiority hypotheses in active controlled clinical trials
    • Wang SJ, Hung HMJ, Tsong Y, et al. Group sequential test strategies for superiority and non-inferiority hypotheses in active controlled clinical trials. Stat Med 2001; 20: 1903-1912.
    • (2001) Stat Med , vol.20 , pp. 1903-1912
    • Wang, S.J.1    Hung, H.M.J.2    Tsong, Y.3
  • 37
    • 0037366620 scopus 로고    scopus 로고
    • Sequential tests for non-inferiority and superiority
    • Brannath W, Bauer P, Maurer W, et al. Sequential tests for non-inferiority and superiority. Biometrics 2003; 59: 106-114.
    • (2003) Biometrics , vol.59 , pp. 106-114
    • Brannath, W.1    Bauer, P.2    Maurer, W.3
  • 38
    • 23244448022 scopus 로고    scopus 로고
    • A framework for two-stage adaptive procedures to simultaneously test non-inferiority and superiority
    • Koyama T, Sampson AR, Gleser LJ,. A framework for two-stage adaptive procedures to simultaneously test non-inferiority and superiority. Stat Med 2005; 24: 2439-2456.
    • (2005) Stat Med , vol.24 , pp. 2439-2456
    • Koyama, T.1    Sampson, A.R.2    Gleser, L.J.3
  • 39
    • 77949409796 scopus 로고    scopus 로고
    • Optimal group-sequential designs for simultaneous testing of superiority and non-inferiority
    • Öhrn F, Jennison C,. Optimal group-sequential designs for simultaneous testing of superiority and non-inferiority. Stat Med 2010; 29: 743-759.
    • (2010) Stat Med , vol.29 , pp. 743-759
    • Öhrn, F.1    Jennison, C.2
  • 40
    • 22044446634 scopus 로고    scopus 로고
    • Meta-analyses and adaptive group sequential designs in the clinical development process
    • Jennison C, Turnbull BW,. Meta-analyses and adaptive group sequential designs in the clinical development process. J Biopharm Stat 2005; 15: 537-558.
    • (2005) J Biopharm Stat , vol.15 , pp. 537-558
    • Jennison, C.1    Turnbull, B.W.2


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