메뉴 건너뛰기




Volumn 16, Issue 1, 2018, Pages

Adaptive designs in clinical trials: Why use them, and how to run and report them

(16)  Pallmann, Philip a   Bedding, Alun W b   Choodari Oskooei, Babak c   Dimairo, Munyaradzi d   Flight, Laura e   Hampson, Lisa V a,f   Holmes, Jane g   Mander, Adrian P h   Odondi, Lang'o g   Sydes, Matthew R c   Villar, Sofía S h   Wason, James M S h,i   Weir, Christopher J j   Wheeler, Graham M c,h   Yap, Christina k   Jaki, Thomas a  


Author keywords

Adaptive design; Design modification; Flexible design; Interim analysis; Seamless design; Statistical methods

Indexed keywords

ADULT; CLINICAL RESEARCH; FEMALE; FUNDING; HUMAN; HUMAN EXPERIMENT; LETTER; MALE; MONEY; NOMENCLATURE; REPRODUCIBILITY; STATISTICAL ANALYSIS; STUDY DESIGN; CLINICAL TRIAL (TOPIC); METHODOLOGY; PROCEDURES; STANDARDS;

EID: 85042559447     PISSN: None     EISSN: 17417015     Source Type: Journal    
DOI: 10.1186/s12916-018-1017-7     Document Type: Letter
Times cited : (491)

References (154)
  • 2
    • 33748560823 scopus 로고    scopus 로고
    • Plan to be flexible: a commentary on adaptive designs
    • Shih WJ. Plan to be flexible: a commentary on adaptive designs. Biometrical J. 2006; 48:656-9.
    • (2006) Biometrical J , vol.48 , pp. 656-659
    • Shih, W.J.1
  • 3
    • 85042556950 scopus 로고    scopus 로고
    • What is adaptive design?
    • Accessed 7 Jul 2017.
    • Berry Consultants. What is adaptive design? 2016. http://www.berryconsultants.com/adaptive-designs. Accessed 7 Jul 2017.
    • (2016)
  • 4
    • 84889662412 scopus 로고    scopus 로고
    • Similarities and differences of Bayesian designs and adaptive designs for medical devices: a regulatory view
    • Campbell G. Similarities and differences of Bayesian designs and adaptive designs for medical devices: a regulatory view. Stat Biopharm Res. 2013; 5:356-68.
    • (2013) Stat Biopharm Res , vol.5 , pp. 356-368
    • Campbell, G.1
  • 6
    • 0348149055 scopus 로고    scopus 로고
    • Sample size re-estimation in group-sequential response-adaptive clinical trials
    • Morgan CC. Sample size re-estimation in group-sequential response-adaptive clinical trials. Stat Med. 2003; 22:3843-57.
    • (2003) Stat Med , vol.22 , pp. 3843-3857
    • Morgan, C.C.1
  • 8
    • 36248945137 scopus 로고    scopus 로고
    • Recent developments in adaptive designs for phase I/II dose-finding studies
    • Zohar S, Chevret S. Recent developments in adaptive designs for phase I/II dose-finding studies. J Biopharm Stat. 2007; 17:1071-83.
    • (2007) J Biopharm Stat , vol.17 , pp. 1071-1083
    • Zohar, S.1    Chevret, S.2
  • 9
    • 84910057278 scopus 로고    scopus 로고
    • Novel statistical designs for phase I/II and phase II clinical trials with dose-finding objectives
    • Sverdlov O, Wong WK. Novel statistical designs for phase I/II and phase II clinical trials with dose-finding objectives. Ther Innov Regul Sci. 2014; 48:601-12.
    • (2014) Ther Innov Regul Sci , vol.48 , pp. 601-612
    • Sverdlov, O.1    Wong, W.K.2
  • 10
    • 33750854113 scopus 로고    scopus 로고
    • Adaptive seamless phase II/III designs-background, operational aspects, and examples
    • Maca J, Bhattacharya S, Dragalin V, Gallo P, Krams M. Adaptive seamless phase II/III designs-background, operational aspects, and examples. Drug Inf J. 2006; 40:463-73.
    • (2006) Drug Inf J , vol.40 , pp. 463-473
    • Maca, J.1    Bhattacharya, S.2    Dragalin, V.3    Gallo, P.4    Krams, M.5
  • 11
  • 12
    • 22044434097 scopus 로고    scopus 로고
    • Statistical consideration of adaptive methods in clinical development
    • Chow SC, Chang M, Pong A. Statistical consideration of adaptive methods in clinical development. J Biopharm Stat. 2005; 15:575-91.
    • (2005) J Biopharm Stat , vol.15 , pp. 575-591
    • Chow, S.C.1    Chang, M.2    Pong, A.3
  • 13
    • 43649105816 scopus 로고    scopus 로고
    • Maintaining confidentiality of interim data to enhance trial integrity and credibility
    • Fleming TR, Sharples K, McCall J, Moore A, Rodgers A, Stewart R. Maintaining confidentiality of interim data to enhance trial integrity and credibility. Clin Trials. 2008; 5:157-67.
    • (2008) Clin Trials , vol.5 , pp. 157-167
    • Fleming, T.R.1    Sharples, K.2    McCall, J.3    Moore, A.4    Rodgers, A.5    Stewart, R.6
  • 15
    • 78249253755 scopus 로고    scopus 로고
    • Type I error rate control in adaptive designs for confirmatory clinical trials with treatment selection at interim
    • Posch M, Maurer W, Bretz F. Type I error rate control in adaptive designs for confirmatory clinical trials with treatment selection at interim. Pharm Stat. 2011; 10:96-104.
    • (2011) Pharm Stat , vol.10 , pp. 96-104
    • Posch, M.1    Maurer, W.2    Bretz, F.3
  • 16
    • 79957950338 scopus 로고    scopus 로고
    • Maximum inflation of the type 1 error rate when sample size and allocation rate are adapted in a pre-planned interim look
    • Graf AC, Bauer P. Maximum inflation of the type 1 error rate when sample size and allocation rate are adapted in a pre-planned interim look. Stat Med. 2011; 30:1637-47.
    • (2011) Stat Med , vol.30 , pp. 1637-1647
    • Graf, A.C.1    Bauer, P.2
  • 17
    • 84903637900 scopus 로고    scopus 로고
    • Maximum type 1 error rate inflation in multiarmed clinical trials with adaptive interim sample size modifications
    • Graf AC, Bauer P, Glimm E, Koenig F. Maximum type 1 error rate inflation in multiarmed clinical trials with adaptive interim sample size modifications. Biometrical J. 2014; 56:614-30.
    • (2014) Biometrical J , vol.56 , pp. 614-630
    • Graf, A.C.1    Bauer, P.2    Glimm, E.3    Koenig, F.4
  • 18
    • 84890421989 scopus 로고    scopus 로고
    • Simultaneous confidence intervals that are compatible with closed testing in adaptive designs
    • Magirr D, Jaki T, Posch M, Klinglmueller F. Simultaneous confidence intervals that are compatible with closed testing in adaptive designs. Biometrika. 2013; 100:985-96.
    • (2013) Biometrika , vol.100 , pp. 985-996
    • Magirr, D.1    Jaki, T.2    Posch, M.3    Klinglmueller, F.4
  • 19
    • 84891382810 scopus 로고    scopus 로고
    • A comparison of methods for constructing confidence intervals after phase II/III clinical trials
    • Kimani PK, Todd S, Stallard N. A comparison of methods for constructing confidence intervals after phase II/III clinical trials. Biometrical J. 2014; 56:107-28.
    • (2014) Biometrical J , vol.56 , pp. 107-128
    • Kimani, P.K.1    Todd, S.2    Stallard, N.3
  • 20
    • 84864278404 scopus 로고    scopus 로고
    • The practical application of adaptive study design in early phase clinical trials: a retrospective analysis of time savings
    • Lorch U, Berelowitz K, Ozen C, Naseem A, Akuffo E, Taubel J. The practical application of adaptive study design in early phase clinical trials: a retrospective analysis of time savings. Eur J Clin Pharmacol. 2012; 68:543-51.
    • (2012) Eur J Clin Pharmacol , vol.68 , pp. 543-551
    • Lorch, U.1    Berelowitz, K.2    Ozen, C.3    Naseem, A.4    Akuffo, E.5    Taubel, J.6
  • 21
    • 84954167098 scopus 로고    scopus 로고
    • Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls
    • Bauer P, Bretz F, Dragalin V, König F, Wassmer G. Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Stat Med. 2016; 35:325-47.
    • (2016) Stat Med , vol.35 , pp. 325-347
    • Bauer, P.1    Bretz, F.2    Dragalin, V.3    König, F.4    Wassmer, G.5
  • 22
    • 66849118694 scopus 로고    scopus 로고
    • Dose escalation methods in phase I cancer clinical trials
    • Le Tourneau C, Lee JJ, Siu LL. Dose escalation methods in phase I cancer clinical trials. J Natl Cancer Inst. 2009; 101:708-20.
    • (2009) J Natl Cancer Inst , vol.101 , pp. 708-720
    • Lee, J.J.1    Siu, L.L.2
  • 23
    • 84861200623 scopus 로고    scopus 로고
    • Bayesian adaptive clinical trials: a dream for statisticians only?
    • Chevret S. Bayesian adaptive clinical trials: a dream for statisticians only?Stat Med. 2012; 31:1002-13.
    • (2012) Stat Med , vol.31 , pp. 1002-1013
    • Chevret, S.1
  • 24
    • 84876557281 scopus 로고    scopus 로고
    • Uptake of novel statistical methods for early-phase clinical studies in the UK public sector
    • Jaki T. Uptake of novel statistical methods for early-phase clinical studies in the UK public sector. Clin Trials. 2013; 10:344-6.
    • (2013) Clin Trials , vol.10 , pp. 344-346
    • Jaki, T.1
  • 26
    • 84942308496 scopus 로고    scopus 로고
    • Missing steps in a staircase: a qualitative study of the perspectives of key stakeholders on the use of adaptive designs in confirmatory trials
    • Dimairo M, Boote J, Julious SA, Nicholl JP, Todd S. Missing steps in a staircase: a qualitative study of the perspectives of key stakeholders on the use of adaptive designs in confirmatory trials. Trials. 2015; 16:430.
    • (2015) Trials , vol.16 , pp. 430
    • Dimairo, M.1    Boote, J.2    Julious, S.A.3    Nicholl, J.P.4    Todd, S.5
  • 27
    • 84951808541 scopus 로고    scopus 로고
    • Cross-sector surveys assessing perceptions of key stakeholders towards barriers, concerns and facilitators to the appropriate use of adaptive designs in confirmatory trials
    • Dimairo M, Julious SA, Todd S, Nicholl JP, Boote J. Cross-sector surveys assessing perceptions of key stakeholders towards barriers, concerns and facilitators to the appropriate use of adaptive designs in confirmatory trials. Trials. 2015; 16:585.
    • (2015) Trials , vol.16 , pp. 585
    • Dimairo, M.1    Julious, S.A.2    Todd, S.3    Nicholl, J.P.4    Boote, J.5
  • 28
    • 84961201800 scopus 로고    scopus 로고
    • Adaptive designs undertaken in clinical research: a review of registered clinical trials
    • Hatfield I, Allison A, Flight L, Julious SA, Dimairo M. Adaptive designs undertaken in clinical research: a review of registered clinical trials. Trials. 2016; 17:150.
    • (2016) Trials , vol.17 , pp. 150
    • Hatfield, I.1    Allison, A.2    Flight, L.3    Julious, S.A.4    Dimairo, M.5
  • 29
    • 84979680959 scopus 로고    scopus 로고
    • Attitudes and opinions regarding confirmatory adaptive clinical trials: a mixed methods analysis from the Adaptive Designs Accelerating Promising Trials into Treatments (ADAPT-IT) project
    • Meurer WJ, Legocki L, Mawocha S, Frederiksen SM, Guetterman TC, Barsan W, et al.Attitudes and opinions regarding confirmatory adaptive clinical trials: a mixed methods analysis from the Adaptive Designs Accelerating Promising Trials into Treatments (ADAPT-IT) project. Trials. 2016; 17:373.
    • (2016) Trials , vol.17 , pp. 373
    • Meurer, W.J.1    Legocki, L.2    Mawocha, S.3    Frederiksen, S.M.4    Guetterman, T.C.5    Barsan, W.6
  • 30
    • 0348129532 scopus 로고    scopus 로고
    • Effects of ranolazine with atenolol, amlodipine, or diltiazem on exercise tolerance and angina frequency in patients with severe chronic angina: a randomized controlled trial
    • Chaitman BR, Pepine CJ, Parker JO, Skopal J, Chumakova G, Kuch J, et al.Effects of ranolazine with atenolol, amlodipine, or diltiazem on exercise tolerance and angina frequency in patients with severe chronic angina: a randomized controlled trial. J Am Med Assoc. 2004; 291:309-16.
    • (2004) J Am Med Assoc , vol.291 , pp. 309-316
    • Chaitman, B.R.1    Pepine, C.J.2    Parker, J.O.3    Skopal, J.4    Chumakova, G.5    Kuch, J.6
  • 31
    • 84945584299 scopus 로고    scopus 로고
    • Telmisartan and insulin resistance in HIV (TAILoR): protocol for a dose-ranging phase II randomised open-labelled trial of telmisartan as a strategy for the reduction of insulin resistance in HIV-positive individuals on combination antiretroviral therapy
    • Pushpakom SP, Taylor C, Kolamunnage-Dona R, Spowart C, Vora J, García-Fiñana M, et al. Telmisartan and insulin resistance in HIV (TAILoR): protocol for a dose-ranging phase II randomised open-labelled trial of telmisartan as a strategy for the reduction of insulin resistance in HIV-positive individuals on combination antiretroviral therapy. BMJ Open. 2015; 5:e009566.
    • (2015) BMJ Open , vol.5
    • Pushpakom, S.P.1    Taylor, C.2    Kolamunnage-Dona, R.3    Spowart, C.4    García-Fiñana, M.5
  • 32
    • 84861646016 scopus 로고    scopus 로고
    • A generalized Dunnett test for multi-arm multi-stage clinical studies with treatment selection
    • Magirr D, Jaki T, Whitehead J. A generalized Dunnett test for multi-arm multi-stage clinical studies with treatment selection. Biometrika. 2012; 99:494-501.
    • (2012) Biometrika , vol.99 , pp. 494-501
    • Magirr, D.1    Jaki, T.2    Whitehead, J.3
  • 33
    • 0038811777 scopus 로고    scopus 로고
    • Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia
    • Giles FJ, Kantarjian HM, Cortes JE, Garcia-Manero G, Verstovsek S, Faderl S, et al.Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia. J Clin Oncol. 2003; 21:1722-7.
    • (2003) J Clin Oncol , vol.21 , pp. 1722-1727
    • Giles, F.J.1    Kantarjian, H.M.2    Cortes, J.E.3    Garcia-Manero, G.4    Verstovsek, S.5    Faderl, S.6
  • 34
    • 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; 30:3267-84.
    • (2011) Stat Med , vol.30 , pp. 3267-3284
    • Mehta, C.R.1    Pocock, S.J.2
  • 35
    • 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; 34:3793-810.
    • (2015) Stat Med , vol.34 , pp. 3793-3810
    • Jennison, C.1    Turnbull, B.W.2
  • 36
    • 84891827000 scopus 로고    scopus 로고
    • Empirical Bayes estimation of the selected treatment mean for two-stage drop-the-loser trials: a meta-analytic approach
    • Bowden J, Brannath W, Glimm E. Empirical Bayes estimation of the selected treatment mean for two-stage drop-the-loser trials: a meta-analytic approach. Stat Med. 2014; 33:388-400.
    • (2014) Stat Med , vol.33 , pp. 388-400
    • Bowden, J.1    Brannath, W.2    Glimm, E.3
  • 37
    • 85006010367 scopus 로고    scopus 로고
    • Developing a Bayesian adaptive design for a phase I clinical trial: a case study for a novel HIV treatment
    • Mason AJ, Gonzalez-Maffe J, Quinn K, Doyle N, Legg K, Norsworthy P, et al.Developing a Bayesian adaptive design for a phase I clinical trial: a case study for a novel HIV treatment. Stat Med. 2017; 36:754-71.
    • (2017) Stat Med , vol.36 , pp. 754-771
    • Mason, A.J.1    Gonzalez-Maffe, J.2    Quinn, K.3    Doyle, N.4    Legg, K.5    Norsworthy, P.6
  • 38
    • 85042561918 scopus 로고    scopus 로고
    • Accessed 7 Jul 2017.
    • Wellcome Trust. Joint Global Health Trials scheme. 2017. https://wellcome.ac.uk/funding/joint-global-health-trials-scheme. Accessed 7 Jul 2017.
    • (2017) Joint Global Health Trials scheme
  • 39
    • 85042556617 scopus 로고    scopus 로고
    • Accessed 7 Jul 2017.
    • National Institutes of Health. NIH Planning Grant Program (R34). 2014. https://grants.nih.gov/grants/funding/r34.htm. Accessed 7 Jul 2017.
    • (2014) NIH Planning Grant Program (R34)
  • 40
    • 70449645866 scopus 로고    scopus 로고
    • Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design
    • Accessed 7 Jul 2017.
    • European Medicines Agency. Reflection paper on methodological issues in confirmatory clinical trials planned with an adaptive design. 2007. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003616.pdf. Accessed 7 Jul 2017.
    • (2007)
  • 41
    • 78249234519 scopus 로고    scopus 로고
    • Adaptive design clinical trials for drugs and biologics: guidance for industry (draft)
    • Accessed 7 Jul 2017.
    • US Food & Drug Administration. Adaptive design clinical trials for drugs and biologics: guidance for industry (draft). 2010. https://www.fda.gov/downloads/drugs/guidances/ucm201790.pdf. Accessed 7 Jul 2017.
    • (2010)
  • 42
    • 85021428107 scopus 로고    scopus 로고
    • Adaptive designs for medical device clinical studies: guidance for industry and Food and Drug Administration staff
    • Accessed 7 Jul 2017.
    • Food US & Drug Administration. Adaptive designs for medical device clinical studies: guidance for industry and Food and Drug Administration staff.2016. https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm446729.pdf. Accessed 7 Jul 2017.
    • (2016)
  • 43
    • 84891486765 scopus 로고    scopus 로고
    • Perspective on adaptive designs: 4 years European Medicines Agency reflection paper, 1 year draft US FDA guidance-where are we now?
    • Gaydos B, Koch A, Miller F, Posch M, Vandemeulebroecke M, Wang SJ. Perspective on adaptive designs: 4 years European Medicines Agency reflection paper, 1 year draft US FDA guidance-where are we now?Clin Investig. 2012; 2:235-40.
    • (2012) Clin Investig , vol.2 , pp. 235-240
    • Gaydos, B.1    Koch, A.2    Miller, F.3    Posch, M.4    Vandemeulebroecke, M.5    Wang, S.J.6
  • 44
    • 84907946289 scopus 로고    scopus 로고
    • Adaptive clinical trial designs for European marketing authorization: a survey of scientific advice letters from the European Medicines Agency
    • Elsäßer A, Regnstrom J, Vetter T, Koenig F, Hemmings RJ, Greco M, et al.Adaptive clinical trial designs for European marketing authorization: a survey of scientific advice letters from the European Medicines Agency. Trials. 2014; 15:383.
    • (2014) Trials , vol.15 , pp. 383
    • Elsäßer, A.1    Regnstrom, J.2    Vetter, T.3    Koenig, F.4    Hemmings, R.J.5    Greco, M.6
  • 45
    • 2442667708 scopus 로고    scopus 로고
    • The independent statistician for data monitoring committees
    • DeMets DL, Fleming TR. The independent statistician for data monitoring committees. Stat Med. 2004; 23:1513-17.
    • (2004) Stat Med , vol.23 , pp. 1513-1517
    • DeMets, D.L.1    Fleming, T.R.2
  • 46
    • 33745175516 scopus 로고    scopus 로고
    • Operational challenges in adaptive design implementation
    • Gallo P. Operational challenges in adaptive design implementation. Pharm Stat. 2006; 5:119-24.
    • (2006) Pharm Stat , vol.5 , pp. 119-124
    • Gallo, P.1
  • 47
    • 13844318810 scopus 로고    scopus 로고
    • A proposed charter for clinical trial data monitoring committees: helping them to do their job well
    • Grant AM, Altman DG, Babiker AG, Campbell MK, Clemens F, Darbyshire JH, et al.A proposed charter for clinical trial data monitoring committees: helping them to do their job well. Lancet. 2005; 365:711-22.
    • (2005) Lancet , vol.365 , pp. 711-722
    • Grant, A.M.1    Altman, D.G.2    Babiker, A.G.3    Campbell, M.K.4    Clemens, F.5    Darbyshire, J.H.6
  • 50
    • 84990043625 scopus 로고    scopus 로고
    • Data monitoring committees-expect the unexpected
    • DeMets DL, Ellenberg SS. Data monitoring committees-expect the unexpected. N Engl J Med. 2016; 375:1365-71.
    • (2016) N Engl J Med , vol.375 , pp. 1365-1371
    • DeMets, D.L.1    Ellenberg, S.S.2
  • 51
    • 85026442641 scopus 로고    scopus 로고
    • Recommendations for data monitoring committees from the Clinical Trials Transformation Initiative
    • Calis KA, Archdeacon P, Bain R, DeMets D, Donohue M, Elzarrad MK, et al.Recommendations for data monitoring committees from the Clinical Trials Transformation Initiative. Clin Trials. 2017; 14:342-8.
    • (2017) Clin Trials , vol.14 , pp. 342-348
    • Calis, K.A.1    Archdeacon, P.2    Bain, R.3    DeMets, D.4    Donohue, M.5    Elzarrad, M.K.6
  • 52
    • 84947076569 scopus 로고    scopus 로고
    • Trial steering committees in randomised controlled trials: a survey of registered clinical trials units to establish current practice and experiences
    • Conroy EJ, Harman NL, Lane JA, Lewis SC, Murray G, Norrie J, et al.Trial steering committees in randomised controlled trials: a survey of registered clinical trials units to establish current practice and experiences. Clin Trials. 2015; 12:664-76.
    • (2015) Clin Trials , vol.12 , pp. 664-676
    • Conroy, E.J.1    Harman, N.L.2    Lane, J.A.3    Lewis, S.C.4    Murray, G.5    Norrie, J.6
  • 53
    • 84953379166 scopus 로고    scopus 로고
    • Exploring the role and function of trial steering committees: results of an expert panel meeting
    • Harman NL, Conroy EJ, Lewis SC, Murray G, Norrie J, Sydes MR, et al.Exploring the role and function of trial steering committees: results of an expert panel meeting. Trials. 2015; 16:597.
    • (2015) Trials , vol.16 , pp. 597
    • Harman, N.L.1    Conroy, E.J.2    Lewis, S.C.3    Murray, G.4    Norrie, J.5    Sydes, M.R.6
  • 54
    • 84977073551 scopus 로고    scopus 로고
    • What are the roles and valued attributes of a trial steering committee? Ethnographic study of eight clinical trials facing challenges
    • Daykin A, Selman LE, Cramer H, McCann S, Shorter GW, Sydes MR, et al.What are the roles and valued attributes of a trial steering committee? Ethnographic study of eight clinical trials facing challenges. Trials. 2016; 17:307.
    • (2016) Trials , vol.17 , pp. 307
    • Daykin, A.1    Selman, L.E.2    Cramer, H.3    McCann, S.4    Shorter, G.W.5    Sydes, M.R.6
  • 55
    • 85009444691 scopus 로고    scopus 로고
    • Addressing challenges and opportunities of 'less well-understood' adaptive designs
    • He W, Gallo P, Miller E, Jemiai Y, Maca J, Koury K, et al.Addressing challenges and opportunities of 'less well-understood' adaptive designs. Ther Innov Regul Sci. 2017; 51:60-8.
    • (2017) Ther Innov Regul Sci , vol.51 , pp. 60-68
    • He, W.1    Gallo, P.2    Miller, E.3    Jemiai, Y.4    Maca, J.5    Koury, K.6
  • 56
    • 79960950486 scopus 로고    scopus 로고
    • Group sequential methods and software applications
    • Zhu L, Ni L, Yao B. Group sequential methods and software applications. Am Stat. 2011; 65:127-35.
    • (2011) Am Stat , vol.65 , pp. 127-135
    • Zhu, L.1    Ni, L.2    Yao, B.3
  • 57
    • 84954366072 scopus 로고    scopus 로고
    • A review of available software and capabilities for adaptive designs
    • In: He W, Pinheiro J, Kuznetsova OM, editors. Practical considerations for adaptive trial design and implementation. New York: Springer
    • Tymofyeyev Y. A review of available software and capabilities for adaptive designs In: He W, Pinheiro J, Kuznetsova OM, editors. Practical considerations for adaptive trial design and implementation. New York: Springer: 2014. p. 139-55.
    • (2014) , pp. 139-155
    • Tymofyeyev, Y.1
  • 58
    • 33646245937 scopus 로고    scopus 로고
    • Adaptive designs in clinical drug development-an executive summary of the PhRMA Working Group
    • Gallo P, Chuang-Stein C, Dragalin V, Gaydos B, Krams M, Pinheiro J. Adaptive designs in clinical drug development-an executive summary of the PhRMA Working Group. J Biopharm Stat. 2006; 16:275-83.
    • (2006) J Biopharm Stat , vol.16 , pp. 275-283
    • Gallo, P.1    Chuang-Stein, C.2    Dragalin, V.3    Gaydos, B.4    Krams, M.5    Pinheiro, J.6
  • 59
    • 33750893179 scopus 로고    scopus 로고
    • Implementing adaptive designs: logistical and operational considerations
    • Quinlan J, Krams M. Implementing adaptive designs: logistical and operational considerations. Drug Inf J. 2006; 40:437-44.
    • (2006) Drug Inf J , vol.40 , pp. 437-444
    • Quinlan, J.1    Krams, M.2
  • 60
    • 44949171503 scopus 로고    scopus 로고
    • Adaptive design methods in clinical trials-a review
    • Chow SC, Chang M. Adaptive design methods in clinical trials-a review. Orphanet J Rare Dis. 2008; 3:11.
    • (2008) Orphanet J Rare Dis , vol.3 , pp. 11
    • Chow, S.C.1    Chang, M.2
  • 61
  • 62
    • 77953666447 scopus 로고    scopus 로고
    • Barriers and opportunities for implementation of adaptive designs in pharmaceutical product development
    • Quinlan J, Gaydos B, Maca J, Krams M. Barriers and opportunities for implementation of adaptive designs in pharmaceutical product development. Clin Trials. 2010; 7:167-73.
    • (2010) Clin Trials , vol.7 , pp. 167-173
    • Quinlan, J.1    Gaydos, B.2    Maca, J.3    Krams, M.4
  • 64
    • 84987938831 scopus 로고    scopus 로고
    • Practical considerations for adaptive trial design and implementation
    • New York: Springer
    • He W, Pinheiro J, Kuznetsova OM. Practical considerations for adaptive trial design and implementation. New York: Springer; 2014.
    • (2014)
    • He, W.1    Pinheiro, J.2    Kuznetsova, O.M.3
  • 65
    • 85021098083 scopus 로고    scopus 로고
    • The role of adaptive trial designs in drug development
    • Curtin F, Heritier S. The role of adaptive trial designs in drug development. Expert Rev Clin Pharmacol. 2017; 10:727-36.
    • (2017) Expert Rev Clin Pharmacol , vol.10 , pp. 727-736
    • Curtin, F.1    Heritier, S.2
  • 66
    • 84959515785 scopus 로고    scopus 로고
    • Implementation of adaptive methods in early-phase clinical trials
    • Petroni GR, Wages NA, Paux G, Dubois F. Implementation of adaptive methods in early-phase clinical trials. Stat Med. 2017; 36:215-24.
    • (2017) Stat Med , vol.36 , pp. 215-224
    • Petroni, G.R.1    Wages, N.A.2    Paux, G.3    Dubois, F.4
  • 67
    • 67650216185 scopus 로고    scopus 로고
    • Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial
    • Sydes MR, Parmar MKB, James ND, Clarke NW, Dearnaley DP, Mason MD, et al.Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial. Trials. 2009; 10:39.
    • (2009) Trials , vol.10 , pp. 39
    • Sydes, M.R.1    Parmar, M.K.B.2    James, N.D.3    Clarke, N.W.4    Dearnaley, D.P.5    Mason, M.D.6
  • 68
    • 84883027307 scopus 로고    scopus 로고
    • Operational challenges and solutions with implementation of an adaptive seamless phase 2/3 study
    • Spencer K, Colvin K, Braunecker B, Brackman M, Ripley J, Hines P, et al.Operational challenges and solutions with implementation of an adaptive seamless phase 2/3 study. J Diabetes Sci Technol. 2012; 6:1296-304.
    • (2012) J Diabetes Sci Technol , vol.6 , pp. 1296-1304
    • Spencer, K.1    Colvin, K.2    Braunecker, B.3    Brackman, M.4    Ripley, J.5    Hines, P.6
  • 69
    • 85009465989 scopus 로고    scopus 로고
    • DIA's Adaptive Design Scientific Working Group (ADSWG): best practices case studies for 'less well-understood' adaptive designs
    • Miller E, Gallo P, He W, Kammerman LA, Koury K, Maca J, et al.DIA's Adaptive Design Scientific Working Group (ADSWG): best practices case studies for 'less well-understood' adaptive designs. Ther Innov Regul Sci. 2017; 51:77-88.
    • (2017) Ther Innov Regul Sci , vol.51 , pp. 77-88
    • Miller, E.1    Gallo, P.2    He, W.3    Kammerman, L.A.4    Koury, K.5    Maca, J.6
  • 70
    • 33748560148 scopus 로고    scopus 로고
    • An overview of statistical approaches for adaptive designs and design modifications
    • Schäfer H, Timmesfeld N, Müller HH. An overview of statistical approaches for adaptive designs and design modifications. Biom J. 2006; 48:507-20.
    • (2006) Biom J , vol.48 , pp. 507-520
    • Schäfer, H.1    Timmesfeld, N.2    Müller, H.H.3
  • 73
    • 85042565901 scopus 로고    scopus 로고
    • In: Group sequential methods with applications to clinical trials. Boca Raton: Chapman & Hall/CRC
    • Jennison C, Turnbull BW. Analysis following a sequential test. In: Group sequential methods with applications to clinical trials. Boca Raton: Chapman & Hall/CRC: 2000. p. 171-87.
    • (2000) Analysis following a sequential test , pp. 171-187
    • Jennison, C.1    Turnbull, B.W.2
  • 74
    • 0001136460 scopus 로고
    • Parameter estimation following group sequential hypothesis testing
    • Emerson SS, Fleming TR. Parameter estimation following group sequential hypothesis testing. Biometrika. 1990; 77:875-92.
    • (1990) Biometrika , vol.77 , pp. 875-892
    • Emerson, S.S.1    Fleming, T.R.2
  • 75
    • 0000949287 scopus 로고    scopus 로고
    • Unbiased estimation following a group sequential test
    • Liu A, Hall WJ. Unbiased estimation following a group sequential test. Biometrika. 1999; 86:71-8.
    • (1999) Biometrika , vol.86 , pp. 71-78
    • Liu, A.1    Hall, W.J.2
  • 76
    • 54349104178 scopus 로고    scopus 로고
    • Unbiased estimation of selected treatment means in two-stage trials
    • Bowden J, Glimm E. Unbiased estimation of selected treatment means in two-stage trials. Biometrical J. 2008; 50:515-27.
    • (2008) Biometrical J , vol.50 , pp. 515-527
    • Bowden, J.1    Glimm, E.2
  • 77
    • 84894522079 scopus 로고    scopus 로고
    • Conditionally unbiased and near unbiased estimation of the selected treatment mean for multistage drop-the-losers trials
    • Bowden J, Glimm E. Conditionally unbiased and near unbiased estimation of the selected treatment mean for multistage drop-the-losers trials. Biometrical J. 2014; 56:332-49.
    • (2014) Biometrical J , vol.56 , pp. 332-349
    • Bowden, J.1    Glimm, E.2
  • 78
    • 0001547530 scopus 로고
    • On the bias of maximum likelihood estimation following a sequential test
    • Whitehead J. On the bias of maximum likelihood estimation following a sequential test. Biometrika. 1986; 73:573-81.
    • (1986) Biometrika , vol.73 , pp. 573-581
    • Whitehead, J.1
  • 79
    • 78650239326 scopus 로고    scopus 로고
    • An exact method for analysis following a two-stage phase II cancer clinical trial
    • Jovic G, Whitehead J. An exact method for analysis following a two-stage phase II cancer clinical trial. Stat Med. 2010; 29:3118-25.
    • (2010) Stat Med , vol.29 , pp. 3118-3125
    • Jovic, G.1    Whitehead, J.2
  • 80
    • 84876448156 scopus 로고    scopus 로고
    • Shrinkage estimation in two-stage adaptive designs with midtrial treatment selection
    • Carreras M, Brannath W. Shrinkage estimation in two-stage adaptive designs with midtrial treatment selection. Stat Med. 2013; 32:1677-90.
    • (2013) Stat Med , vol.32 , pp. 1677-1690
    • Carreras, M.1    Brannath, W.2
  • 81
    • 85020407913 scopus 로고    scopus 로고
    • Estimation in multi-arm two-stage trials with treatment selection and time-to-event endpoint
    • Brueckner M, Titman A, Jaki T. Estimation in multi-arm two-stage trials with treatment selection and time-to-event endpoint. Stat Med. 2017; 36:3137-53.
    • (2017) Stat Med , vol.36 , pp. 3137-3153
    • Brueckner, M.1    Titman, A.2    Jaki, T.3
  • 82
    • 84870060247 scopus 로고    scopus 로고
    • Identifying combined design and analysis procedures in two-stage trials with a binary end point
    • Bowden J, Wason J. Identifying combined design and analysis procedures in two-stage trials with a binary end point. Stat Med. 2012; 31:3874-84.
    • (2012) Stat Med , vol.31 , pp. 3874-3884
    • Bowden, J.1    Wason, J.2
  • 83
    • 84872592627 scopus 로고    scopus 로고
    • Impact of lack-of-benefit stopping rules on treatment effect estimates of two-arm multi-stage (TAMS) trials with time to event outcome
    • Choodari-Oskooei B, Parmar MK, Royston P, Bowden J. Impact of lack-of-benefit stopping rules on treatment effect estimates of two-arm multi-stage (TAMS) trials with time to event outcome. Trials. 2013; 14:23.
    • (2013) Trials , vol.14 , pp. 23
    • Choodari-Oskooei, B.1    Parmar, M.K.2    Royston, P.3    Bowden, J.4
  • 85
    • 84884703868 scopus 로고    scopus 로고
    • Exact inference for adaptive group sequential designs
    • Gao P, Liu L, Mehta C. Exact inference for adaptive group sequential designs. Stat Med. 2013; 32:3991-4005.
    • (2013) Stat Med , vol.32 , pp. 3991-4005
    • Gao, P.1    Liu, L.2    Mehta, C.3
  • 86
    • 84935687765 scopus 로고    scopus 로고
    • Estimation after subpopulation selection in adaptive seamless trials
    • Kimani PK, Todd S, Stallard N. Estimation after subpopulation selection in adaptive seamless trials. Stat Med. 2015; 34:2581-601.
    • (2015) Stat Med , vol.34 , pp. 2581-2601
    • Kimani, P.K.1    Todd, S.2    Stallard, N.3
  • 88
    • 0029589385 scopus 로고
    • Designed extension of studies based on conditional power
    • Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics. 1995; 51:1315-24.
    • (1995) Biometrics , vol.51 , pp. 1315-1324
    • Proschan, M.A.1    Hunsberger, S.A.2
  • 89
    • 0345329168 scopus 로고    scopus 로고
    • Simple procedures for blinded sample size adjustment that do not affect the type I error rate
    • Kieser M, Friede T. Simple procedures for blinded sample size adjustment that do not affect the type I error rate. Stat Med. 2003; 22:3571-81.
    • (2003) Stat Med , vol.22 , pp. 3571-3581
    • Kieser, M.1    Friede, T.2
  • 90
    • 84952701035 scopus 로고    scopus 로고
    • Maximum type I error rate inflation from sample size reassessment when investigators are blind to treatment labels
    • zebrowska M, Posch M, Magirr D. Maximum type I error rate inflation from sample size reassessment when investigators are blind to treatment labels. Stat Med. 2016; 35:1972-84.
    • (2016) Stat Med , vol.35 , pp. 1972-1984
    • Zebrowska, M.1    Posch, M.2    Magirr, D.3
  • 91
    • 84976869630 scopus 로고    scopus 로고
    • Type I error rates of multi-arm multi-stage clinical trials: strong control and impact of intermediate outcomes
    • Bratton DJ, Parmar MKB, Phillips PPJ, Choodari-Oskooei B. Type I error rates of multi-arm multi-stage clinical trials: strong control and impact of intermediate outcomes. Trials. 2016; 17:309.
    • (2016) Trials , vol.17 , pp. 309
    • Bratton, D.J.1    Parmar, M.K.B.2    Phillips, P.P.J.3    Choodari-Oskooei, B.4
  • 92
    • 74749107462 scopus 로고    scopus 로고
    • Hierarchical testing of multiple endpoints in group-sequential trials
    • Glimm E, Maurer W, Bretz F. Hierarchical testing of multiple endpoints in group-sequential trials. Stat Med. 2010; 29:219-28.
    • (2010) Stat Med , vol.29 , pp. 219-228
    • Glimm, E.1    Maurer, W.2    Bretz, F.3
  • 93
    • 84874972543 scopus 로고    scopus 로고
    • A group sequential Holm procedure with multiple primary endpoints
    • Ye Y, Li A, Liu L, Yao B. A group sequential Holm procedure with multiple primary endpoints. Stat Med. 2013; 32:1112-24.
    • (2013) Stat Med , vol.32 , pp. 1112-1124
    • Ye, Y.1    Li, A.2    Liu, L.3    Yao, B.4
  • 94
    • 85042563633 scopus 로고    scopus 로고
    • In: Dmitrienko A, Tamhane AC, Bretz F, editors. Multiple testing problems in pharmaceutical statistics. Boca Raton: Chapman & Hall/CRC
    • Maurer W, Branson M, Posch M. Adaptive designs and confirmatory hypothesis testing In: Dmitrienko A, Tamhane AC, Bretz F, editors. Multiple testing problems in pharmaceutical statistics. Boca Raton: Chapman & Hall/CRC: 2010.
    • (2010) Adaptive designs and confirmatory hypothesis testing
    • Maurer, W.1    Branson, M.2    Posch, M.3
  • 95
    • 30944469129 scopus 로고    scopus 로고
    • Testing and estimation in flexible group sequential designs with adaptive treatment selection
    • Posch M, Koenig F, Branson M, Brannath W, Dunger-Baldauf C, Bauer P. Testing and estimation in flexible group sequential designs with adaptive treatment selection. Stat Med. 2005; 24:3697-714.
    • (2005) Stat Med , vol.24 , pp. 3697-3714
    • Posch, M.1    Koenig, F.2    Branson, M.3    Brannath, W.4    Dunger-Baldauf, C.5    Bauer, P.6
  • 96
    • 84910039155 scopus 로고    scopus 로고
    • Correcting for multiple-testing in multi-arm trials: is it necessary and is it done?
    • Wason JMS, Stecher L, Mander AP. Correcting for multiple-testing in multi-arm trials: is it necessary and is it done?Trials. 2014; 15:364.
    • (2014) Trials , vol.15 , pp. 364
    • Wason, J.M.S.1    Stecher, L.2    Mander, A.P.3
  • 97
    • 79957528017 scopus 로고    scopus 로고
    • Regulatory perspectives on multiplicity in adaptive design clinical trials throughout a drug development program
    • Wang SJ, Hung HMJ, O'Neill R. Regulatory perspectives on multiplicity in adaptive design clinical trials throughout a drug development program. J Biopharm Stat. 2011; 21:846-59.
    • (2011) J Biopharm Stat , vol.21 , pp. 846-859
    • Wang, S.J.1    Hung, H.M.J.2    O'Neill, R.3
  • 98
    • 85034804316 scopus 로고    scopus 로고
    • Accessed 7 Jul 2017.
    • European Medicines Agency. Guideline on multiplicity issues in clinical trials (draft). 2017. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2017/03/WC500224998.pdf. Accessed 7 Jul 2017.
    • (2017) Guideline on multiplicity issues in clinical trials (draft)
  • 103
    • 33847666453 scopus 로고    scopus 로고
    • Practical Bayesian adaptive randomisation in clinical trials
    • Thall PF, Wathen JK. Practical Bayesian adaptive randomisation in clinical trials. Eur J Cancer. 2007; 43:859-66.
    • (2007) Eur J Cancer , vol.43 , pp. 859-866
    • Thall, P.F.1    Wathen, J.K.2
  • 105
    • 84864019962 scopus 로고    scopus 로고
    • Practical guidelines for adaptive seamless phase II/III clinical trials that use Bayesian methods
    • Kimani PK, Glimm E, Maurer W, Hutton JL, Stallard N. Practical guidelines for adaptive seamless phase II/III clinical trials that use Bayesian methods. Stat Med. 2012; 31:2068-85.
    • (2012) Stat Med , vol.31 , pp. 2068-2085
    • Kimani, P.K.1    Glimm, E.2    Maurer, W.3    Hutton, J.L.4    Stallard, N.5
  • 106
    • 84960334957 scopus 로고    scopus 로고
    • An overview of the design and conduct of the BATTLE trials
    • Liu S, Lee JJ. An overview of the design and conduct of the BATTLE trials. Chin Clin Oncol. 2015; 4:33.
    • (2015) Chin Clin Oncol , vol.4 , pp. 33
    • Liu, S.1    Lee, J.J.2
  • 107
    • 24144464254 scopus 로고    scopus 로고
    • Bayesian adaptive designs for clinical trials
    • Cheng Y, Shen Y. Bayesian adaptive designs for clinical trials. Biometrika. 2005; 92:633-46.
    • (2005) Biometrika , vol.92 , pp. 633-646
    • Cheng, Y.1    Shen, Y.2
  • 108
    • 34250366338 scopus 로고    scopus 로고
    • Bayesian decision-theoretic group sequential clinical trial design based on a quadratic loss function: a frequentist evaluation
    • Lewis RJ, Lipsky AM, Berry DA. Bayesian decision-theoretic group sequential clinical trial design based on a quadratic loss function: a frequentist evaluation. Clin Trials. 2007; 4:5-14.
    • (2007) Clin Trials , vol.4 , pp. 5-14
    • Lewis, R.J.1    Lipsky, A.M.2    Berry, D.A.3
  • 109
    • 84926142505 scopus 로고    scopus 로고
    • Bayesian designs and the control of frequentist characteristics: a practical solution
    • Ventz S, Trippa L. Bayesian designs and the control of frequentist characteristics: a practical solution. Biometrics. 2015; 71:218-26.
    • (2015) Biometrics , vol.71 , pp. 218-226
    • Ventz, S.1    Trippa, L.2
  • 110
    • 36348967229 scopus 로고    scopus 로고
    • Frequentist evaluation of group sequential clinical trial designs
    • Emerson SS, Kittelson JM, Gillen DL. Frequentist evaluation of group sequential clinical trial designs. Stat Med. 2007; 26:5047-80.
    • (2007) Stat Med , vol.26 , pp. 5047-5080
    • Emerson, S.S.1    Kittelson, J.M.2    Gillen, D.L.3
  • 111
    • 33947102564 scopus 로고    scopus 로고
    • Bayesian evaluation of group sequential clinical trial designs
    • Emerson SS, Kittelson JM, Gillen DL. Bayesian evaluation of group sequential clinical trial designs. Stat Med. 2007; 26:1431-49.
    • (2007) Stat Med , vol.26 , pp. 1431-1449
    • Emerson, S.S.1    Kittelson, J.M.2    Gillen, D.L.3
  • 113
    • 21244466674 scopus 로고    scopus 로고
    • Decision-making in a phase II clinical trial: a new approach combining Bayesian and frequentist concepts
    • Stallard N, Whitehead J, Cleall S. Decision-making in a phase II clinical trial: a new approach combining Bayesian and frequentist concepts. Pharm Stat. 2005; 4:119-28.
    • (2005) Pharm Stat , vol.4 , pp. 119-128
    • Stallard, N.1    Whitehead, J.2    Cleall, S.3
  • 114
    • 84864020956 scopus 로고    scopus 로고
    • A Bayesian-frequentist two-stage single-arm phase II clinical trial design
    • Dong G, Shih WJ, Moore D, Quan H, Marcella S. A Bayesian-frequentist two-stage single-arm phase II clinical trial design. Stat Med. 2012; 31:2055-67.
    • (2012) Stat Med , vol.31 , pp. 2055-2067
    • Dong, G.1    Shih, W.J.2    Moore, D.3    Quan, H.4    Marcella, S.5
  • 115
    • 84861098716 scopus 로고    scopus 로고
    • Adaptive blinded sample size adjustment for comparing two normal means-a mostly Bayesian approach
    • Hartley AM. Adaptive blinded sample size adjustment for comparing two normal means-a mostly Bayesian approach. Pharm Stat. 2012; 11:230-40.
    • (2012) Pharm Stat , vol.11 , pp. 230-240
    • Hartley, A.M.1
  • 116
    • 33750884934 scopus 로고    scopus 로고
    • Confidentiality and trial integrity issues for adaptive designs
    • Gallo P. Confidentiality and trial integrity issues for adaptive designs. Drug Inf J. 2006; 40:445-50.
    • (2006) Drug Inf J , vol.40 , pp. 445-450
    • Gallo, P.1
  • 117
    • 84897954467 scopus 로고    scopus 로고
    • Predicting clinical trial results based on announcements of interim analyses
    • Broglio KR, Stivers DN, Berry DA. Predicting clinical trial results based on announcements of interim analyses. Trials. 2014; 15:73.
    • (2014) Trials , vol.15 , pp. 73
    • Broglio, K.R.1    Stivers, D.N.2    Berry, D.A.3
  • 118
    • 84863475669 scopus 로고    scopus 로고
    • On the independence of data monitoring committee in adaptive design clinical trials
    • Chow SC, Corey R, Lin M. On the independence of data monitoring committee in adaptive design clinical trials. J Biopharm Stat. 2012; 22:853-67.
    • (2012) J Biopharm Stat , vol.22 , pp. 853-867
    • Chow, S.C.1    Corey, R.2    Lin, M.3
  • 119
    • 65649112030 scopus 로고    scopus 로고
    • Exploring changes in treatment effects across design stages in adaptive trials
    • Friede T, Henderson R. Exploring changes in treatment effects across design stages in adaptive trials. Pharm Stat. 2009; 8:62-72.
    • (2009) Pharm Stat , vol.8 , pp. 62-72
    • Friede, T.1    Henderson, R.2
  • 120
    • 65649086704 scopus 로고    scopus 로고
    • What should be the role of homogeneity testing in adaptive trials?
    • Gallo P, Chuang-Stein C. What should be the role of homogeneity testing in adaptive trials?Pharm Stat. 2009; 8:1-4.
    • (2009) Pharm Stat , vol.8 , pp. 1-4
    • Gallo, P.1    Chuang-Stein, C.2
  • 121
    • 84930399719 scopus 로고    scopus 로고
    • No solution yet for combining two independent studies in the presence of heterogeneity
    • Gonnermann A, Framke T, Großhennig A, Koch A. No solution yet for combining two independent studies in the presence of heterogeneity. Stat Med. 2015; 34:2476-80.
    • (2015) Stat Med , vol.34 , pp. 2476-2480
    • Gonnermann, A.1    Framke, T.2    Großhennig, A.3    Koch, A.4
  • 122
    • 74749102937 scopus 로고    scopus 로고
    • Testing for qualitative interactions between stages in an adaptive study
    • Parker RA. Testing for qualitative interactions between stages in an adaptive study. Stat Med. 2010; 29:210-18.
    • (2010) Stat Med , vol.29 , pp. 210-218
    • Parker, R.A.1
  • 123
    • 84888137381 scopus 로고    scopus 로고
    • Unblinded adaptive statistical information design based on clinical endpoint or biomarker
    • Wang SJ, Brannath W, Brückner M, Hung HMJ, Koch A. Unblinded adaptive statistical information design based on clinical endpoint or biomarker. Stat Biopharm Res. 2013; 5:293-310.
    • (2013) Stat Biopharm Res , vol.5 , pp. 293-310
    • Wang, S.J.1    Brannath, W.2    Brückner, M.3    Hung, H.M.J.4    Koch, A.5
  • 124
    • 0035822324 scopus 로고    scopus 로고
    • Systematic reviews in health care: assessing the quality of controlled clinical trials
    • Jüni P, Altman DG, Egger M. Systematic reviews in health care: assessing the quality of controlled clinical trials. BMJ. 2001; 323:42-6.
    • (2001) BMJ , vol.323 , pp. 42-46
    • Jüni, P.1    Altman, D.G.2    Egger, M.3
  • 125
    • 77950189829 scopus 로고    scopus 로고
    • CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials
    • Schulz KF, Altman DG, Moher D. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010; 340:c332.
    • (2010) BMJ , vol.340
    • Schulz, K.F.1    Altman, D.G.2    Moher, D.3
  • 126
    • 33646257465 scopus 로고    scopus 로고
    • Application of adaptive designs-a review
    • Bauer P, Einfalt J. Application of adaptive designs-a review. Biometrical J. 2006; 48:493-506.
    • (2006) Biometrical J , vol.48 , pp. 493-506
    • Bauer, P.1    Einfalt, J.2
  • 127
    • 84889644506 scopus 로고    scopus 로고
    • Standards for the design, conduct, and evaluation of adaptive randomized clinical trials
    • Washington: Patient-Centered Outcomes Research Institute, Accessed 7 Jul 2017.
    • Detry MA, Lewis RJ, Broglio KR, Connor JT, Berry SM, Berry DA. Standards for the design, conduct, and evaluation of adaptive randomized clinical trials. Washington: Patient-Centered Outcomes Research Institute; 2012. http://www.pcori.org/assets/Standards-for-the-Design-Conduct-and-Evaluation-of-Adaptive-Randomized-Clinical-Trials.pdf. Accessed 7 Jul 2017.
    • (2012)
    • Detry, M.A.1    Lewis, R.J.2    Broglio, K.R.3    Connor, J.T.4    Berry, S.M.5    Berry, D.A.6
  • 128
    • 84903361151 scopus 로고    scopus 로고
    • Three steps to writing adaptive study protocols in the early phase clinical development of new medicines
    • Lorch U, O'Kane M, Taubel J. Three steps to writing adaptive study protocols in the early phase clinical development of new medicines. BMC Med Res Methodol. 2014; 14:84.
    • (2014) BMC Med Res Methodol , vol.14 , pp. 84
    • Lorch, U.1    O'Kane, M.2    Taubel, J.3
  • 129
    • 84951133315 scopus 로고    scopus 로고
    • An investigation of the shortcomings of the CONSORT 2010 statement for the reporting of group sequential randomised controlled trials: a methodological systematic review
    • Stevely A, Dimairo M, Todd S, Julious SA, Nicholl J, Hind D, et al.An investigation of the shortcomings of the CONSORT 2010 statement for the reporting of group sequential randomised controlled trials: a methodological systematic review. PLoS One. 2015; 10:e0141104.
    • (2015) PLoS One , vol.10
    • Stevely, A.1    Dimairo, M.2    Todd, S.3    Julious, S.A.4    Nicholl, J.5    Hind, D.6
  • 131
    • 84971508133 scopus 로고    scopus 로고
    • Rendering the 3 + 3 design to rest: more efficient approaches to oncology dose-finding trials in the era of targeted therapy
    • Nie L, Rubin EH, Mehrotra N, Pinheiro J, Fernandes LL, Roy A, et al.Rendering the 3 + 3 design to rest: more efficient approaches to oncology dose-finding trials in the era of targeted therapy. Clin Cancer Res. 2016; 22:2623-9.
    • (2016) Clin Cancer Res , vol.22 , pp. 2623-2629
    • Nie, L.1    Rubin, E.H.2    Mehrotra, N.3    Pinheiro, J.4    Fernandes, L.L.5    Roy, A.6
  • 132
    • 85042564335 scopus 로고    scopus 로고
    • A quick guide why not to use A+B designs
    • Accessed 7 Jul 2017.
    • Adaptive Designs Working Group of the MRC Network of Hubs for Trials Methodology Research. A quick guide why not to use A+B designs. 2016. https://www.methodologyhubs.mrc.ac.uk/files/6814/6253/2385/A_quick_guide_why_not_to_use_AB_designs.pdf. Accessed 7 Jul 2017.
    • (2016)
  • 133
    • 84904500274 scopus 로고    scopus 로고
    • When is a seamless study desirable? Case studies from different pharmaceutical sponsors
    • Cuffe RL, Lawrence D, Stone A, Vandemeulebroecke M. When is a seamless study desirable? Case studies from different pharmaceutical sponsors. Pharm Stat. 2014; 13:229-37.
    • (2014) Pharm Stat , vol.13 , pp. 229-237
    • Cuffe, R.L.1    Lawrence, D.2    Stone, A.3    Vandemeulebroecke, M.4
  • 134
    • 84999532085 scopus 로고    scopus 로고
    • Multi-arm clinical trials with treatment selection: what can be gained and at what price?
    • Jaki T. Multi-arm clinical trials with treatment selection: what can be gained and at what price?Clin Investig. 2015; 5:393-9.
    • (2015) Clin Investig , vol.5 , pp. 393-399
    • Jaki, T.1
  • 135
    • 46349091495 scopus 로고    scopus 로고
    • Adaptive clinical trials: progress and challenges
    • Coffey CS, Kairalla JA. Adaptive clinical trials: progress and challenges. Drugs R D. 2008; 9:229-42.
    • (2008) Drugs R D , vol.9 , pp. 229-242
    • Coffey, C.S.1    Kairalla, J.A.2
  • 136
    • 85038437325 scopus 로고    scopus 로고
    • A pragmatic approach to the design and calibration of a Bayesian CRM dose finding trial
    • Cole M, Stocken D, Yap C. A pragmatic approach to the design and calibration of a Bayesian CRM dose finding trial. Trials. 2015;16 Suppl2:P210.
    • (2015) Trials , vol.16
    • Cole, M.1    Stocken, D.2    Yap, C.3
  • 137
    • 85038438739 scopus 로고    scopus 로고
    • Dose transition pathways: the missing link between complex dose-finding designs and simple decision making
    • Yap C, Billingham LJ, Cheung YK, Craddock C, O'Quigley J. Dose transition pathways: the missing link between complex dose-finding designs and simple decision making. Clin Cancer Res. 2017; 23:7440-7.
    • (2017) Clin Cancer Res , vol.23 , pp. 7440-7447
    • Yap, C.1    Billingham, L.J.2    Cheung, Y.K.3    Craddock, C.4    O'Quigley, J.5
  • 138
    • 85038419303 scopus 로고    scopus 로고
    • Implementation of adaptive dose-finding designs in two early phase haematological trials: clinical, operational, and methodological challenges
    • Yap C, Craddock C, Collins G, Khan J, Siddique S, Billingham L. Implementation of adaptive dose-finding designs in two early phase haematological trials: clinical, operational, and methodological challenges. Trials. 2013;14 Suppl 1:O75.
    • (2013) Trials , vol.14
    • Yap, C.1    Craddock, C.2    Collins, G.3    Khan, J.4    Siddique, S.5    Billingham, L.6
  • 140
    • 84866145248 scopus 로고    scopus 로고
    • Flexible trial design in practice-stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial
    • Sydes MR, Parmar MKB, Mason MD, Clarke NW, Amos C, Anderson J, et al.Flexible trial design in practice-stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial. Trials. 2012; 13:168.
    • (2012) Trials , vol.13 , pp. 168
    • Sydes, M.R.1    Parmar, M.K.B.2    Mason, M.D.3    Clarke, N.W.4    Amos, C.5    Anderson, J.6
  • 141
    • 85042558588 scopus 로고    scopus 로고
    • The design of a multi-arm multi-stage (MAMS) phase III randomised controlled trial comparing alternative regimens for escalating (COMPARE) treatment of intermediate and high-risk oropharyngeal cancer with reflections on the complications of introducing a new experimental arm
    • Gaunt P, Mehanna H, Yap C. The design of a multi-arm multi-stage (MAMS) phase III randomised controlled trial comparing alternative regimens for escalating (COMPARE) treatment of intermediate and high-risk oropharyngeal cancer with reflections on the complications of introducing a new experimental arm. Trials. 2015;16 Suppl 2:O16.
    • (2015) Trials , vol.16
    • Gaunt, P.1    Mehanna, H.2    Yap, C.3
  • 142
    • 84943753935 scopus 로고    scopus 로고
    • Prospective study evaluating the relative sensitivity of 18F-NaF PET/CT for detecting skeletal metastases from renal cell carcinoma in comparison to multidetector CT and 99mTc-MDP bone scintigraphy, using an adaptive trial design
    • Gerety EL, Lawrence EM, Wason J, Yan H, Hilborne S, Buscombe J, et al.Prospective study evaluating the relative sensitivity of 18F-NaF PET/CT for detecting skeletal metastases from renal cell carcinoma in comparison to multidetector CT and 99mTc-MDP bone scintigraphy, using an adaptive trial design. Ann Oncol. 2015; 26:2113-8.
    • (2015) Ann Oncol , vol.26 , pp. 2113-2118
    • Gerety, E.L.1    Lawrence, E.M.2    Wason, J.3    Yan, H.4    Hilborne, S.5    Buscombe, J.6
  • 143
    • 84864238949 scopus 로고    scopus 로고
    • Efficacy and tolerability of rizatriptan in pediatric migraineurs: results from a randomized, double-blind, placebo-controlled trial using a novel adaptive enrichment design
    • Ho TW, Pearlman E, Lewis D, Hämäläinen M, Connor K, Michelson D, et al.Efficacy and tolerability of rizatriptan in pediatric migraineurs: results from a randomized, double-blind, placebo-controlled trial using a novel adaptive enrichment design. Cephalalgia. 2012; 32:750-65.
    • (2012) Cephalalgia , vol.32 , pp. 750-765
    • Ho, T.W.1    Pearlman, E.2    Lewis, D.3    Hämäläinen, M.4    Connor, K.5    Michelson, D.6
  • 144
    • 84888132560 scopus 로고    scopus 로고
    • Adaptive enrichment with subpopulation selection at interim: methodologies, applications and design considerations
    • Wang SJ, Hung HMJ. Adaptive enrichment with subpopulation selection at interim: methodologies, applications and design considerations. Contemp Clin Trials. 2013; 36:673-81.
    • (2013) Contemp Clin Trials , vol.36 , pp. 673-681
    • Wang, S.J.1    Hung, H.M.J.2
  • 145
  • 146
    • 84901931670 scopus 로고    scopus 로고
    • Rationale and study design of the adaptive study of IL-2 dose on regulatory T cells in type 1 diabetes (DILT1D): a non-randomised, open label, adaptive dose finding trial
    • Waldron-Lynch F, Kareclas P, Irons K, Walker NM, Mander A, Wicker LS, et al. Rationale and study design of the adaptive study of IL-2 dose on regulatory T cells in type 1 diabetes (DILT1D): a non-randomised, open label, adaptive dose finding trial. BMJ Open. 2014; 4:e005559.
    • (2014) BMJ Open , vol.4
    • Waldron-Lynch, F.1    Kareclas, P.2    Irons, K.3    Walker, N.M.4    Mander, A.5    Wicker, L.S.6
  • 147
    • 84944316022 scopus 로고    scopus 로고
    • Patient-centric trials for therapeutic development in precision oncology
    • Biankin AV, Piantadosi S, Hollingsworth SJ. Patient-centric trials for therapeutic development in precision oncology. Nature. 2015; 526:361-70.
    • (2015) Nature , vol.526 , pp. 361-370
    • Biankin, A.V.1    Piantadosi, S.2    Hollingsworth, S.J.3
  • 148
    • 84977650991 scopus 로고    scopus 로고
    • Biomarker-guided adaptive trial designs in phase II and phase III: a methodological review
    • Antoniou M, Jorgensen AL, Kolamunnage-Dona R. Biomarker-guided adaptive trial designs in phase II and phase III: a methodological review. PLoS One. 2016; 11:e0149803.
    • (2016) PLoS One , vol.11
    • Antoniou, M.1    Jorgensen, A.L.2
  • 149
    • 84921847585 scopus 로고    scopus 로고
    • Low-dose dexamethasone as a treatment for women with heavy menstrual bleeding: protocol for response-adaptive randomised placebo-controlled dose-finding parallel group trial (DexFEM)
    • Warner P, Weir CJ, Hansen CH, Douglas A, Madhra M, Hillier SG, et al. Low-dose dexamethasone as a treatment for women with heavy menstrual bleeding: protocol for response-adaptive randomised placebo-controlled dose-finding parallel group trial (DexFEM). BMJ Open. 2015; 5:e006837.
    • (2015) BMJ Open , vol.5
    • Warner, P.1    Weir, C.J.2    Hansen, C.H.3    Douglas, A.4    Madhra, M.5    Hillier, S.G.6
  • 151
    • 84991273589 scopus 로고    scopus 로고
    • Response-adaptive clinical trials: case studies in the medical literature
    • Grieve AP. Response-adaptive clinical trials: case studies in the medical literature. Pharm Stat. 2017; 16:64-86.
    • (2017) Pharm Stat , vol.16 , pp. 64-86
    • Grieve, A.P.1
  • 152
    • 84864022490 scopus 로고    scopus 로고
    • A novel phase I/IIa design for early phase oncology studies and its application in the evaluation of MK-0752 in pancreatic cancer
    • Whitehead J, Thygesen H, Jaki T, Davies S, Halford S, Turner H, et al.A novel phase I/IIa design for early phase oncology studies and its application in the evaluation of MK-0752 in pancreatic cancer. Stat Med. 2012; 31:1931-43.
    • (2012) Stat Med , vol.31 , pp. 1931-1943
    • Whitehead, J.1    Thygesen, H.2    Jaki, T.3    Davies, S.4    Halford, S.5    Turner, H.6
  • 153
    • 85042557717 scopus 로고    scopus 로고
    • Practical implementation of an adaptive phase I/II design in chronic myeloid leukaemia: evaluating both efficacy and toxicity using the EffTox design
    • Khan J, Yap C, Clark R, Fenwick N, Marin D. Practical implementation of an adaptive phase I/II design in chronic myeloid leukaemia: evaluating both efficacy and toxicity using the EffTox design. Trials. 2013;14 Suppl 1:P20.
    • (2013) Trials , vol.14
    • Khan, J.1    Yap, C.2    Clark, R.3    Fenwick, N.4    Marin, D.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.