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Volumn 33, Issue 24, 2014, Pages 4186-4201

Bayesian methods for the design and interpretation of clinical trials in very rare diseases

Author keywords

Allocation ratio; Bayesian model; Expert opinion; Prior elicitation; Prior power; Rare diseases

Indexed keywords

CORTICOSTEROID; CYCLOPHOSPHAMIDE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; MYCOPHENOLATE MOFETIL; MYCOPHENOLIC ACID;

EID: 84908042854     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.6225     Document Type: Article
Times cited : (81)

References (38)
  • 1
    • 84908046239 scopus 로고    scopus 로고
    • Guideline on clinical trials in small populations,Accessed 11 June 2014.
    • European Medicines Agency. Guideline on clinical trials in small populations, 2006. http://www.ema.europa.eu/docs/en_ GB/document_library/Scientific_guideline/2009/09/WC500003615.pdf, Accessed 11 June 2014.
    • (2006)
  • 2
    • 0029586133 scopus 로고
    • Clinical trials and rare diseases: a way out of a conundrum
    • Lilford RJ, Thornton JG, Braunholtz D. Clinical trials and rare diseases: a way out of a conundrum. British Medical Journal 1995; 311(7020):1621-1625.
    • (1995) British Medical Journal , vol.311 , Issue.7020 , pp. 1621-1625
    • Lilford, R.J.1    Thornton, J.G.2    Braunholtz, D.3
  • 3
    • 84908021409 scopus 로고    scopus 로고
    • Small sample sizes in clinical trials: a statistician's perspective
    • Billingham L,Malottki K, Steven N. Small sample sizes in clinical trials: a statistician's perspective. Clinical Investigation 2012; 2(7):655-657.
    • (2012) Clinical Investigation , vol.2 , Issue.7 , pp. 655-657
    • Billingham, L.1    Malottki, K.2    Steven, N.3
  • 6
    • 84883134848 scopus 로고    scopus 로고
    • Using historical control information for the design and analysis of clinical trials with overdispersed count data
    • Gsteiger S, Neuenschwander B, Mercier F, Schmidli H. Using historical control information for the design and analysis of clinical trials with overdispersed count data. Statistics in Medicine 2013; 32(21):3609-3622.
    • (2013) Statistics in Medicine , vol.32 , Issue.21 , pp. 3609-3622
    • Gsteiger, S.1    Neuenschwander, B.2    Mercier, F.3    Schmidli, H.4
  • 7
    • 0002083004 scopus 로고    scopus 로고
    • Power prior distributions for regression models
    • Ibrahim JG, Chen M-H. Power prior distributions for regression models. Statistical Science 2000; 15(1):46-60.
    • (2000) Statistical Science , vol.15 , Issue.1 , pp. 46-60
    • Ibrahim, J.G.1    Chen, M.-H.2
  • 9
    • 24944463173 scopus 로고    scopus 로고
    • A Bayesian approach to randomized controlled trials in children utilizing information from adults: the case of Guillain-Barré
    • Goodman SN, Sladky JT. A Bayesian approach to randomized controlled trials in children utilizing information from adults: the case of Guillain-Barré. Clinical Trials 2005; 2(4):305-310.
    • (2005) Clinical Trials , vol.2 , Issue.4 , pp. 305-310
    • Goodman, S.N.1    Sladky, J.T.2
  • 10
    • 70449723061 scopus 로고    scopus 로고
    • Bayesian design using adult data to augment pediatric trials
    • Schoenfeld DA, Zheng H, Finkelstein DM. Bayesian design using adult data to augment pediatric trials. Clinical Trials 2009; 6(4):297-304.
    • (2009) Clinical Trials , vol.6 , Issue.4 , pp. 297-304
    • Schoenfeld, D.A.1    Zheng, H.2    Finkelstein, D.M.3
  • 11
    • 84950631713 scopus 로고
    • Cognitive processes and the assessment of subjective probability distributions
    • Hogarth RM. Cognitive processes and the assessment of subjective probability distributions. Journal of the American Statistical Association 1975; 70(350):271-289.
    • (1975) Journal of the American Statistical Association , vol.70 , Issue.350 , pp. 271-289
    • Hogarth, R.M.1
  • 14
    • 84874977112 scopus 로고    scopus 로고
    • Structured approach to the elicitation of expert beliefs for a Bayesian-designed clinical trial: a case study
    • Kinnersley N, Day S. Structured approach to the elicitation of expert beliefs for a Bayesian-designed clinical trial: a case study. Pharmaceutical Statistics 2013; 12(2):104-113.
    • (2013) Pharmaceutical Statistics , vol.12 , Issue.2 , pp. 104-113
    • Kinnersley, N.1    Day, S.2
  • 16
    • 0346856880 scopus 로고    scopus 로고
    • Eliciting expert beliefs in substantial practical applications
    • O'Hagan A. Eliciting expert beliefs in substantial practical applications. The Statistician 1998; 47(1):21-35.
    • (1998) The Statistician , vol.47 , Issue.1 , pp. 21-35
    • O'Hagan, A.1
  • 18
    • 0035961574 scopus 로고    scopus 로고
    • Quantifying and documenting prior beliefs in clinical trials
    • Chaloner K, Rhame FS. Quantifying and documenting prior beliefs in clinical trials. Statistics in Medicine 2001; 20(4): 581-600.
    • (2001) Statistics in Medicine , vol.20 , Issue.4 , pp. 581-600
    • Chaloner, K.1    Rhame, F.S.2
  • 20
    • 0037385232 scopus 로고    scopus 로고
    • Elicitation of prior distributions for a phase III randomized controlled trial of adjuvant therapy with surgery for hepatocellular carcinoma
    • Tan S-B, Chung Y-FA, Tai B-C, Cheung Y-B, Machin D. Elicitation of prior distributions for a phase III randomized controlled trial of adjuvant therapy with surgery for hepatocellular carcinoma. Controlled Clinical Trials 2003; 24(2): 110-121.
    • (2003) Controlled Clinical Trials , vol.24 , Issue.2 , pp. 110-121
    • Tan, S.-B.1    Chung, Y.-F.A.2    Tai, B.-C.3    Cheung, Y.-B.4    Machin, D.5
  • 21
    • 60849133005 scopus 로고    scopus 로고
    • A practical approach for eliciting expert prior beliefs about cancer survival in phase III randomized trial
    • Hiance A, Chevret S, Levy V. A practical approach for eliciting expert prior beliefs about cancer survival in phase III randomized trial. Journal of Clinical Epidemiology 2009; 62(4):431-437.
    • (2009) Journal of Clinical Epidemiology , vol.62 , Issue.4 , pp. 431-437
    • Hiance, A.1    Chevret, S.2    Levy, V.3
  • 22
    • 0040235596 scopus 로고
    • An improved method for deriving optimal one-sided group sequential tests
    • Eales JD, Jennison C. An improved method for deriving optimal one-sided group sequential tests. Biometrika 1992; 79(1):13-24.
    • (1992) Biometrika , vol.79 , Issue.1 , pp. 13-24
    • Eales, J.D.1    Jennison, C.2
  • 28
    • 84908046236 scopus 로고    scopus 로고
    • shiny: web application framework for R. R package version 0.7.099, Accessed 16 September 2013.
    • RStudio and Inc. shiny: web application framework for R. R package version 0.7.099., 2013. http://www.rstudio.com/shiny/, Accessed 16 September 2013.
    • (2013)
  • 29
  • 30
    • 43749123682 scopus 로고    scopus 로고
    • Determining the effective sample size of a parametric prior
    • Morita S, Thall PF, Müller P. Determining the effective sample size of a parametric prior. Biometrics 2008; 64(2):595-602.
    • (2008) Biometrics , vol.64 , Issue.2 , pp. 595-602
    • Morita, S.1    Thall, P.F.2    Müller, P.3
  • 32
    • 0017272279 scopus 로고
    • The combination of randomized and historical controls in clinical trials
    • Pocock S. The combination of randomized and historical controls in clinical trials. Journal of Chronic Disease 1976; 29(3):175-88.
    • (1976) Journal of Chronic Disease , vol.29 , Issue.3 , pp. 175-188
    • Pocock, S.1
  • 33
    • 44949170805 scopus 로고    scopus 로고
    • Bayesian sample size for exploratory clinical trials incorporating historical data
    • Whitehead J, Valdes-Marquez E, Johnson P, Graham G. Bayesian sample size for exploratory clinical trials incorporating historical data. Statistics in Medicine 2008; 27(13):2307-2327.
    • (2008) Statistics in Medicine , vol.27 , Issue.13 , pp. 2307-2327
    • Whitehead, J.1    Valdes-Marquez, E.2    Johnson, P.3    Graham, G.4
  • 34
    • 0029071328 scopus 로고
    • Bayesian decision procedures for dose determining experiments
    • Whitehead J, Brunier H. Bayesian decision procedures for dose determining experiments. Statistics in Medicine 1995; 14(9):885-893.
    • (1995) Statistics in Medicine , vol.14 , Issue.9 , pp. 885-893
    • Whitehead, J.1    Brunier, H.2
  • 35
    • 0002434647 scopus 로고    scopus 로고
    • Easy-to-implement Bayesian methods for dose-escalation studies in healthy volunteers
    • Whitehead J, Patterson S,Webber D, Francis S, Zhou Y. Easy-to-implement Bayesian methods for dose-escalation studies in healthy volunteers. Biostatistics 2001; 2(1):47-61.
    • (2001) Biostatistics , vol.2 , Issue.1 , pp. 47-61
    • Whitehead, J.1    Patterson, S.2    Webber, D.3    Francis, S.4    Zhou, Y.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.