Bayesian clinical trials in action

Statistics in Medicine

Volumn 31, Issue 25, 2012, Pages 2955-2972

  Jack Lee, J ^a   Chu, Caleb T ^a  

^a UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

Author keywords

Adaptive trial design; Bayesian paradigm; Clinical trial conduct; Frequentist paradigm; Trial efficiency; Trial ethics

Indexed keywords

ADAPTIVE TRIAL; ARTICLE; BAYES THEOREM; BAYESIAN LEARNING; CALCULATION; CLINICAL DECISION MAKING; CLINICAL TRIAL (TOPIC); DATA ANALYSIS; DATA EXTRACTION; HUMAN; MATHEMATICAL COMPUTING; MEDICAL RESEARCH; MONITORING; PROBLEM SOLVING; PROCESS DESIGN; STATISTICAL DISTRIBUTION; STATISTICAL MODEL; STUDY DESIGN; TYPES OF STUDY; UNCERTAINTY;

BAYES THEOREM; CLINICAL TRIALS AS TOPIC; LIKELIHOOD FUNCTIONS; RESEARCH DESIGN; SOFTWARE;

EID: 84867439711     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.5404     Document Type: Article

References (59)

1. Streptomycin-in-Tuberculosis-Trials-Committee. Streptomycin treatment of pulmonary tuberculosis: a Medical Research Council investigation. British Medical Journal 1948; 2:769-782, DOI: 10.1136/bmj.2.4582.769.
2. Halperin M, DeMets DL, Ware JH. Early methodological developments for clinical trials at the National Heart, Lung and Blood Institute. Statistics in Medicine 1990; 9(8):881-892, DOI: 10.1002/sim.4780090804.
3. Gehan EA, Schneiderman MA. Historical and methodological developments in clinical trials at the National Cancer Institute. Statistics in Medicine 1990; 9:871-880, DOI: 10.1002/sim.4780090803.
4. Stigler SM. The history of statistics in 1933. Statistical Science 1996; 11:244-252, DOI: 10.1214/ss/1032280216.
5. Gehan EA. Methodological issues in cancer clinical trials: the comparison of therapies. Biomedicine and Pharmacotherapy 1988; 42(3):161-165.
6. Bayarri MJ, Berger JO. The interplay of Bayesian and frequentist analysis. Statistical Science 2004; 19:58-80, DOI: 10.1214/088342304000000116.
7. Bayes T. An essay towards solving a problem in the doctrine of chances. 1763. M.D. Computing: Computers in Medical Practice 1991; 8:157-171.
8. Stigler SM. Thomas Bayes' Bayesian inference. Journal of the Royal Statistical Society, Series A 1982; 145:250-258, DOI: 10.2307/2981538.
9. Bernardo JM, Smith AFM. Bayesian Theory. Wiley:West Sussex, 1994.
10. Meier P. Jerome Cornfield and the methodology of clinical trials. Controlled Clinical Trials 1981; 1(4):339-345.
11. Ederer F. Jerome Cornfield's contributions to the conduct of clinical trials. Biometrics 1982; 38 Suppl:25-32.
12. Ashby D. Bayesian statistics in medicine: a 25 year review. Statistics in Medicine 2006; 25(21):3589-3631, DOI: 10.1002/sim.2672.
13. Grieve AP. 25 years of Bayesian methods in the pharmaceutical industry: a personal, statistical bummel. Pharmaceutical Statistics 2007; 6:261-281, DOI:10.1002/pst.315.
14. Berger JO, Wolpert RL. The Likelihood Principle. SS Gupta, ed. Institute of Mathematical Statistics:Hayward, CA, 1984. http://projecteuclid.org/euclid.lnms/1215466210.
15. Bernardo JM. Bayesian Reference Analysis, A Postgraduate Tutorial Course Universitat de València:Valencia, Spain. (http://www.uv.es/~bernardo/Monograph.pdf.
16. Bernardo JM. The concept of exchangeability and its applications. Far East Journal of Mathematical Sciences 1996; 4:111-121.
17. Betrò B, Bodini A, Guglielmi A. Generalized moment theory and Bayesian robustness analysis for hierarchical mixture models. Annals of the Institute of Statistical Mathematics 2006; 58(4):721-738.
18. Brutti P, De Santis F, Gubbiotti S. Robust Bayesian sample size determination in clinical trials. Statistics in Medicine 2008; 27(13):2290-2306.
19. Andrade JAA, O'Hagan A. Bayesian robustness modelling of location and scale parameters. Scandinavian Journal of Statistics 2011; 38(4):691-711.
20. Sutton AJ, Abrams KR. Bayesian methods in meta-analysis and evidence synthesis. Statistical Methods in Medical Research 2001; 10(4):277-303, DOI:10.1177/096228020101000404.
21. Berry DA. Bayesian clinical trials. Nature Reviews Drug Discovery 2006; 5(1):27-36, DOI: 10.1038/nrd1927.
22. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. Chapman & Hall/CRC:Boca Raton, FL, 2000.
23. Dmitrienko A, Wang MD. Bayesian predictive approach to interim monitoring in clinical trials. Statistics in Medicine 2006; 25(13):2178-2195, DOI:10.1002/sim.2204.
24. Berry DA. Adaptive trial design. Clinical Advances in Hematology and Oncology 2007; 5(7):522-524. http://www.clinicaladvances.com/article_pdfs/ho-article-200707-drugdev.pdf.
25. Center for Drug Evaluation and Research, Food and Drug Administration. Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics. U.S. Department of Health and Human Services:Rockville, MD, 2010. Accessed 10/14/2011; available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM201790.pdf.
26. Geman S, Geman D. Stochastic relaxation, Gibbs distributions, and the Bayesian restoration of images. IEEE Transactions on Pattern Analysis and Machine Intelligence 1984; PAMI-6:721-741, DOI: 10.1109/TPAMI.1984.4767596.
27. Gelfand AE, Smith AFM. Sampling-based approaches to calculating marginal densities. Journal of the American Statistical Association 1990; 85:398-409, DOI:10.2307/2289776.
28. Lunn D, Spiegelhalter D, Thomas A, Best N. The BUGS project: evolution, critique and future directions. Statistics in Medicine 2009; 28:3049-3067, DOI:10.1002/sim.3680.
29. Goldstein M. Subjective Bayesian analysis: principles and practice. Bayesian Analysis 2006; 1:403-420, DOI: 10.1214/06-BA116.
30. Berger J. The case for objective Bayesian analysis. Bayesian Analysis 2006; 1:385-402, DOI: 10.1214/06-BA115.
31. Fienberg SE. Does it make sense to be an "objective Bayesian" ? (Comment on articles by Berger and by Goldstein). Bayesian Analysis 2006; 1:429-432. DOI:10.1214/06-BA116C.
32. Spiegelhalter DJ, Abrams KR, Myles JP. Bayesian Approaches to Clinical Trials and Health-Care Evaluation. Wiley:West Sussex, 2004.
33. Sheiner LB, Beal SL. Evaluation of methods for estimating population pharmacokinetic parameters. I. Michaelis-Menten model: routine clinical pharmacokinetic data. Journal of Pharmacokinetics and Biopharmaceutics 1980; 8:553-571, DOI: 10.1007/BF01060053.
34. Sheiner LB, Ludden TM. Population pharmacokinetics/dynamics. Annual Review of Pharmacology and Toxicology 1992; 32:185-209, DOI:10.1146/annurev.pa.32.040192.001153.
35. Whiting B, Kelman AW, Grevel J. Population pharmacokinetics. Theory and clinical application. Clinical Pharmacokinetics 1986; 11:387-401, DOI:10.2165/00003088-198611050-00004.
36. Bauer RJ, Guzy S, Ng C. A survey of population analysis methods and software for complex pharmacokinetic and pharmacodynamic models with examples. The AAPS Journal 2007; 9:E60-E83, DOI: 10.1208/aapsj0901007.
37. Buzdar AU, Ibrahim NK, Francis D, Booser DJ, Thomas ES, Theriault RL, Pusztai L, Green MC, Arun BK, Giordano SH, Cristofanilli M, Frye DK, Smith TL, Hunt KK, Singletary SE, Sahin AA, Ewer MS, Buchholz TA, Berry D, Hortobagyi GN. Significantly higher pathologic complete remission rate after neoadjuvant therapy with trastuzumab, paclitaxel, and epirubicin chemotherapy: results of a randomized trial in human epidermal growth factor receptor 2-positive operable breast cancer. Journal of Clinical Oncology 2005; 23:3676-3685. DOI:10.1200/JCO.2005.07.032.
38. Kim ES, Herbst RS, Wistuba II, Lee JJ, Jr. GRB, Tsao A, Stewart DJ, Hicks ME, Jr JE, Gupta S, Alden CM, Liu S, Tang X, Khuri FR, Tran HT, Johnson BE, Heymach JV, Mao L, Fossella F, Kies MS, Papadimitrakopoulou V, Davis SE, Lippman SM, Hong WK. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discovery 2011; 1:44-53, DOI:10.1158/2159-8274.CD-10-0010.
39. Julian TB, Blumencranz P, Deck K, Whitworth P, Berry DA, Berry SM, Rosenberg A, Chagpar AB, Reintgen D, Beitsch P, Simmons R, Saha S, Mamounas EP, Giuliano A. Novel intraoperative molecular test for sentinel lymph node metastases in patients with early-stage breast cancer. Journal of Clinical Oncology 2008; 26:3338-3345, DOI: 10.1200/JCO.2007.14.0665.
40. de Lima M, Champlin RE, Thall PF, Wang X, Martin TG 3rd, Cook JD, McCormick G, Qazilbash M, Kebriaei P, Couriel D, Shpall EJ, Khouri I, Anderlini P, Hosing C, Chan KW, Andersson BS, Patah PA, Caldera Z, Jabbour E, Giralt S. Phase I/II study of gemtuzumab ozogamicin added to fludarabine, melphalan and allogeneic hematopoietic stem cell transplantation for high-risk CD33 positive myeloid leukemias and myelodysplastic syndrome. Leukemia 2008; 22(2):258-264.
41. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics 1990; 46:33-48, DOI:10.2307/2531628.
42. Babb J, Rogatko A, Zacks S. Cancer phase I clinical trials: efficient dose escalation with overdose control. Statistics in Medicine 1998; 17:1103-1120, DOI: 10.1002/(SICI)1097-0258(19980530)17:10<1103::AID-SIM793>3.0.CO;2-9.
43. Chevret S. Bayesian adaptive clinical trials: a dream for statisticians only? Statistics in Medicine 2011, DOI: 10.1002/sim.4363. [Sept 9 Epub ahead of print].
44. Biswas S, Liu DD, Lee JJ, Berry DA. Bayesian clinical trials at the University of Texas M. D. Anderson Cancer Center. Clinical Trials 2009; 6:205-216, DOI:10.1177/1740774509104992.
45. Zhou X, Liu S, Kim ES, Herbst RS, Lee JJ. Bayesian adaptive design for targeted therapy development in lung cancer-a step toward personalized medicine. Clinical Trials 2008; 5:181-193, DOI: 10.1177/1740774508091815.
46. Tighiouart M, Rogatko A. Dose finding with escalation with overdose control (EWOC) in cancer clinical trials. Statistical Science 2010; 25:217-226, DOI:10.1214/10-STS333.
47. Rogatko A, Schoeneck D, Jonas W, Tighiouart M, Khuri FR, Porter A. Translation of innovative designs into phase I trials. Journal of Clinical Oncology 2007; 25:4982-4986, DOI: 10.1200/JCO.2007.12.1012.
48. Chi G, Hung HMJ, O'Neill R. 2002. Some comments on "Adaptive Trials and Bayesian Statistics in Drug Development" by Don Berry, Technical Report In Pharmaceutical Report. Vol 9, 1-11.
49. Resnic FS, Zou KH, Do DV, Apostolakis G, Ohno-Machado L. Exploration of a Bayesian updating methodology to monitor the safety of interventional cardiovascular procedures. Medical Decision Making 2004; 24(4):399-407.
50. Matheny ME, Ohno-Machado L, Resnic FS. Exploration of a Bayesian updating tool to provide real-time safety monitoring for new medical devices, AMIA. Annual Symposium proceedings/AMIA Symposium, AMIA Symposium, 2005;1045.
51. Center for Devices and Radiological Health, Food and Drug Administration. Guidance for the Use of Bayesian statistics in Medical Device Clinical Trials. U.S. Department of Health and Human Services:Rockville, MD, 2010. Accessed 10/14/2011; available at http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071121.pdf.
52. Campbell G. Bayesian Statistics at the FDA: The Trailblazing Experience with Medical Devices, Presentation at Rutgers, the State University of New Jersey, (April 3, 2009). Accessed 10/14/2011; available at http://www.stat.rutgers.edu/iob/bioconf09/slides/Campbell.pdf.
53. Altman DG, Goodman SN. Transfer of technology from statistical journals to the biomedical literature-past trends and future predictions. JAMA 1994; 272:129-132, DOI: 10.1001/jama.272.2.129.
54. Berry DA. Adaptive clinical trials in oncology. Nature Reviews Clinical Oncology 2012; 9:199-207, DOI: 10.1038/nrclinonc.2011.165.
55. Barker AD, Sigman CC, Kelloff GJ, Hylton NM, Berry DA, Esserman LJ. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clinical Pharmacology and Therapeutics 2009; 86:97-100, DOI:10.1038/clpt.2009.68.
56. Berger JO, Christensen R. Could Fisher, Jeffreys and Neyman have agreed on testing? Statistical Science 2003; 18(1):1-32.
57. Efron B. Bayesians, frequentists, and scientists. Journal of the American Statistical Association 2005; 100:1-5, DOI: 10.1198/016214505000000033.
58. Little RJ. Calibrated Bayes: a Bayes/frequentist roadmap. American Statistician 2006; 60:213-223, DOI: 10.1198/000313006X117837.
59. Arjas E. On future directions in statistical methodologies-some speculations. Scandinavian Journal of Statistics 2011; 38:185-194, DOI:10.1111/j.1467-9469.2011.00737.x.