Selection of the treatment effect for sample size determination in a superiority clinical trial using a hybrid classical and Bayesian procedure

Contemporary Clinical Trials

Volumn 41, Issue , 2015, Pages 160-171

  Ciarleglio, Maria M ^a   Arendt, Christopher D ^b   Makuch, Robert W ^a   Peduzzi, Peter N ^a  

^a YALE UNIVERSITY   (United States)

^b AIR FORCE INSTITUTE OF TECHNOLOGY   (United States)

Author keywords

Clinical trial; Clinically relevant difference; Hybrid classical Bayesian; Sample size; Treatment effect

Indexed keywords

ALTERNATIVE HYPOTHESIS; ARTICLE; BAYES THEOREM; CALCULATION; CLINICAL TRIAL (TOPIC); HYBRIDIZATION; SAMPLE SIZE; STUDY DESIGN; TREATMENT PLANNING; UNCERTAINTY; HUMAN; STATISTICS;

BAYES THEOREM; CLINICAL TRIALS AS TOPIC; HUMANS; SAMPLE SIZE; STATISTICS AS TOPIC;

EID: 84922252348     PISSN: 15517144     EISSN: 15592030     Source Type: Journal    
DOI: 10.1016/j.cct.2015.01.002     Document Type: Article

References (29)

1. Lachin J.M. Introduction to sample size determination and power analysis for clinical trials. Control Clin Trials 1981, 2(2):93-113.
2. Adcock C.J. Sample size determination: a review. J R Stat Soc D Stat 1997, 46(2):261-283.
3. Spiegelhalter D.J., Freedman L.S. A predictive approach to selecting the size of a clinical trial, based on subjective clinical opinion. Stat Med 1986, 5(1):1-13.
4. Spiegelhalter D.J., Freedman L.S., Parmar M.K. Bayesian approaches to randomized trials. J R Stat Soc A Stat Soc 1994, 357-416.
5. Lenth R.V. Some practical guidelines for effective sample size determination. Am Stat 2001, 55(3):187-193.
6. Wittes J. Sample size calculations for randomized controlled trials. Epidemiol Rev 2002, 24(1):39-53.
7. Spiegelhalter D.J., Abrams K.R., Myles J.P. Bayesian approaches to clinical trials and health-care evaluation 2004, Wiley, Chichester. 1st ed.
8. Korn E.L. Projecting power from a previous study: maximum likelihood estimation. Am Stat 1990, 44(4):290-292.
9. Gillett R. An average power criterion for sample size estimation. J R Stat Soc Ser D Stat 1993, 389-394.
10. Brown B.W., Herson J., Atkinson E.N., Rozell M.E. Projection from previous studies: a Bayesian and frequentist compromise. Control Clin Trials 1987, 8(1):29-44.
11. Moussa M.A. Exact, conditional, and predictive power in planning clinical trials. Control Clin Trials 1989, 10(4):378-385.
12. Spiegelhalter D.J., Freedman L.S., Parmar M.K. Applying Bayesian ideas in drug development and clinical trials. Stat Med 1993, 12(15-16):1501-1511.
13. Shih J.H. Sample size calculation for complex clinical trials with survival endpoints. Control Clin Trials 1995, 16(6):395-407.
14. O'Hagan A., Stevens J.W., Campbell M.J. Assurance in clinical trial design. Pharm Stat 2005, 4(3):187-201.
15. Chuang-Stein C. Sample size and the probability of a successful trial. Pharm Stat 2006, 5(4):305-309.
16. Lan K.G., Wittes J.T. Some thoughts on sample size: a Bayesian-frequentist hybrid approach. Clin Trials 2012, 9(5):561-569.
17. George S.L., Desu M. Planning the size and duration of a clinical trial studying the time to some critical event. J Chronic Dis 1974, 27(1):15-24.
18. Freedman L., Spiegelhalter D. The assessment of the subjective opinion and its use in relation to stopping rules for clinical trials. J R Stat Soc Ser D Stat 1993, 153-160.
19. Chaloner K., Church T., Louis T.A., Matts J.P. Graphical elicitation of a prior distribution for a clinical trial. J R Stat Soc Ser D Stat 1993, 341-353.
20. Chaloner K. Elicitation of prior distributions. Bayesian biostatistics 1996, 141-156. CRC Press. D.A. Berry, D.K. Stangl (Eds.).
21. Chaloner K., Rhame F.S. Quantifying and documenting prior beliefs in clinical trials. Stat Med 2001, 20(4):581-600.
22. Freiman J.A., Chalmers T.C., Smith H., Kuebler R.R. The importance of beta, the type II error and sample size in the design and interpretation of the randomized control trial. N Engl J Med 1978, 299(13):690-694.
23. Halpern S.D., Karlawish J.H.T., Berlin J.A. The continuing unethical conduct of underpowered clinical trials. JAMA 2002, 288(3):358-362.
24. Aberegg S.K., Richards D.R., O'Brien J.M. Delta inflation: a bias in the design of randomized controlled trials in critical care medicine. Crit Care 2010, 14:R77.
25. Freedman B. Scientific value and validity as ethical requirements for research: a proposed explication. IRB Rev Hum Subj Res 1987, 9(6):7-10.
26. Ioannidis J.P.A. Contradicted and initially stronger effects in highly cited clinical research. JAMA 2005, 294(2):218-228.
27. Ioannidis J.P.A., Haidich A.B., Pappa M., Pantazis N., Kokori S.I., Tektonidou M.G., et al. Comparison of evidence of treatment effects in randomized and nonrandomized studies. JAMA 2001, 286(7):821-830.
28. Concato J., Shah N., Horwitz R.I. Randomized, controlled trials, observational studies, and the hierarchy of research designs. N Engl J Med 2000, 342(25):1887-1892.
29. Pereira T.V., Horwitz R.I., Ioannidis J.P.A. Empirical evaluation of very large treatment effects of medical interventions. JAMA 2012, 308(16):1676-1684.