Sample Size Re-estimation Designs In Confirmatory Clinical Trials—Current State, Statistical Considerations, and Practical Guidance

Statistics in Biopharmaceutical Research

Volumn 7, Issue 4, 2015, Pages 309-321

  Pritchett, Yili L ^a   Menon, Sandeep ^b   Marchenko, Olga ^c   Antonijevic, Zoran ^c   Miller, Eva ^d   Sanchez Kam, Matilde ^e   Morgan Bouniol, Caroline C ^f   Nguyen, Ha ^g   Prucka, William R ^h  

^a MEDIMMUNE   (United States)

^b BOSTON UNIVERSITY SCHOOL OF PUBLIC HEALTH   (United States)

^c AMGEN INC   (United States)

^d SYNEOS HEALTH   (United States)

^e ARENA PHARMACEUTICALS   (United States)

^f Cytel Incorporated   (United States)

^g PFIZER INC   (United States)

^h ELI LILLY AND COMPANY   (United States)

Author keywords

Adaptive designs; Blinded sample size re estimation; Group sequential designs; Implementation of sample size re estimation designs; Unblinded sample size re estimation

Indexed keywords

ARTICLE; CLINICAL TRIAL (TOPIC); CONFIDENCE INTERVAL; DRUG EFFECT; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; HEALTH CARE ORGANIZATION; HEALTH CARE PLANNING; HUMAN; INFORMATION PROCESSING; SAMPLE SIZE; SAMPLE SIZE REESTIMATION DESIGN; SEQUENTIAL ANALYSIS; STATISTICAL ANALYSIS; STUDY DESIGN;

EID: 84950313552     PISSN: None     EISSN: 19466315     Source Type: Journal    
DOI: 10.1080/19466315.2015.1098564     Document Type: Article

References (62)

1. V.V.Anisimov, (2011), “Predictive Event Modelling in Multicentre Clinical Trials with Waiting Time to Response,” Pharmaceutical Statistics, 10, 517–522.
2. V.V.Anisimov,, and V.V.Fedorov, (2007), “Modelling, Prediction and Adaptive Adjustment of Recruitment in Multicentre Trials,” Statistics in Medicine, 26, 4958–4975.
3. Z.Antonijevic,, P.Gallo,, C.Chuang-Stein,, V.Dragalin,, J.Loewy,, S.Menon,, E.R.Miller,, C.C.Morgan,, and M.Sanchez, (2013), “Views on Emerging Issues Pertaining to Data Monitoring Committees for Adaptive Trials,” Therapeutic Innovation & Regulatory Science, 47, 495–502.
4. P.Bauer,, and K.Köhne, (1994), “Evaluation of Experiments with Adaptive Interim Analyses,” Biometrics, 50, 1029–1041.
5. D.L.Bhatt,, (2013), “Effect of Platelet Inhibition with Cangrelor During PCI on Ischemic Events,” The New England Journal of Medicine, 368:1303–1313.
6. J.Bowden,, and A.Mander, (2014), “A Review and Re-Interpretation of a Group-Sequential Approach to Sample Size Re-Estimation in Two-Stage Trials,” Pharmaceutical Statistics, 13, 163–172.
7. W.Brannath,, C.R.Mehta,, and M.Posch, (2009), “Exact Confidence Bounds Following Adaptive Group Sequential Tests,” Biometrics, 65, 539–546.
8. K.R.Broglio,, J.T.Connor,, and S.M.Berry, (2014), “Not Too Big, Not Too Small: A Goldilocks Approach to Sample Size Selection,” Journal of Biopharmaceutical Statistics, 24, 685–705.
9. Y.H.Chen,, D.L.DeMets,, and K.K.Lan, (2004), “Increasing the Sample Size When the Unblinded Interim Result Is Promising,” Statistics in Medicine, 23, 1023–1038.
10. C.Chuang-Stein,, K.Anderson,, P.Gallo,, and S.Collins, (2006), “Sample Size Re-estimation: A Review and Recommendations,” Drug Information Journal, 40, 475–484.
11. L.Cui,, H.M.J.Hung,, and S.J.Wang, (1999), “Modification of Sample Size in Group Sequential Trials,” Biometrics, 55, 853–857.
12. J.S.Denne, (2001), “Sample Size Recalculation Using Conditional Power,” Statistic in Medicine, 20, 2645–2660.
13. S.S.Emerson,, and T.R.Fleming, (1989), “Symmetric Group Sequential Designs,” Biometrics, 45, 905–923.
14. L.D.Fisher, (1998), “Self-Designing Clinical Trials,” Statistics in Medicine, 17, 1551–1562.
15. T.Friede, and M.Kieser, (2006), “Sample Size Recalculation in Internal Pilot Study Designs: A Review,” Biometrical Journal, 48, 537–555.
16. P.Gallo,, C.Chuang-Stein,, V.Draglin,, B.Gaydos,, M.Krams,, and J.Pinheiro, (2006), “Adaptive Designs in Clinical Drug Development – An Executive Summary of PhRMA Working Group,” Journal of Biopharmaceutical Statistics, 16, 275–283.
17. H.K.Gan,, B.You,, G.R.Pond,, and E.X.Chen, (2012), “Assumptions of Expected Benefits in Randomized Phase III Trials Evaluating Systemic Treatments for Cancer,” Journal National Cancer Institute, 104, 590–598.
18. P.Gao,, L.Liu,, and C.R.Mehta, (2013), “Exact Inference for Adaptive Group Sequential Designs,” Statistics in Medicine, 32, 3991–4005.
19. P.Gao,, J.H.Ware,, and C.R.Mehta, (2008), “Sample Size Re-Estimation for Adaptive Sequential Design in Clinical Trials,” Journal of Biopharmaceutical Statistics, 18, 1184–1196.
20. B.Gaydos,, K.M.Anderson,, D.Berry,, N.Burnham,, C.Chuang-Stein,, J.Dudinak,, P.Fardipour,, P.Gallo,, S.Givens,, R.Lewis,, J.Maca,, J.Pinheiro,, Y.Pritchett,, and M.Krams, (2009), “Good Practices for Adaptive Clinical Trials,” Drug Information Journal, 43, 539–556.
21. E.M.Hade,, D.Jarjoura,, and L.Wei, (2010), “Sample Size Re-Estimation in a Breast Cancer Trial,” Journal of Clinical Trials, 7, 219–226.
22. J.L.Haybittle, (1971), “Repeated Assessment of Results in Clinical Trials of Cancer Treatment,” British Journal of Radiology, 44, 793–797.
23. W.He,, O.M.Kuznetsova,, M.Harmer,, C.Leahy,, K.Anderson,, N.Dossin,, L.Li,, J.Bolognese,, Y.Tymofyeyev,, and J.Schindler, (2012), “Practical Considerations and Strategies for Executing Adaptive Clinical Trials,” Drug Information Journal, 46, 160–174.
24. H.M.J.Hung,, S.J.Wang,, and R.O’Neill, (2012), “Paradigms for Adaptive Statistical Information Designs: Practical Experiences and Strategies,” Statistics in Medicine, 31, 3011–3023.
25. I.K.Hwang,, W.J.Shih,, and J.S.DeCani, (1990), “Group Sequential Designs Using a Family of Type I Error Probability Spending Functions,” Statistics in Medicine, 9, 1439–1445.
26. S.Irle, and H.Shafer, (2012), “Interim Design Modifications in Time-to-Event Studies,” Journal of the American Statistical Association, 107, 341–348.
27. C.Jennison,, and B.W.Turnbull, (2000), Group Sequential Methods with Applications to Clinical Trials, New York: Chapman & Hall.
28. M.Kieser,, and T.Friede, (2003), “Simple Procedures for Blinded Sample Size Adjustment that Do Not Affect the Type I Error Rate,” Statistics in Medicine, 22, 3571–3581.
29. J.M.Kittelson,, and S.S.Emerson, (1999), “A Unifying Family of Group Sequential Test Designs,” Biometrics, 55, 874–882.
30. M.Krams,,C.F.Burman,,V.Dragalin,,B.Gaydos,,A.P.Grieve,,J.Pinheiro,,W.Maurer,, andP.Gallo,, (2006), “Adaptive Designs: Opportunities, Challenges and Scope in Drug Development” in PhRMA/FDA-Sponsored Workshop, Bethesda, MD, November 13–14.
31. J.M.Lachin, (2005), “A Review of Methods for Futility Stopping Based on Conditional Power,” Statistics in Medicine, 24, 2747–2764.
32. K.K.G.Lan,, and D.L.DeMets, (1983), “Discrete Sequential Boundaries for Clinical Trials,” Biometrika, 70, 659–663.
33. W.Lehmacher,, and G.Wassmer, (1999), “Adaptive Sample Size Calculations in Group Sequential Trials,” Biometrics, 55, 1286–1290.
34. D.Magirr,, T.Jaki,, F.Koening, and M.Posch, (2014), “Adaptive Survival Trials,” Technical report, Section of Medical Statistics, Medical University of Vienna, Vienna, Austria.
35. C.R.Mehta,, P.Gao,, D.L.Bhatt,, R.A.Harrington,, S.Skerjanec,, and J.H.Ware, (2009), “Optimizing Trial Designs: Sequential, Adaptive, and Enrichement Strategies,” Circulation, 119, 597–605.
36. C.R.Mehta,, P.Bauer,, M.Posch,, and W.Brannath, (2007), “Repeated Confidence Intervals for Adaptive Group Sequential Trials,” Statistics in Medicine, 26, 5422–5433.
37. C.R.Mehta,, and L.Liu,. (2015), “An Objective Re-Evaluation of Adaptive Sample Size Re-Estimation: Commentary on ‘12 Years of Confirmatory Adaptive Designs,’” Statistics in Medicine, doi: 10.102/sim 6614.
38. C.R.Mehta,, and N.Patel, (2006), “Adaptive, Group Sequential and Decision Theoretical Approaches for Sample Size Determination,” Statistics in Medicine, 25, 3250–3269.
39. C.R.Mehta,, and S.J.Pocock, (2011), “Adaptive Increase in Sample Size When Interim Results Are Promising: A Practical Guide with Examples,” Statistics in Medicine 30, 3267–3284.
40. C.R.Mehta,, and A.A.Tsiatis,. (2001), “Flexible Sample Size Consideration Using Interim Monitoring,” Drug Information Journal, 35, 1095–1112.
41. C.C.Morgan,, S.Huyck,, M.Jenkins,, L.Chen,, A.Bedding,, C.Coffey,, B.Gaydos,, and J.K.Wathen, (2014), “Adaptive Design: Results of 2012 Survey on Perception and Use,” Therapeutic Innovation & Regulatory Science, 48, 473–481.
42. H.H.Müller,, and H.Schäfer, (2001), “Adaptive Group Sequential Designs for Clinical Trials: Combining the Advantages of Adaptive and of Classical Group Sequential Approaches,” Biometrics, 57, 886–891.
43. P.C.O’Brien,, and T.R.Fleming, (1979), “A Multiple Testing Procedure for Clinical Trials,” Biometrics, 35, 549–556.
44. S.Pampallona,, and A.A.Tsiatis, (1994), “Group Sequential Designs for One-Sided and Two-Sided Hypothesis Testing with Provision for Early Stopping in Favor of the Null Hypothesis,” Journal of Statistical Planning and Inference, 42, 19–35.
45. R.Peto,, M.C.Pike,, P.Armitage,, N.E.Breslow,, D.R.Cox,, S.V.Howard,, N.Mantel,, K.McPherson,, J.Peto,, and P.G.Smith, (1976), “Design and Analysis of Randomized Clinical Trials Requiring Prolonged Observation of Each Patient: I. Introduction and Design,” British Journal of Cancer, 34, 585–612.
46. S.J.Pocock, (1977), “Group Sequential Methods in the Design and Analysis of Clinical Trials,” Biometrika, 64, 191–199.
47. Y.Pritchett,, Y.Jemiai,, Y.Chang,, I.Bhan,, R.Agarwal,, C.Zoccali,, C.Wanner,, D.Lloyd-Jones,, J.B.Cannata-Andía,, T.Thompson,, E.Appelbaum,, P.Audhya,, D.Andress,, S.Solomon,, W.J.Manning,, and R.Thadhani, (2011), “A Group Sequential, Information-Based Sample Size Re-estimation Design of the PRIMO (Paricalcitol Capsules Benefits in Renal Failure-Induced Cardiac Morbidity in Chronic Kidney Disease) Study,” Journal of Clinical Trials, 8, 166–174.
48. M.Proschan, (2009), “Sample Size Re-Estimation in Clinical Trials,” Biometrical Journal, 51, 348–357.
49. M.A.Proschan,, and S.A.Hunsberger,. (1995). “Designed Extension of Studies Based on Conditional Power,” Biometrics, 51, 1315–1324.
50. J.A.Quinlan,, B.Gaydos,, J.Maca,, and M.Krams, (2010), “Barriers and Opportunities for Implementation of Adaptive Designs in Pharmaceutical Product Development,” Clinical Trials, 7, 167–173.
51. J.A.Quinlan,, and M.Krams, (2006), “Implementing Adaptive Designs: Logistical and Operational Considerations,” Drug Information Journal, 40, 437–444.
52. Spiegelhalter, D. J., Abrams, K. R., and Myles, J. P. (2004), Bayesian Approaches to Clinical Trials and Health Care Evaluation, West Sussex, UK: Wiley.
53. C.Stein, (1945), “A Two-Sample Test for a Linear Hypothesis Whose Power Is Independent of the Variance,” Annals of Mathematical Statistics, 16, 243–258.
54. A.C.Tamhane,, Y.Wu,, and C.Mehta, (2012a), “Adaptive Extensions of a Two-Stage Group Sequential Procedure for Testing Primary and Secondary Endpoints (I): Unknown Correlation Between the Endpoints,” Statistics in Medicine, 31, 2027–2040.
55. ——— (2012b), “Adaptive Extensions of a Two-Stage Group Sequential Procedure for Testing Primary and Secondary Endpoints (II): Sample Size Re-Estimation,” Statistics in Medicine, 31, 2041–2054.
56. A.A.Tsiatis,, and C.R.Mehta, (2003), “On the Inefficiency of the Adaptive Design for Monitoring Clinical Trials,” Biometrika, 90, 367–378.
57. A.A.Tsiatis,, G.L.Rosner,, and C.R.Mehta, (1984), “Exact Confidence Intervals Following a Group Sequential Test,” Biometrics, 40, 797–803.
58. U.S. Food and Drug Administration (FDA) (February 2010), Draft Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics. Available at http://www.fda.gov/downloads/DrugsGuidanceComplianceRegulatoryInformation/Guidances/UCM201790.pdf
59. ——— (May 2015), Draft Guidance for Industry and Food and Drug Administration Staff: Adaptive Designs for Medical Device Clinical Studies. Available at http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm446729.pdf
60. J.Wang, (2010), “Many-to-One Comparison After Sample Size Reestimation for Trials with Multiple Treatment Arms and Treatment Selection,” Journal of Biopharmaceutical Statistics, 20, 927–940.
61. S.K.Wang,, and A.A.Tsiatis, (1987), “Approximately Optimal One-Parameter Boundaries for Group Sequential Trials,” Biometrics, 43, 193–200.
62. D.M.Zucker,, J.T.Wittes,, O.Schabenberger,, and E.Brittain,. (1999), “Internal Pilot Studies II: Comparison of Various Procedures,” Statistics in Medicine, 18, 3493–3509.