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Chapter 3 - Characteristics and Principles of Adaptive Trial Designs

from Part II - Basic Ingredients for Adaptive Trial Designs and Common Types

Published online by Cambridge University Press:  20 March 2023

Jay J. H. Park
Affiliation:
McMaster University, Ontario
Edward J. Mills
Affiliation:
McMaster University, Ontario
J. Kyle Wathen
Affiliation:
Cytel, Cambridge, Massachusetts
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Summary

This chapter discusses the property and principles of adaptive trial designs. Adaptive trial designs refer to trial designs that offer pre-planned opportunities to modify the design of an ongoing trial based on accumulating trial data. Decisions for potential adaptations are made during the trial based on interim data, but flexibilities in adaptive trial designs are established and outlined in the study documents before any patient is recruited. For statistical planning, a simulation-guided approach is often used to evaluate the statistical properties of the design. It is generally required to demonstrate control of false positive rates for the regulatory, ethics, and funding bodies. Measures to mitigate and plan for operational bias and complexity in adaptive trial designs are needed.

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Publisher: Cambridge University Press
Print publication year: 2023

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References

Dimairo, M, Pallmann, P, Wason, J, et al. The Adaptive Designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. BMJ. 2020;369 :m115.Google Scholar
Dimairo, M, Pallmann, P, Wason, J, et al. The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design. Trials. 2020;21(1):528.Google Scholar
Thorlund, K, Haggstrom, J, Park, JJ, Mills, EJ. Key design considerations for adaptive clinical trials: a primer for clinicians. BMJ. 2018;360:k698.CrossRefGoogle ScholarPubMed
Pallmann, P, Bedding, AW, Choodari-Oskooei, B, et al. Adaptive designs in clinical trials: why use them, and how to run and report them. BMC Med. 2018;16(1):29.Google Scholar
ICH Harmonised Tripartite Guideline. Statistical principles for clinical trials. International Conference on Harmonisation E9 Expert Working Group. Stat Med. 1999;18(15):1905–42.Google Scholar
U.S. Food and Drug Administration. The Establishment and Operation of Clinical Trial Data Monitoring Committees for Clinical Trial Sponsors. United States Department of Health and Human Services; 2006.Google Scholar
Bhatt, DL, Mehta, C. Adaptive designs for clinical trials. N Engl J Med. 2016;375(1):6574.Google Scholar
Park, JJ, Thorlund, K, Mills, EJ. Critical concepts in adaptive clinical trials. Clin Epidemiol. 2018;10:343–51.Google Scholar
Fogel, DB. Factors associated with clinical trials that fail and opportunities for improving the likelihood of success: a review. Contemp Clin Trials Commun. 2018;11:156–64.Google Scholar
Tehranisa, JS, Meurer, WJ. Can response-adaptive randomization increase participation in acute stroke trials? Stroke. 2014;45(7):2131–3.CrossRefGoogle ScholarPubMed
Florey, CD. Sample size for beginners. BMJ. 1993;306(6886):1181–4.Google Scholar
Hummel, J, Wang, S, Kirkpatrick, J. Using simulation to optimize adaptive trial designs: applications in learning and confirmatory phase trials. Clin Invest. 2015;5(4):401–13.CrossRefGoogle Scholar
Detry, MA, Lewis, RJ, Broglio, KR, Standards for the design, conduct, and evaluation of adaptive randomized clinical trials. Patient-Centered Outcomes Research Institute (PCORI); 2012.Google Scholar
Sanchez-Kam, M, Gallo, P, Loewy, J, et al. A practical guide to data monitoring committees in adaptive trials. Ther Innov Regul Sci. 2014;48(3):316–26.Google Scholar
U.S. Food and Drug Administration. Adaptive Designs for Medical Device Clinical Studies Guidance for Industry and Food and Drug Administration Staff. United States Department of Health and Human Services; 2016.Google Scholar
United States Department of Health and Human Services, Food and Drug Administration. Adaptive Designs for Clinical Trials of Drugs and Biologics. Guidance for Industry. Center for Biologics Evaluation and Research (CBER); 2019.Google Scholar
Bauer, P, Bretz, F, Dragalin, V, Konig, F, Wassmer, G. Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Stat Med. 2016;35(3):325–47.Google Scholar
Burton, A, Altman, DG, Royston, P, Holder, RL. The design of simulation studies in medical statistics. Stat Med. 2006;25(24):4279–92.Google Scholar

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