Skip to main content Accessibility help
×
Hostname: page-component-848d4c4894-pjpqr Total loading time: 0 Render date: 2024-06-15T21:26:41.963Z Has data issue: false hasContentIssue false

Chapter 12 - Standards and Guidelines for Adaptive Trial Designs and Master Protocols

from Part IV - Case Studies of Adaptive Trial Designs and Master Protocols

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
Get access

Summary

In this chpater, we review the standards and guidelines for adaptive trial designs and master protocols. Methodological rigour and transparent reporting are required in all clinical trials. Adaptive trial designs and master protocols are no exception. The standard reporting guidelines and risk-of-bias assessments for conventional trials can be applied to adaptive trial designs and master protocols. Adaptive trial designs have pre-planned adaptations and analysis plans that are specified in a formal way that outline how the data will be used to guide the design. Planning of adaptive clinical trials, basket trials, umbrella trials, and platform trials will require early engagement with the stakeholders and methodologists to think through potential hurdles and challenges of the statistical design and its implementation.

Type
Chapter
Information
Publisher: Cambridge University Press
Print publication year: 2023

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Friedman, LM, Furberg, C, DeMets, DL, Reboussin, D, Granger, CB. Fundamentals of Clinical Trials. Springer; 2015.Google Scholar
Chan, AW, Tetzlaff, JM, Altman, DG, et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann Intern Med. 2013;158(3):200207.Google Scholar
Schulz, KF, Altman, DG, Moher, D, Group C. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Trials. 2010;11:32.Google Scholar
Schulz, KF, Altman, DG, Moher, D, Group C. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c332.Google Scholar
Sterne, JAC, Savovic, J, Page, MJ, et al. RoB 2: a revised tool for assessing risk of bias in randomised trials. BMJ. 2019;366:l4898.CrossRefGoogle ScholarPubMed
Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.Google Scholar
Bhatt, DL, Mehta, C. Adaptive designs for clinical trials. N Engl J Med. 2016;375(1):6574.CrossRefGoogle ScholarPubMed
Van Norman, GA. Phase II trials in drug development and adaptive trial design. JACC Basic Transl Sci. 2019;4(3):428–37.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.CrossRefGoogle ScholarPubMed
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.CrossRefGoogle ScholarPubMed
Detry, MA, Lewis, RJ, Broglio, KR, et al. Standards for the design, conduct, and evaluation of adaptive randomized clinical trials. Patient-Centered Outcomes Research Institute (PCORI), 2012.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
Sydes, MR, Parmar, MK, James, ND, et al. Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial. Trials. 2009;10:39.CrossRefGoogle ScholarPubMed
Park, JJH, Harari, O, Dron, L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:18.Google Scholar
Mayer, C, Perevozskaya, I, Leonov, S, et al. Simulation practices for adaptive trial designs in drug and device development. Statistics in Biopharmaceutical Research. 2019;11(4):325–35.CrossRefGoogle 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
He, W, Kuznetsova, OM, Harmer, M, et al. Practical considerations and strategies for executing adaptive clinical trials. DIJ. 2012;46(2):160–74.Google Scholar
Fisher, MR, Roecker, EB, DeMets, DL. The role of an independent statistical analysis center in the institutes of health model. DIJ. 2001;35(1):115–29.Google Scholar
Juszczak, E, Altman, DG, Hopewell, S, Schulz, K. Reporting of multi-arm parallel-group randomized trials: extension of the CONSORT 2010 statement. JAMA. 2019;321(16):1610–20.Google Scholar
Logullo, P, MacCarthy, A, Kirtley, S, Collins, GS. Reporting guideline checklists are not quality evaluation forms: they are guidance for writing. Health Sci Rep. 2020;3(2).Google Scholar
Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019; 20(1):572.CrossRefGoogle ScholarPubMed
Siden, EG, Park, JJ, Zoratti, MJ, et al. Reporting of master protocols towards a standardized approach: a systematic review. Contemp Clin Trials Commun. 2019;15:100406.Google Scholar
Schiavone, F, Bathia, R, Letchemanan, K, et al. This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols. Trials. 2019; 20(1):264.Google Scholar
Morrell, L, Hordern, J, Brown, L, et al. Mind the gap? The platform trial as a working environment. Trials. 2019; 20(1):16.Google Scholar
Hague, D, Townsend, S, Masters, L, et al. Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons. Trials. 2019; 20(1):116.CrossRefGoogle ScholarPubMed
Park, JJH, Hsu, G, Siden, EG, Thorlund, K, Mills, EJ. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020;70(2):125–37.CrossRefGoogle ScholarPubMed
Antoniou, M, Kolamunnage-Dona, R, Wason, J, et al. Biomarker-guided trials: challenges in practice. Contemp Clin Trials Commun. 2019;16:100493.Google Scholar
Park, JJH, Detry, MA, Murthy, S, Guyatt, G, Mills, EJ. How to use and interpret the results of a platform trial: users’ guide to the medical literature. JAMA. 2022;327(1):6774.Google Scholar
Orkin, AM, Gill, PJ, Ghersi, D, et al. Guidelines for reporting trial protocols and completed trials modified due to the COVID-19 pandemic and other extenuating circumstances: the CONSERVE 2021 statement. JAMA. 2021;326(3):257–65.Google Scholar
Angus, DC, Derde, L, Al-Beidh, F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: The REMAP-CAP COVID-19 corticosteroid domain randomized clinical trial. JAMA. 2020;324(13):1317–29.Google Scholar
Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693704.Google Scholar

Save book to Kindle

To save this book to your Kindle, first ensure coreplatform@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Save book to Dropbox

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Dropbox.

Available formats
×

Save book to Google Drive

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Google Drive.

Available formats
×