Skip to main content Accessibility help

We use cookies to distinguish you from other users and to provide you with a better experience on our websites. Close this message to accept cookies or find out how to manage your cookie settings.

Close cookie message
×
Hostname: page-component-848d4c4894-x24gv Total loading time: 0 Render date: 2024-04-30T15:01:32.485Z Has data issue: false hasContentIssue false

Chapter 2 - History of Clinical Trial Research

from Part I - Introduction and History of Clinical Trial Research

Published online by Cambridge University Press:  20 March 2023

Jay J. H. Park ,
Edward J. Mills  and
J. Kyle Wathen
By
Jay J. H. Park ,
J. Kyle Wathen  and
Edward J. Mills
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 chapter, we review the history of clinical trials. We review the famous 1948 streptomycin trial for tuberculosis to highlight how the discipline of randomised clinical trials was born out of economic hardship. Other historical events of adaptive trial designs and master protocols are discussed in this chapter.

Keywords

History of clinical trialsECMO trialplay the winneradaptive trial designsmaster protocols1948 streptomycin trialFDAprecision medicine
Type
Chapter
Information
Introduction to Adaptive Trial Designs and Master Protocols , pp. 15 - 20
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

Bothwell, LE, Podolsky, SH. The emergence of the randomized, controlled trial. N Engl J Med. 2016;375(6):501–4.CrossRefGoogle ScholarPubMed
Jones, DS, Podolsky, SH. The history and fate of the gold standard. Lancet. 2015;385(9977):1502–3.Google Scholar
Hill, AB. Suspended judgment. Memories of the British Streptomycin Trial in Tuberculosis. The first randomized clinical trial. Control Clin Trials. 1990;11(2):77–9.Google Scholar
Hill, AB. Principles of Medical Statistics. 1st ed. Lancet. 1937.Google Scholar
Hill, AB. I. – The aim of the statistical method. Lancet. 1937;229(5914):41–3.CrossRefGoogle Scholar
Crofton, J. The MRC randomized trial of streptomycin and its legacy: a view from the clinical front line. J R Soc Med. 2006;99(10):531–4.CrossRefGoogle ScholarPubMed
Schatz, A, Waksman, SA. Effect of streptomycin and other antibiotic substances upon Mycobacterium tuberculosis and related organisms. Proc Soc Exp Biol Med. 1944;57(2):244–8.CrossRefGoogle Scholar
Bartlett, RH, Roloff, DW, Cornell, RG, et al. Extracorporeal circulation in neonatal respiratory failure: a prospective randomized study. Pediatrics. 1985;76(4):479–87.CrossRefGoogle ScholarPubMed
Angus, DC. Optimizing the trade-off between learning and doing in a pandemic. JAMA. 2020;323(19):1895–6.Google Scholar
Zelen, M. Play the winner rule and the controlled clinical trial. J Am Stat Assoc. 1969;64(325):131–46.Google Scholar
Wei, L, Durham, S. The randomized play-the-winner rule in medical trials. J Am Stat Assoc. 1978;73(364):840–3.Google Scholar
O’Rourke, PP, Crone, RK, Vacanti, JP, et al. Extracorporeal membrane oxygenation and conventional medical therapy in neonates with persistent pulmonary hypertension of the newborn: a prospective randomized study. Pediatrics. 1989;84(6):957–63.Google Scholar
UK Collaborative ECMO Trial Group. UK Collaborative Randomised Trial of Neonatal Extracorporeal Membrane Oxygenation. Lancet. 1996;348(9020):75–82.Google Scholar
Kumar-Sinha, C, Chinnaiyan, AM. Precision oncology in the age of integrative genomics. Nat Biotechnol. 2018;36(1):46–60.Google Scholar
Ke, X, Shen, L. Molecular targeted therapy of cancer: the progress and future prospect. Front Lab Med. 2017;1(2):69–75.CrossRefGoogle Scholar
Redman, MW, Allegra, CJ. The master protocol concept. Semin Oncol. 2015;42(5):724–30.CrossRefGoogle ScholarPubMed
Hirakawa, A, Asano, J, Sato, H, Teramukai, S. Master protocol trials in oncology: review and new trial designs. Contemp Clin Trials Commun. 2018;12:1–8.CrossRefGoogle ScholarPubMed
U.S. Department of Health and Human Services, U.S. Food and Drug Administration. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry (Draft Guidance). United States Department of Health and Human Services; 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM621817.pdfGoogle Scholar
Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med. 2017;377(1):62–70.CrossRefGoogle ScholarPubMed
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.Google Scholar
Bogin, V. Master protocols: new directions in drug discovery. Contemp Clin Trials Commun. 2020;18:100568.Google Scholar
Vanderbeek, AM, Bliss, JM, Yin, Z, Yap, C. Implementation of platform trials in the COVID-19 pandemic: a rapid review. Contemp Clin Trials. 2022;112:106625.Google Scholar
Park, JJ, Mogg, R, Smith, GE, et al. How COVID-19 has fundamentally changed clinical research in global health. Lancet Glob Health. 2021;9(5):e711–e20.Google Scholar
Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.CrossRefGoogle ScholarPubMed
Armitage, P. Sequential medical trials. Biomedicine. 1978;28 Spec No:40–1.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
Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med. 2017; 377(1):62–70.CrossRefGoogle ScholarPubMed

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
×