Skip to Main Content
HBS Home
  • About
  • Academic Programs
  • Alumni
  • Faculty & Research
  • Baker Library
  • Giving
  • Harvard Business Review
  • Initiatives
  • News
  • Recruit
  • Map / Directions
Faculty & Research
  • Faculty
  • Research
  • Featured Topics
  • Academic Units
  • …→
  • Harvard Business School→
  • Faculty & Research→
Publications
Publications
  • October 2019
  • Article
  • Nature Reviews: Drug Discovery

Adaptive Platform Trials: Definition, Design, Conduct and Reporting Considerations

By: Derek C. Angus, Brian M. Alexander, Scott Berry, Meredith Buxton, Roger Lewis, Melissa Paoloni, Steven A. R. Webb, Steven Arnold, Anna Barker, Donald A. Berry, Marc J. M. Bonten, Mary Brophy, Christopher Butler, Timothy F. Cloughesy, Lennie P. G. Derde, Laura J. Esserman, Ryan Ferguson, Louis Fiore, Sarah C. Gaffey, J. Michael Gaziano, Kathy Giusti, Herman Goossens, Stephane Heritier, Bradley Hyman, Michael Krams, Kay Larholt, Lisa M. LaVange, Philip Lavori, Andrew W. Lo, Alexander J. London, Victoria Manax, Colin McArthur, Genevieve O’Neill, Giovanni Parmigiani, Jane Perlmutter, Elizabeth A. Petzold, Craig Ritchie, Kathryn M. Rowan, Christopher W. Seymour, Nathan I. Shapiro, Diane M. Simeone, Bradley Smith, Bradley Spellberg, Ariel Dora Stern, Lorenzo Trippa, Mark Trusheim, Kert Viele, Patrick Y. Wen and Janet Woodcock
  • Format:Print
ShareBar

Abstract

Researchers, clinicians, policymakers, and patients are increasingly interested in questions about therapeutic interventions that are difficult or costly to answer with traditional, free-standing, parallel-group randomized controlled trials (RCTs). Examples include scenarios in which there is a desire to compare multiple interventions, to generate separate effect estimates across subgroups of patients with distinct but related conditions or clinical features, or to minimize downtime between trials. In response, researchers have proposed new RCT designs such as adaptive platform trials (APTs), which are able to study multiple interventions in a disease or condition in a perpetual manner, with interventions entering and leaving the platform on the basis of a predefined decision algorithm. APTs offer innovations that could reshape clinical trials, and several APTs are now funded in various disease areas. With the aim of facilitating the use of APTs, here we review common features and issues that arise with such trials and offer recommendations to promote best practices in their design, conduct, oversight, and reporting.

Keywords

Adaptive Platform Trials; Health Testing and Trials

Citation

Angus, Derek C., Brian M. Alexander, Scott Berry, Meredith Buxton, Roger Lewis, Melissa Paoloni, Steven A. R. Webb, Steven Arnold, Anna Barker, Donald A. Berry, Marc J. M. Bonten, Mary Brophy, Christopher Butler, Timothy F. Cloughesy, Lennie P. G. Derde, Laura J. Esserman, Ryan Ferguson, Louis Fiore, Sarah C. Gaffey, J. Michael Gaziano, Kathy Giusti, Herman Goossens, Stephane Heritier, Bradley Hyman, Michael Krams, Kay Larholt, Lisa M. LaVange, Philip Lavori, Andrew W. Lo, Alexander J. London, Victoria Manax, Colin McArthur, Genevieve O’Neill, Giovanni Parmigiani, Jane Perlmutter, Elizabeth A. Petzold, Craig Ritchie, Kathryn M. Rowan, Christopher W. Seymour, Nathan I. Shapiro, Diane M. Simeone, Bradley Smith, Bradley Spellberg, Ariel Dora Stern, Lorenzo Trippa, Mark Trusheim, Kert Viele, Patrick Y. Wen, and Janet Woodcock. "Adaptive Platform Trials: Definition, Design, Conduct and Reporting Considerations." Nature Reviews: Drug Discovery 18, no. 10 (October 2019): 797–807.
  • Find it at Harvard
  • Read Now

More from the Authors

    • June 2024
    • Faculty Research

    Maven Clinic: Women's Health in the Digital Age

    By: Ariel D. Stern and Sarah Mehta
    • June 2024
    • Faculty Research

    Aidoc: Building a Hospital-Centric AI Platform

    By: Ariel D. Stern and Susan Pinckney
    • May 2024
    • Clinical Pharmacology & Therapeutics

    Tepid Uptake of Digital Health Technologies in Clinical Trials by Pharmaceutical and Medical Device Firms

    By: Caroline Marra and Ariel D. Stern
More from the Authors
  • Maven Clinic: Women's Health in the Digital Age By: Ariel D. Stern and Sarah Mehta
  • Aidoc: Building a Hospital-Centric AI Platform By: Ariel D. Stern and Susan Pinckney
  • Tepid Uptake of Digital Health Technologies in Clinical Trials by Pharmaceutical and Medical Device Firms By: Caroline Marra and Ariel D. Stern
ǁ
Campus Map
Harvard Business School
Soldiers Field
Boston, MA 02163
→Map & Directions
→More Contact Information
  • Make a Gift
  • Site Map
  • Jobs
  • Harvard University
  • Trademarks
  • Policies
  • Accessibility
  • Digital Accessibility
Copyright © President & Fellows of Harvard College.