Platform Trial Designs in Drug Development

Platform trials test multiple therapies in one indication, one therapy for multiple indications, or both. These novel clinical trial designs can dramatically increase the cost-effectiveness of drug development, leading to life-altering medicines for people suffering from serious illnesses, possibly at lower cost. Currently, the cost of drug development is unsustainable. Furthermore, there are particular problems in rare diseases and small biomarker defined subsets in oncology, where the required sample sizes for traditional clinical trial designs may not be feasible.


The editors recruited the key innovators in this domain. The 20 articles discuss trial designs from perspectives as diverse as quantum computing, patient's rights to information, and international health.


The book begins with an overview of platform trials from multiple perspectives. It then describes impacts of platform trials on the pharmaceutical industry's key stakeholders: patients, regulators, and payers. Next it provides advanced statistical methods that address multiple aspects of platform trials, before concluding with a pharmaceutical executive's perspective on platform trials. Except for the statistical methods section, only a basic qualitative knowledge of clinical trials is needed to appreciate the important concepts and novel ideas presented.

I. Overview of Platform Clinical TrialsIntroductionRobert A. Beckman and Zoran AntonijevicI-SPY2: Unlocking the potential of the platform trialLaura Esserman, Nola Hylton, Smita Asare, Christina Yau, Doug Yee, Angie Demichele, Jane Perlmutter, Fraser Symmans, Laura van't Veer, Jeff Matthews, Don Berry, and Anna BarkerThe challenges with Multi-arm targeted therapy trialsRyan J. Sullivan and Keith Flaherty. Basket trials at the confirmatory stageRobert A. Beckman and Cong ChenHarnessing Real World Data to Inform Platform Trial DesignDaphne A. Guinn, Subha Madhavan, and Robert A. BeckmanImpact of Platform Trials on Pharmaceutical FrameworksZoran Antonijevic, Edward Mills, Jonas Häggström, and Kristian Thorlund. II. StakeholdersFriends of Cancer Research Perspective on Platform trialsJeff D. Allen, Madison Wempe, Ryan Hohman, and Ellen Sigal Regulatory and Policy aspects of Platform trialsRasika Kalamegham, Ramzi Dagher, and Peter Honig Multi-arm, multi-drug trials from a reimbursement perspectiveAnja Schiel, and Olivier CollignonHighly Efficient Clinical Trials: A resource-saving solution for global healthEdward Mills, J