Platform Trial Designs

Oleksandr Sverdlov (), Ekkehard Glimm () and Peter Mesenbrink ()
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Oleksandr Sverdlov: Novartis Pharmaceuticals Corporation
Ekkehard Glimm: Novartis Pharma AG
Peter Mesenbrink: Novartis Pharmaceuticals Corporation

Chapter 76 in Principles and Practice of Clinical Trials, 2022, pp 1455-1485 from Springer

Abstract: Abstract Modern drug development is increasingly complex and requires novel approaches to the design and analysis of clinical trials. With the precision medicine paradigm, there is a strong need to evaluate multiple experimental therapies across a spectrum of indications, in different subgroups of patients, while controlling the chance of false positive and false negative findings. The concept of master protocols provides a new approach to clinical trial design that can help drug developers to enhance efficiency of clinical trials by addressing multiple research questions within the same overall trial infrastructure. There are three general types of trials requiring a master protocol: basket trials, umbrella trials, and platform trials. The present chapter provides an overview of platform trial designs. We discuss operating models for implementing platform trials in practice, as well as some important statistical considerations for design and analysis of such trials. We also discuss four real-life examples of platform trials: the EPAD-PoC study in Alzheimer’s disease; the I-SPY COVID-19 study for rapid screening of re-purposed and novel treatments for COVID-19; the GBM AGILE study in glioblastoma; and the FOCUS4 study in metastatic colorectal cancer.

Keywords: Master protocols; Multi-arm randomized controlled trials; Multiple comparisons (search for similar items in EconPapers)
Date: 2022
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DOI: 10.1007/978-3-319-52636-2_107

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