Watch Module 5 of Adaptive Trial Designs series: platform trials

Module 5 of Ecraid's six-part training series on Adaptive Clinical Trial Designs is now online. As usual, it's available for free with a myERS account.

In this module, we go over the nuts and bolts of platform trials, including the novel Personalised Randomised Controlled Trial (PRACTical) design. Our esteemed speakers use several high-profile trials – RECOVERY, REMAP-CAP, and NeoSep1 – to illustrate the benefits of platform designs, particularly during pandemics.

Big thanks to Pavel Mozgunov, Thomas Jaki, Lennie Derde, Sarah Walker, and Julia Bielicki for their insightful presentations.

5.1 Introduction to platform trials

In this lecture, we introduce the concept of platform trials and discuss important statistical considerations when designing such trials. Special attention is paid to the type I error adjustments needed and various definitions of power that can be used. The lecture concludes with a discussion on planned and unplanned changes to the platform and the use of a shared and concurrent control arm.

Presented by Dr Pavel Mozgunov, Programme Leader Track at the University of Cambridge's MRC Biostatistics Unit.

5.2 Platform trial case study: RECOVERY

The need for quick evaluation of treatments during the COVID-19 pandemic led to platform trials being used more frequently. The RECOVERY trial is one of the most influential COVID-19 platform trials conducted so far. Next to an overview of the trial, we share lessons learned for research during pandemics.

Presented by Thomas Jaki, Professor of Computational Statistics at the University of Regensburg and Professor of Statistics/Programme Leader at the MRC Biostatistics Unit at the University of Cambridge.

5.3 Platform trial case study: REMAP-CAP

We take a close look at the history of the global REMAP-CAP trial and its main design elements, focusing on two key features: its multifactorial design and the adaptive aspect. The lecture also features an overview of recent developments and results generated in the trial.

Presented by Dr Lennie Derde, ICU consultant and Assistant Professor at University Medical Centre Utrecht.

5.4 Introduction to the PRACTical design

The Personalised Randomised Controlled Trial (PRACTical) is a novel platform design with distinct advantages and disadvantages over standard platform designs. In this lecture, we zoom in on its particular approach to analysis, interpretation, and sample size calculation. We also advise on the situations for which the PRACTical design is best suited.

Presented by (Ann) Sarah Walker, Professor of Medical Statistics and Epidemiology at the Nuffield Department of Medicine at the University of Oxford, as well as Professor of Medical Statistics at the MRC Clinical Trials Unit at University College London

5.5 PRACTical case study: NeoSep1

The PRACTical design is brought to life through the GARDP-sponsored NeoSep1 trial. We explain the rationale for choosing this design in combination with a sequential multiple assignment randomisation in the context of investigating empiric antibiotic treatment of neonatal sepsis in settings with high ESBL-Gram-negative prevalence. Key considerations around operationalising the PRACTical design and their role within NeoSep1 are discussed.

Presented by Dr Julia Bielicki, consultant in Paediatric Infectious Diseases, Senior Physician at University of Basel Children’s Hospital, and paediatrician with the Paediatric Infectious Diseases Research Group at St George’s University of London.

Module 6 – the last in this series – is coming on 29 November. It addresses ethical, administrative, regulatory, and legal challenges. Stay informed about Module 6, as well as our other courses, workshops, and webinars by subscribing for Ecraid's Education newsletter.