Journal Article

The statistical design and analysis of pandemic platform trials: Implications for the future

Grund, Birgit, Geller, Nancy, LaVange, Lisa, Berry, Scott, Lindsell, Christopher J., Jagannathan, Prasanna, Proschan, Michael, Stewart, Thomas, Leifer, Eric, Touloumi, Giota, Brittain, Erica, Thomas, Sonia, Anstrom, Kevin J., Price, Karen L., Hughes, Michael D., Harrell, Frank E., Moser, Carlee B., Shotwell, Matthew

2024

Overview

The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Cross-Trial Statistics Group gathered lessons learned from statisticians responsible for the design and analysis of the 11 ACTIV therapeutic master protocols to inform contemporary trial design as well as preparation for a future pandemic. The ACTIV master protocols were designed to rapidly assess what treatments might save lives, keep people out of the hospital, and help them feel better faster. Study teams initially worked without knowledge of the natural history of disease and thus without key information for design decisions. Moreover, the science of platform trial design was in its infancy. Here, we discuss the statistical design choices made and the adaptations forced by the changing pandemic context. Lessons around critical aspects of trial design are summarized, and recommendations are made for the organization of master protocols in the future.

• Publisher: Cambridge University Press
• Subject: Clinical trials / COVID-19 / COVID-19 Therapeutic Platform Trials Lessons Learned / Design / Hospitalization / Mortality / pandemic preparedness / Pandemics / platform trials / research design / Special Communication / Statistical analysis / Statistics / Teams / Therapeutic applications / Working groups