Perpetual observational studies: new strategies to support efficient implementation of observational studies and randomized trials in infectious diseases
Hassoun-Kheir N, van Werkhoven CH, Dunning J, Jaenisch T, van Beek J, Bielicki J, Butler CC, Francois B, Harbarth S, Hernandez Padilla AC, Horby P, Koopmans M, Lee J, Rodriguez-Baño J, Tacconelli E, Themistocleous Y, van der Velden AW, Bonten M, Goossens H, de Kraker MEA
Background: Emerging infectious diseases are a growing threat, through population growth, increased trade and travel, urbanization, deforestation, and climate change. Clinical research in response to emerging infectious diseases is challenging; selecting and contracting the appropriate study sites for a trial is time consuming, and often too few patients can be timely recruited to acquire high-quality evidence about the best treatment strategies. In 2014, the EU-funded PREPARE project (Platform foR European Preparedness Against (Re-)emerging Epidemics) was initiated to rapidly respond to severe infectious diseases outbreaks. It initiated two adaptive platform trials, the REMAP-CAP and the ALIC4E trial, determining the effectiveness of multiple treatment strategies for a single disease. The REMAP-CAP trial was designed for a pandemic of severe community-acquired pneumonia and expanded globally during the COVID-19 pandemic. It has already delivered 10 important conclusions for better treatment of COVID-19...