Platform trials

Matthieu Roustit (1) , Olivier Demarcq (2) , Silvy Laporte (3) , Philippe Barthélémy (4) , Olivier Chassany (5) , Michel Cucherat (6) , Jacques Demotes (7) , Vincent Diebolt (8) , Hélène Espérou (9) , Cécile Fouret (10) , Ariane Galaup (11) , Laetitia Gambotti (12) , Charlotte Gourio (13) , Aurélie Guérin (2) , Carine Labruyère (3) , Xavier Paoletti (14) , Raphaël Porcher (15, 16, 17) , Tabassome Simon (18) , Nathalie Varoqueaux (11)

1 CIC Grenoble - Centre d'Investigation Clinique [Grenoble]
2 Pfizer France
3 SAINBIOSE - Santé Ingénierie Biologie Saint-Etienne
4 IRFAC - Inserm U1113 - Interface de Recherche Fondamentale et Appliquée en Cancérologie
5 ECEVE (U1123 / UMR_S_1123) - Epidémiologie Clinique et Evaluation Economique Appliquées aux Populations Vulnérables
6 LBBE - Laboratoire de Biométrie et Biologie Evolutive - UMR 5558
7 ECRIN - European Clinical Research Infrastructures Network [Paris]
8 Plateforme F-CRIN
9 PRC - Pôle de Recherche Clinique [Paris]
10 Medtronic SNT
11 Auteur indépendant
12 INCa - Institut national du cancer
13 CHU Caen
14 Cancer et génome: Bioinformatique, biostatistiques et épidémiologie d'un système complexe
15 CRESS (U1153 / UMR_A 1125) - Centre for Research in Epidemiology and Statistics | Centre de Recherche Épidémiologie et Statistiques
16 METHODS [CRESS - U1153 / UMR_A 1125] - Methods of therapeutic evaluation of chronic diseases | Méthodes de l’évaluation thérapeutique des maladies chroniques
17 Centre d'épidémiologie Clinique [Hôtel-Dieu]
18 SU - Sorbonne Université

For the past few years, platform trials have experienced a significant increase, recently amplified by the COVID-19 pandemic. The implementation of a platform trial is par-ticularly useful in certain pathologies, particularly when there is a significant number of drug candidates to be assessed, a rapid evolution of the standard of care or in situations of urgent need for evaluation, during which the pooling of protocols and infrastructure optimizes the number of patients to be enrolled, the costs, and the deadlines for carrying out the investiga-tion. However, the specificity of platform trials raises methodological, ethical, and regulatory issues, which have been the subject of the round table and which are presented in this article. The round table was also an opportunity to discuss the complexity of sponsorship and data management related to the multiplicity of partners, funding, and governance of these trials, and the level of acceptability of their findings by the competent authorities.

Mots clés: Platform trial; Adaptive trial; Randomized controlled trial

Domaines: Santé publique et épidémiologie; Méthodologie [stat.ME]

Matthieu Roustit, Olivier Demarcq, Silvy Laporte, Philippe Barthélémy, Olivier Chassany, et al.. Platform trials. Therapies, 2023, 78 (1), pp.29-38. ⟨10.1016/j.therap.2022.12.003⟩. ⟨hal-04249795⟩