• Langue : Anglais, Français
• Discipline : Pharmacie
• Identifiant : 2019LILUE193
• Type de thèse : Doctorat de pharmacie
• Date de soutenance : 04/07/2019

Résumé en langue originale

Afin d'améliorer la façon dont les nouveaux médicaments sont recherchés et mis au point, l'accent a été mis ces dernières années sur l'utilisation d'essais cliniques innovants à toutes les phases du développement d'un médicament.

Résumé traduit

To improve how new medicines are researched and developed, there has been a focus in recent years to use innovative clinical trials (basket or bucket trial, umbrella trial, platform trials) throughout all phases of drug development. Although these types of clinical trials are gaining significant momentum in oncology and beyond allowing conduct of clinical developments in a most efficient and effective manner, the uptake in EU has been rather shy, especially in registration trials context. Key factors that have reduced the use of more complex designs in EU include a lack of understanding of innovative design strategies and statistical methodologies by Health Authorities and concerns by the Industry on the acceptability of the evidence generated via these trials by Regulators and HTA. Promoting awareness and understanding across stakeholders of such new approaches is essential to ensure that patients can benefit from the ongoing advances in the drug development landscape (innovative designs, personalised medicines, genomics and biomarkers, decentralised trials, digital and eHealth…). It is therefore crucial to explore how the pharmaceutical industry and drug makers can engage with regulators to discuss on issues and challenges faced by the various stakeholders, to align on acceptable approaches to meet regulatory standards for decision-making and agree on a path forward to bring innovative solutions to patients.