Méthodologies adaptatives d’études cliniques : quelles sont-elles ? Intérêts et limites pour l’évaluation de nouveaux traitements.

Details

Supervisors

Pas de promoteur

Faculty

Faculté de pharmacie et des sciences biomédicales

Degree label

Master [120] en sciences pharmaceutiques, à finalité spécialisée

Abstract

Adaptive designs in clinical trials allow for modification of a design element in interim analysis with a statistical methodology that controls for Type I error. Adaptive designs studies can offer many advantages when properly conducted. They make better use of accumulated data, saving time and resources, and they limit the number of participants compared to conventional designs with a fixed sample size. They make the trial more ethical, flexible and efficient. Through pre- specification and appropriate adjustment of possible changes during the trial, these benefits are achieved while maintaining the validity and integrity of the trial. However, the implementation of these designs in clinical research remains uncommon. The main reasons for this are limited access to existing studies, as well as a lack of information regarding the several adaptations, their advantages and limitations. First, this master thesis will develop the different adaptation methodologies described in the literature as well as the regulations. Then, the interests and limitations will be discussed. Finally, five clinical trials using the most common adaptive methodologies will be presented to illustrate their uses.