STATISTICAL AND METHODOLOGICAL ISSUES IN CLINICAL TRIALS – EXPERIENCE FROM THE REGULATORY SETTING FOR MEDICINES AND MEDICAL DEVICES

In Europe, medicines are regulated by national (and one European) regulatory agencies. In order to introduce a new medicine into the market, drug manufacturers must apply for a license (marketing authorisation) for that medicine. Marketing authorisation applications (MAAs) are usually supported by a number of clinical studies, with the aim of demonstrating safety and efficacy of the product and ensuring that its risk-benefit is positive. In parallel with clinical (and other) aspects of these trials, it is extremely important to ensure the studies are planned and conducted following appropriate statistical methodology, so that their results can be trusted. A biostatistician is, therefore, an essential member of a multidisciplinary team assessing MAAs.