Newly approved agents for relapsing remitting multiple sclerosis: how real-world evidence compares with randomized clinical trials?

ABSTRACT

INTRODUCTION: In recent years, many treatment options have become available for relapsing remitting MS. Randomized clinical trials and real-world studies are complementary sources of information, and together have the potential to offer a comprehensive understanding of the safety and efficacy profiles of each drug, a critical factor for a personalized management of the disease. AREAS COVERED In this review, the authors provide an up-to-date review of both RCTs and real-world studies assessing the safety and efficacy profiles of recently developed disease-modifying drugs for relapsing remitting MS. These include fingolimod, teriflunomide, dimethyl fumarate, alemtuzumab and ocrelizumab. EXPERT OPINION From the authors' review of the literature, the efficacy profiles resulted from RCTs were confirmed by observational studies with regard to the disease-modifying drugs considered. The magnitude of the effects on annualized relapse rates and MRI active lesions was generally even larger in the observational studies compared to RCTs. From the safety point of view, observational studies revealed new adverse events, mostly in the area of bacterial and opportunistic infections, not seen in the relative registration programme. This is a very important gain because it allows to elaborate appropriate strategies to prevent and handle the risks.