Post-marketing Safety and Efficacy Surveillance of Cell and Gene Therapies in the EU: A Critical Review

Published: 2019
Cell & Gene Therapy Insights 2019; 5(11), 1505-1521


The implementation of new regulatory tools, such as the PRIority MEdicine (PRIME) scheme, by regulatory authorities in Europe enabled faster patient access to innovative therapies. This early access tool goes along with a clear need for a thorough assessment of safety and efficacy upon marketing authorization. Due to the higher degree of uncertainty when evaluating novel therapies such as advanced therapy medicinal products (ATMPs), post-marketing surveillance studies for these products should be designed to make up the evidential shortfall and provide additional evidence to inform clinical practice. Here, we describe the status and regulatory requirements of post-marketing surveillance for ATMPs, which we found often resembling traditional, pre-market trials, focusing on biological mechanisms and efficacy in narrowly defined patient populations. We close by proposing the pragmatic trial concept as a potential solution to improve data quality and evidence generation in settings closer to real-world.

Enrico Fritsche
Magdi Elsallab
Michaela Schaden
Spencer Phillips Hey
Mohamed Abou El-Enein