Post-marketing safety and efficacy surveillance of cell and gene therapies in the EU: A critical review

Published: 18 November 2019
Expert Insight
Enrico Fritsche,
Enrico Fritsche
Project Manager, Clinical Development at Charité - University Medicine Berlin
Magdi Elsallab,
Magdi Elsallab
Doctoral Student at Berlin-Brandenburg Center for Regenerative Therapies (BCRT)
Michaela Schaden,
Michaela Schaden
Project Manager, Clinical Trials Coordination Center at Medical University of Vienna
Spencer Phillips Hey,
Spencer Phillips Hey
Mohamed Abou-El-Enein
Mohamed Abou-El-Enein

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.

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