Risk Minimisation Measures of Advanced Therapy Medicinal Products Authorised in the EU Between 2009 and 2023: A Cross-Sectional Study

Sharon C M Essink; Inge M Zomerdijk; Sabine M J M Straus; Helga Gardarsdottir; Marie L De Bruin

doi:10.1007/s40264-025-01550-9

Risk Minimisation Measures of Advanced Therapy Medicinal Products Authorised in the EU Between 2009 and 2023: A Cross-Sectional Study

Drug Saf. 2025 Apr 10. doi: 10.1007/s40264-025-01550-9. Online ahead of print.

Authors

Sharon C M Essink^{1

2}, Inge M Zomerdijk², Sabine M J M Straus^{2

3}, Helga Gardarsdottir^{1

4

5}, Marie L De Bruin⁶

Affiliations

¹ Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
² Department of Pharmacovigilance, Medicines Evaluation Board, Utrecht, The Netherlands.
³ Department of Medical Informatics, Erasmus Medical Centre, Rotterdam, The Netherlands.
⁴ Department of Clinical Pharmacy, University Medical Centre Utrecht, Utrecht, The Netherlands.
⁵ Faculty of Pharmaceutical Sciences, University of Iceland, Reykjavik, Iceland.
⁶ Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands. m.l.debruin@uu.nl.

PMID: 40208555
DOI: 10.1007/s40264-025-01550-9

Abstract

Introduction: Because of the novelty of advanced therapy medicinal products (ATMPs), pro-active risk management is needed post-authorisation; for example, through implementation of additional risk minimisation measures (aRMMs).

Objective: We described which aRMMs were introduced at marketing authorisation (MA) for ATMPs authorised in the European Union (EU), and for what safety concerns.

Methods: We included all ATMPs ever authorised in the EU until December 31, 2023. Data on safety concerns and aRMMs was collected from the European public assessment reports (EPARs) related to initial MA for each ATMP. Safety concerns were categorised using the Medical Dictionary for Regulatory Activities (MedDRA^®) or context of use, where appropriate.

Results: Of the 25 included ATMPs, most (n = 23, 92.0%) were authorised with aRMMs. Of these 23 ATMPs, all (100%) had educational material for healthcare professionals. Additionally, educational material for patients/caregivers was in place for 18 (78.3%) ATMPs and a controlled distribution or controlled access programme for 16 (69.6%). Safety concerns related to 'Long term effects' (n = 23, 92.0%), 'Injury, poisoning and procedural complications' (n = 22, 88.0%), and 'Use in special populations' (e.g., use in pregnancy) (n = 20, 80.0%) were common among all 25 ATMPs. ATMPs often had aRMMs introduced that addressed safety concerns related to 'Injury, poisoning and procedural complications' (n = 19/23; 82.6%), 'General disorders and administration site conditions' (n = 8, 34.8%), and/or 'Immune system disorders' (n = 8, 34.8%).

Conclusion: The majority of ATMPs were authorised with aRMMs. Whilst educational materials were most prevalent, controlled distribution or controlled access programmes were also commonly introduced. For many ATMPs, aRMMs addressed risks related to 'Injury, poisoning and procedural complications'.