(CSPOT43) Risk Management of Authorised Advanced Therapy Medicinal Products in the EU

Background: Advanced therapy medicinal products (ATMPs) comprise innovative gene therapy, somatic cell therapy, and tissue based products often used for orphan diseases. Because of ATMPs’ novelty, uncertainty about their efficacy and safety at time of marketing authorisation (MA) approval exists. For these reasons, pro-active risk management of ATMPs post-marketing is needed.

Objectives: We assessed which additional risk minimisation measures (aRMMs) are in place at MA for ATMPs authorised in the European Union (EU), including the safety concerns addressed.

Methods: We included ATMPs authorised in the EU until 31 December 2023 using the ‘Committee for Advanced Therapies (CAT) quarterly highlights and approved ATMPs’. Data on safety concerns and aRMMs was collected from the ‘EPAR - Public assessment report’ related to the initial MA procedure. Safety concerns were described using the Medical Dictionary for Regulatory Activities (MedDRA) 26.1 and categorised into the most applicable High Level Term and corresponding System Organ Class (broad term) in order to assimilate safety concerns among ATMPs. For safety concerns that could not be classified using MedDRA, we used our created categories. Data on the type of aRMMs was collected as categorised within the public assessment report. We used descriptive statistics to analyse our data.

Results: A total of 25 ATMPs were authorised in the EU until 31 December 2023. Among the ATMPs, 338 safety concerns were identified. We observed that safety concerns within the broad terms ‘Long term effects’ (n=23, 93%), ‘Injury, poisoning and procedural complication’ (n=22, 88%), and ‘Special population’ (n=20, 80%) were most common among ATMPs. The majority (n=23, 92%) of ATMPs were authorised with aRMMs. All of these ATMPs (n=23, 100%) had educational materials for healthcare professionals and 18 (72%) also had educational materials for patients in place. Additionally, a controlled distribution programme was introduced for 13 (52%) ATMPs and controlled access programme for three (12%). aRMMs were most commonly required for ATMPs with safety concerns within the broad terms ‘Injury, poisoning and procedural complication’ (n=19, 76%), ‘General disorders and administration site conditions’ (n=8, 32%), and/or ‘Immune system disorders’ (n=8, 32%).

Conclusions: Most ATMPs were authorised with aRMMs. Whilst educational materials were most prevalent, controlled distribution or controlled access programmes were also commonly implemented. For many ATMPs, aRMMs addressed safety concerns related to ‘Injury, poisoning and procedural complication’.