EMA releases draft revised guidance on safety and efficacy follow-up and risk management for ATMPs

The European Medicines Agency (EMA) has released a draft revised guideline on the safety and efficacy follow-up and risk management for advanced therapy medicinal products (ATMPs). The revised guidance is part of the joint action plan published by the European Commission and EMA in October 2017 which aims to streamline procedures and better address the specific requirements of ATMP developers.

The guideline was adopted by the Committee for Advanced Therapies (CAT), the Pharmacovigilance Risk Assessment Committee (PRAC) and the Committee for Medicinal Products for Human Use (CHMP) and is the first revision of the original ATMP guideline on safety and efficacy follow-up and risk management. The guideline has been revised to take into consideration the experience gained with the authorisation of these products, and to illustrate their risks and their risk minimisation measures. Furthermore, guidance on methodology in order to design post-authorisation safety and efficacy follow-up studies is provided.

The Pharmacovigilance System Master File (PSMF) and the Risk Management Plan (RMP) are two documents from the Marketing Authorisation Holder (MAH) that are directly impacted by this revised guideline. Financial penalties and regulatory measures are mentioned in the guidance, highlighting the consequences of non-compliance with the pharmacovigilance and risk minimisation activities agreed in the RMP.

The revised guidance supports ATMP developers by clarifying the requirements of the post-authorisation safety and efficacy studies acknowledged at the time of marketing authorisation. The guidance helps to encourage developers of ATMPs to organise timely interactions with the EMA to secure an efficient development process.

To view the guideline on safety and efficacy follow-up and risk management of ATMPs, please click here.