The European Medicines Agency (EMA) has released a draft revised guideline on the safety and efficacy follow-up and risk management of advanced therapy medicinal products (ATMPs), which are medicines based on genes or cells, for a three-month public consultation. In another development, the EMA updated procedural advice to clarify the regulatory process for ATMPs.

The revision is part of a joint action plan published by the European Commission and the EMA in October 2017 to streamline procedures and better address the specific requirements of ATMP developers. ATMPs offer new opportunities for treating disease and injury based on innovative approaches and technologies, but because of this novelty, ATMPs may be associated with risks of a different nature to those generally encountered with more conventional medicines, notes the EMA.  This is the first revision of the ATMP guideline.

The guideline now takes into consideration the experience gained with the authorization of these medicines as well as the experience with scientific advice and protocol assistance. It also provides advice on early detection of risks during development and provides a framework for the effective mitigation of their consequences for patients. In addition, the revision gives methodological advice on the design of appropriate post-authorization studies to follow up on the safety and efficacy of these medicines.

As another part of the joint action plan, the EMA has updated its procedural advice on the evaluation of ATMPs. The update aims to streamline some procedural aspects, strengthen collaboration between EMA’s scientific committees, and address specific needs of ATMP developers in the evaluation procedure for initial marketing authorizations to help developers of these medicines—often small and medium-sized enterprises (SMEs) or academic spin-offs—navigate the regulatory process.

ATMPs are assessed by at least three of the EMA’s scientific committees: the Committee for Advanced Therapies (CAT), the Committee for Medicinal Products for Human Use (CHMP), and the Pharmacovigilance Risk Assessment Committee (PRAC). The processes for adopting the list of questions and list of outstanding issues by the committees have been streamlined. The update also clarifies in which situations oral explanations might be needed. While it concentrates on the initial evaluation of new ATMPs, its principles also apply to post-authorization procedures.

In addition, the revised procedure gives developers more time to respond to questions raised by the committees by allowing longer clock-stops (the time period in which the company prepares answers to questions from EMA’s committees).

The update of the procedural advice is the result of a joint effort between the CAT, CHMP and PRAC, in collaboration with the European Commission, and was adopted by all three committees at their respective December 2017 meetings.

Source: European Medicines Agency (guidance statement), European Medicines Agency (revised guideline), European Medicines Agency (procedural advice)