Updated procedural advice clarifies regulatory process for advanced therapy medicinal products
The European Medicines Agency (EMA) has updated its procedural advice on the evaluation of advanced therapy medicinal products (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 authorisations, to help developers of these medicines – often small and medium-sized enterprises (SMEs) or academic spin-offs – navigate the regulatory process in the EU.
It is an important step forward in the support of developers as it addresses their need for more clarity in the evaluation procedures for these innovative medicines.
ATMPs are medicines for human use that are based on genes, tissues or cells. These therapies offer ground-breaking new opportunities for the treatment of disease and injury. They are particularly important for severe, untreatable or chronic diseases for which conventional approaches have proven to be inadequate.
The updated procedure reinforces timely and effective interactions between the applicants, EMA and its committees. ATMPs are assessed by at least three of 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-authorisation 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. It is part of the joint action plan published by the European Commission and EMA in October 2017 to streamline procedures and better address the specific requirements of ATMP developers.