On Friday, October 20, 2017, the European Commission (EC) and the European Medicines Agency (EMA) published a joint action plan to promote advanced therapy medicinal products (ATMPs), the “European Commission-DG Health and Food Safety and European Medicines Agency Action Plan on ATMPs” (Action Plan).
ATMPs (gene therapy, somatic cell therapy and tissue-engineered products) are regulated as medicinal products under Regulation 1394/2007.1 This regulation, however, does not encourage the development of ATMPs as much as had been hoped,2 for many reasons.
Those reasons have been identified and discussed by the EC and the various stakeholders.3 Good Manufacturing Practice (GMP) principles and rules (e.g., practices regarding batch consistency, comparability, etc.) being better adapted to the particularities of ATMPs seems to be a—if not the—key issue, especially for autologous products. Another issue is that EU member states transpose the European rules on tissues and cells differently in their respective national laws.4 An additional difficulty is the application of European rules on genetically modified organisms (GMOs); many ATMPs consist of or contain a GMO, and GMO products as well as operations on or with GMOs or GMO products are subject to very stringent rules, including a prior approval regime.
The Action Plan has 19 items that cover areas from development to pricing reimbursement.
(See the table that begins on page 2.) Some items have already been completed; others are ongoing or still have to be implemented. Certain issues will only start being tackled in a couple of years — the items with 2019 deadlines include the GCP (guideline on comparability) and health technology assessment (HTA) points.
Interestingly, the Action Plan indicates that the EC liaises with the member states to resolve the issue of the “hospital exemption.” The hospital exemption is a derogation from the requirement to obtain a marketing authorization before placing an ATMP on the market. It was meant to be restricted to ATMPs developed by a hospital for a few of its patients. In practice, however, some member states have implemented the exemption in such a broad way that abuses occurred or could occur in a way that negatively impacts the production of ATMPs on an industrial scale. Hopefully, the EC will develop a guideline that explains the limitations to the hospital exemption, leveraging the case law of the Court of Justice of the European Union on the other exceptions to prior marketing authorization.
As more and more companies are investing in ATMPs, easing and improving the regulatory environment for ATMPs is crucial not only for Europe’s competitiveness but also for the industry. Each Action Plan item is an opportunity for trade associations and companies to have their specific interests taken into account, an opportunity that should not be missed.