Actions address specific challenges identified by stakeholders
The European Commission’s Directorate-General for Health and Food Safety (DG SANTE) and the European Medicines Agency (EMA) have published today a joint action plan to foster the development of advanced therapy medicinal products (ATMPs). The main aim is to streamline procedures and better address the specific requirements of ATMP developers.
ATMPs are medicines for human use that are based on genes 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.
ATMPs can be classified into four main groups: gene therapy medicinal products, somatic cell therapy medicinal products, tissue engineered medicinal products and combined ATMPs. EMA has received 18 marketing authorisation applications since the ATMP regulation came into force in 2009. Nine products have been approved.
The Agency’s Committee for Advanced Therapies (CAT) plays a central role in the scientific assessment of ATMPs, as it provides the expertise needed to evaluate these medicines. Other initiatives include European Commission research programmes, the innovation offices in the national competent authorities and EMA’s PRIME scheme.
At international level, a regular forum for dialogue has been set up with the United States Food and Drug Administration (FDA), Health Canada and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) to share experience on ATMPs. EMA and the CAT also contribute to the cell therapy group and gene therapy groupof the International Pharmaceutical Regulators’ Forum (IPRF).
DG SANTE and EMA, in collaboration with the Member States’ competent authorities, are working on some initiatives to support the development and authorisation of high quality, safe and effective ATMPs. The plan published today contains 19 actions in different key areas. Some of the actions are already in place, others are new. Actions were also informed by the ideas collected during a multi-stakeholder workshop hosted by EMA on 27 May 2016. The workshop aimed to explore solutions to identified challenges in the development of ATMPs. Topics discussed ranged from the need for early interaction and guidance from regulators, to more transparency and information sharing, greater harmonisation between Member States in various aspects of ATMP regulation and measures to tackle inequalities in patient access to ATMP treatments.
Examples of upcoming actions in the plan include:
- European Commission guideline on good manufacturing practice for ATMPs, to reduce the administrative burden and adapt the manufacturing requirements to the specific characteristics of ATMPs;
- initiation of dialogue with national competent authorities to address the interplay between the legislation on genetically modified organisms (GMO) and on medicines,to reduce discrepancies across the European Union (EU) regarding the application of GMO rules;
- new EMA scientific guidelines on ATMPs, including investigational ATMPs, to clarify regulatory expectations;
- continuous awareness and training sessions organised by EMA for the EU network on ATMP-related topics.
DG SANTE and EMA will continue monitoring the field and propose further initiatives as appropriate.