Action plan released to streamline the development of ATMPs

The European Commission’s Directorate-General for Health and Food Safety (DG SANTE) and the European Medicines Agency (EMA) have released a joint plan that is intended to facilitate the development of advanced therapy medicinal products (ATMPs).

The development of ATMPs for human use has the potential to allow novel treatments for diseases and injuries for which conventional medicine has previously been less successful. By way of a short introduction to ATMPs, they are developed from genes or cells and classified into one of four groups:

  • Gene therapy
  • Tissue engineered
  • Somatic cell therapy
  • Combined ATMPs.

DG SANTE and EMA, along with the Members States’ competent agencies, have been developing initiatives to streamline the development and authorisation of high quality, safe and effective ATMPs. In their joint plan, they have outlined 19 actions in key areas – some are already implemented, but others are new. These actions have been developed to include ideas and initiatives that were discussed at a stakeholder workshop held by the EMA in May 2016, such as:

  • the need for early interaction and guidance from regulators
  • more transparency and information sharing
  • greater harmonisation between member states in various aspects of ATMP regulation
  • measures to tackle inequalities in patient access to ATMP treatments.

The following actions are a few examples of those included in the joint action plan that should be implemented in due course:

  • 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 that 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
  • awareness and training sessions organised by EMA for the EU network on ATMP-related topics.

DG SANTE and EMA also have regular interactions with US, Canadian and Japanese regulators to share their experiences with ATMPs. The International Pharmaceutical Regulators Forum (IPRF) includes a cell therapy group and gene therapy group, to which the EMA is also a contributor.

The EMA and DG SANTE have stated that they will develop and propose further initiatives as and when appropriate.

The full article announcing the joint plan can be found here.