The European Commission (EC) has adopted a Report on the application of the Regulation (EC) No 1394/2007 on Advanced Therapy Medicinal Products (ATMP Regulation). Through this report, the EC reviews the situation of ATMPs in the EU and analyses the impact of the Regulation on advanced therapies. ATMP covers therapies based on genes, cells and tissues.
The ATMP Regulation was designed to protect patients from unsound treatments and to facilitate the free movement of ATMP’s in the EU. Importantly, the Regulation required that marketing authorization be obtained before the marketing of ATMPs, which involved a rigorous scientific assessment by the European Medicine Agency (EMA) Committee for Advanced Therapies (CAT). ATMPs include three types of products: gene therapies, somatic cell therapies and tissue engineered products.
The Regulation gives Member States the power to authorise the use of custom-made ATMPs prepared on non-routine basis in the absence of a marketing authorisation, provided that the product is used for individual patients in a hospital and under the professional responsibility of a medical practitioner. These are known as ‘hospital exemptions.’
Thus far, only four ATMPs have been granted Marketing Authorisation, which include treatments for prostate cancer, cartilage defects and metastic defects of the knee. Almost 70 per cent of sponsors for clinical trials on ATMPs are non-profits or Small and Medium Entreprises (SME’s), which means they are currently the principal stakeholders in the developments of the ATMP Regulation.
Remarks by the EC
The EC noted that the development of ATMPs is hindered by the fact that researchers usually lack appropriate funding and regulatory expertise to successfully navigate through the marketing authorisation procedures. In turn, the uncertainties in the return for investment are a major deterrent to investors.
Since a number of ATMPs continued to be used under the aforementioned derogation ‘hospital exemptions’, the EC worries that a broad definition of ‘hospital exemptions’ is deterring companies from submitting marketing authorisation applications. This is because it is much cheaper to simply provide a product as a ‘hospital exemption’ than to have to provide continuous efficacy data to the EMA. The EC recognizes the need to find a balance between well-regulated, safe and efficient products, and the need to facilitate early access for new treatments in case of unmet medical needs.
The EC concludes by identifying areas that need to be addressed in order to enable more ATMPs to reach patients:
- Clarifying the scope of the ATMP Regulation by fine-tuning the current definitions of ATMPs;
- Clarifying the conditions for the application of the hospital exemption, as well as the role of data obtained during this exemption for future marketing authorisation procedures;
- Revising the requirements for the authorisation of ATMPs to ensure their applicability and proportionality to ATMPs with specific consideration to autologous products;
- Streamlining the marketing authorisation procedures and considering measures to avoid disparities in the classification of ATMPs in the EU;
- Extending the certification procedure and clarifying the link between the certification and the marketing authorisation procedure;
- Creating a more favourable environment for ATMP developers working in an academic or non-for-profit setting, including by promoting early contacts with the authorities through reduced pre-marketing fees and also by considering possible fee incentives to reduce the financial impact of post marketing obligations.
The EC showed clear indications that four ATMPs were insufficient which, in its opinion, indicated a system failure. During last year’s consultation, the feedback received from industry organizations was broadly supportive of the status quo, although a more restrictive approach could help bigger companies reduce competition in personalized medicine. The point of contention, as highlighted in the report, remains the use of ‘hospital exemptions,’ which circumvents the regulatory process. Importantly, while the EC has suggested serious modifications to this Regulation, they do not indicate how these changes could occur. However, the goals highlighted under ‘Conclusions,’ can only be achieved through a revision of this Regulation which makes an attempt to revise the legislation highly probable in the future.