Strengthening the European translational research ecosystem for advanced therapy medicinal products (ATMPs) for rare diseases
There are over 7 000 rare diseases resulting in 30 million patients1 in Europe with a rare disease. Globally more than 300 million patients2 are affected. In Europe, less than 10 % of rare disease patients receive treatment and only 1 % are managed using an approved treatment. ATMPs such as gene and cell therapies and other related innovative therapeutic modalities, are very promising to treat patients with rare diseases, especially ultra-rare diseases. However, ATMPs rely on complex technologies where the development process is hampered by a lack of standardisation, scalability and reproducibility.
The overall aim of this topic is to optimise and streamline the future development of ATMPs and other related innovative therapeutic modalities for rare diseases by strengthening the ecosystem that facilitates the transition of early pre-clinical proof-of-concept research to clinical development. This topic focuses on the scientific, technological and regulatory barriers that are lim
- Benefits for patients both with rare and ultra‑rare diseases and who may gain from effective and safe advanced therapy medicinal products (ATMPs) and other related innovative therapeutic modalities.
- A better and more cost-effective development of ATMPs and other related innovative therapeutic modalities due to improved scientific and technological processes. This is applicable especially to those ATMPs intended both for the treatment of rare diseases and those that are currently underserved by current therapies (the latter often being of genetic origin).
- Europe to become more attractive for developing ATMPs due to the availability of sustained, interconnected networks of technological and scientific centres of excellence. Although their current focus is on translational research, linkages to clinical networks, including the European reference networks (ERNs) on rare diseases will enhance their activities. The same is true for synergies to be developed with the European Join