Advanced Medicinal Therapies - Step Up The Healthcare Revolution Now

On February 19, EuropaBio and the Alliance for Regenerative Medicines (ARM) organised a debate on the potential of advanced therapies for currently incurable diseases. The event, co-hosted by MEPs Claudia Gamon (Renew Europe) and Tomislav Sokol (EPP), saw the contribution of the European Medicines Agency (EMA), Representatives of DG SANTE, Orchard Therapeutics and the Dutch Haemophilia Patient Organisation, with concrete examples of the value of these treatments for patients, families and society.

Read the conclusion of the meeting below:

1. Advanced therapies offer new therapeutic options including for currently untreatable diseases and, hence, hold great potential to improve the lives of patients and their families. There are over 7000 rare diseases and advanced therapies will first be developed in rare diseases, opening the possibility to treat more common diseases in the future.

2. Many patients are already benefitting from advanced therapies, and in some cases the clinical results are transformative. One-time administration can offer the potential for a permanent correction of a disease.

3. 2019 was another year of significant growth in the development of advanced therapies. We enter 2020 prepared for continued expansion. EMA are expecting to receive up to 20 new applications for advanced therapies until the end of 2021.

4. The EU has a robust, science-based regulatory framework for the approval of advanced therapies which protects the safety of patients.

5. Making these therapies available to patients involves complex manufacturing, logistics and administration. It requires the qualification of treatment centres as well as adequate reimbursement and incentive systems.

6. Harmonisation of rules related to manufacturing and conduct of clinical trials between the EU Member States is necessary to accelerate the approval processes of advanced therapies.

7. To fully maximise the potential of advanced therapies, the EU healthcare systems will need to adapt to new ways of generating evidence, to new payment models and to new ways of delivering care. Qualified centres of excellence may not be available in every country or region, so patients may have to travel to get their treatment.

8. Europe has been leading the field in developing advanced therapies, with 14 products being granted marketing authorisation by the EMA compared to 9 by the US FDA. Europe’s competitiveness needs to be maintained considering the rapid developments in North America and Asia.

9. The European Commission and EMA will continue listening to stakeholders and are committed to support the development of advanced therapies.

10. A dynamic and less fragmented regulatory environment, adequate reimbursement systems recognising the specificities of the different technologies in their assessment process and the right incentives for continued innovation are essential for boosting the development of advanced therapies in the EU.