As a new political mandate kicks off for the EU Institutions, it is imperative that the political spotlight on rare diseases does not fade. The past 20 years have seen a considerable increase of activity at both political and non-political level. Almost all EU Member States developed or are developing Rare Disease Action Plans.
Following the adoption of the Orphan Medicinal Products (OMP) Regulation in 2000, which aimed to address market failures by creating a regulatory and incentive framework to support the development and marketing of OMPs, we witness the number of therapies for rare diseases grow from 8 to over 240. This was accompanied by supporting academic research, establishing European Reference Networks (ERNs), and launching ERDERA, to highlight a few examples. While more work remains to be done, Europe has a robust legacy to build on.
The EU now finds itself at an important junction, with the General Pharmaceutical Legislation (GPL) still under negotiation, and the political priorities for the new mandate being established, including the next Multiannual Financial Framework (MFF). Under the new Commission, we will see the development of a new Biotech Act, review of the Clinical Trial Regulation, and a growing focus on European competitiveness.
These are welcome and needed discussions and must build on existing successes. EUCOPE outlined a number of recommendations for the new mandate in an open letter.
Fundamentally, to ensure that the rare disease ecosystem delivers, legislations, policy and non-legislative initiatives need to work in a holistic manner. In the context of the GPL, the European Parliament called for the creation of a Rare Disease Framework, which EUCOPE whole-heartedly echoes. To achieve the desired impact, legislation cannot exist in silos but must be mutually reinforcing.
Developing therapies for rare diseases is intrinsically complex. There are a host of challenges stemming from limited scientific knowledge and understanding of the conditions. Progress builds on continued and incremental advances, that at time can represent major leaps forward, only after dedicated and painstaking research. On occasion, advances in rare disease treatment also bring benefits to chronic disease populations as knowledge is applied in novel contexts. To continue driving this, a competitive and viable R&D ecosystem must be nurtured. The EU hosts world-leading universities but struggles to support translational research and ensure that great ideas
move from lab to patients.
The rare disease community, including EUCOPE, are not standing idly by. A number of initiatives such as the OD Expert Group, TRANSFORM Alliance, Together4RareDiseases, Rare Disease Moonshot aim to bring novel thinking, solutions and political attention to the rare disease ecosystem. Collaboration is key in driving change. By working together with various stakeholders, including patient organisations, researchers, policymakers, healthcare providers, we can create a more effective and equitable environment.
EUCOPE supports calls for the creation of a comprehensive and forward-looking EU Rare Disease Framework that would facilitate a coherent, collaborative and comprehensive approach to tackling rare diseases across the EU. The rare disease ecosystem must remain in the political spotlight. A comprehensive plan sends a clear message while ensuring a holistic and mutually reinforcing policy programme. EUCOPE remains dedicated working with all stakeholders to build on the existing successes to establish a competitive rare disease ecosystem that promotes innovation, incentivises research, and ensures swift market access for new therapies.