There is broad support from the innovative pharmaceutical industry to establish an EU Rare Disease Action Plan (EU RDAP). While the political agenda in the EU continues to shift between competing priorities, momentum behind this long-standing call is growing. Ensuring rare diseases remain high on the political agenda is crucial to drive innovation and facilitate patient access. This is even more relevant in an increasingly complex global environment, with major geopolitical uncertainty and a pharmaceutical industry facing significant changes.
EUCOPE supports and echoes the urgent calls for an EU RDAP. Many of the challenges faced by the rare disease community require joint or coordinated action, particularly to reduce inequalities between Member States or regions within them. Challenges span the entire care pathway, including screening, research, diagnosis, and treatment. An EU RDAP should serve as a strategic policy framework, identifying synergies and ensuring consistency across mutually reinforcing policy domains. Especially in the rare disease space, multi-stakeholder approaches are essential to achieve meaningful progress.
We cannot wait, as developers and Member States are making decisions today that will shape the rare disease landscape for years to come
EUCOPE is proud to work alongside Rare Diseases International (RDI) and EURORDIS to support the development of an EU Rare Disease Action Plan blueprint. In parallel, by supporting and partnering with initiatives such as ERDERA and the HLM-Rare, industry can contribute its extensive experience in research and development. Sustained EU action is not only critical for people living with rare diseases and their families, but also has the potential to strengthen the competitiveness of the EU pharmaceutical ecosystem and, by extension, the wider European economy.
These efforts must be complemented by measures that deliver tangible change and solutions in the short term. The European Commission has proposed a number of new legislative initiatives addressing different elements of the healthcare and innovation ecosystem. This includes the Biotech Act, which holds the potential to revitalise the research environment and stimulate clinical trials. Yet, the impact of such initiatives will take several years to materialise.
Many of the challenges faced by the rare disease community require joint or coordinated action, particularly to reduce inequalities between Member States or regions within them
EUCOPE urges EU policymakers to move forward with the Rare Disease Action Plan, while leveraging and properly implementing the tools and legislation we have today to reinforce rare disease innovation and access. Since 2021, EUCOPE has contributed, with other partners, to the European Expert Group on Orphan Drug Incentives, a multi-stakeholder forum focused on bringing forward practical solutions. In 2025, the Group published a report outlining an “access toolbox”, acknowledging that the current system is far from optimal, but that opportunities and best practices already exist. Concrete examples include the proper implementation of the Transparency Directive or the wider adoption of early access frameworks, both of which can facilitate access in the short term. The report outlines 15 calls to action across three broad domains.
We cannot wait, as developers and Member States are making decisions today that will shape the rare disease landscape for years to come. Especially where therapies exist, concerted efforts should be made to overcome existing access barriers. The EU should work across two parallel and mutually reinforcing pillars: building a comprehensive Rare Disease Action Plan which looks across the full care spectrum and product lifecycle, and accelerating access and innovation today, by leveraging the tools and resources already at its disposal.
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