The European Union has embarked on crucial trilogue negotiations aimed at overhauling its pharmaceutical legislation, a move that could significantly impact the development and availability of treatments for rare diseases across member states. This initial meeting, held on June 17, 2025, marks the commencement of intensive discussions between the European Parliament, the Council of the EU, and the European Commission.
Progress and Challenges in Legislative Reforms
Both the European Parliament and the Council have presented their positions on the pharmaceutical package, which seeks to streamline the approval and regulation of medicines. While the Parliament’s amendments have been positively received for introducing meaningful improvements, the Council has proposed changes that may undermine certain provisions essential for supporting rare disease treatments. Key issues include the removal of explicit references to orphan medicinal products and modifications to market exclusivity terms, which are vital incentives for pharmaceutical innovation.
EURORDIS Advocates for Strengthened Patient Representation
EURORDIS–Rare Diseases Europe has been a vocal proponent of the legislative reforms, emphasizing the need for precise and ambitious measures to address the unmet medical needs of the 30 million Europeans living with rare conditions. The organization highlights that a significant portion of rare diseases still lack dedicated therapies, with 94% of these conditions without specific treatments. EURORDIS urges policymakers to maintain robust support mechanisms like the PRIME scheme, which has been instrumental in advancing treatments for rare diseases.
– Reinstate explicit mention of orphan medicinal products in regulatory frameworks.
– Preserve the 10-year baseline for market exclusivity to ensure continued innovation.
– Simplify criteria for high unmet medical need to avoid legal ambiguities.
– Strengthen patient representation in regulatory committees to enhance decision-making.
The ongoing negotiations present a pivotal opportunity for the EU to refine its approach to medicine regulation, particularly for rare diseases. Ensuring that the final legislation incorporates clear support for orphan drugs and maintains incentives for pharmaceutical companies is crucial. Additionally, meaningful patient involvement must be safeguarded to ensure that the voices of those most affected guide the regulatory processes. As the trilogue discussions progress, the outcomes will play a decisive role in shaping the future landscape of healthcare access and innovation within the European Union.
Effective legislation should balance the need for regulatory support and market incentives while fostering a collaborative environment that includes patient perspectives. By addressing the current challenges head-on and reinforcing the commitments to rare disease communities, the EU can set a benchmark for inclusive and responsive healthcare policies. Stakeholders must remain engaged and proactive to ensure that the reforms lead to tangible improvements in the lives of those battling rare diseases, ultimately driving forward a more equitable and innovative pharmaceutical sector.
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