Europe strengthens partnerships for rare disease research

The Together4RD initiative, aimed at strengthening the role of public-private partnerships (PPPs) in rare disease research and developing Europe's technological leadership, has been presented in the European Parliament.

The report proposes removing regulatory and organizational barriers to collaborative projects between clinics and industry on rare diseases, as well as securing stable funding for such programs.

The central idea of ​​the initiative is to transform European Reference Networks (ERNs)* from a "framework" for clinical care into an "engine" for research and innovation, based on real-world data (RWD) and clinical trial readiness.

Together4RD key proposals:

• revise Council Statement of Member States on the ERN (2019), restricting data sharing with industry;
• Recognize PPPs as an official instrument of EU policy and stimulate their development in the field of rare diseases;
• Implement standardized templates for data sharing agreements and standard operating mechanisms at EU level;
• Give the ERN legal personality or create a single "umbrella" authority to simplify contracts and distribute responsibilities;
• Secure funding for the development of trial readiness, registry harmonization, the development of patient-centered outcomes (PROs), and cross-border data sharing platforms, as well as training and resource support for ERN teams.

The authors emphasize that it is through the ERN that it is possible to systematicallyincrease evidence base and accelerate the implementation of innovations while strictly adhering to ethical and legal standards.

*ERN is an association of specialized clinics and research centers in groups of rare nosologies that jointly develop diagnostics, patient routing, registries, and clinical research.

Full text: Together4RD — European Parliament Event Report, 2025 (PDF) (https://drive.google.com/file/d/1EN5xsuVoXFvpmF5F0-L0gukKn09gnmnn/view?usp=sharing)