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Science to policy

Translating scientific evidence and shaping policy: ERDERA as a rare disease policy actor

ERDERA is firmly committed to advancing access to knowledge and ensuring that valuable research findings are effectively translated into policy and practice across health and research systems. Through the careful synthesis of robust scientific evidence and its alignment with key European and international policy frameworks, ERDERA develops policy briefs that serve as a strategic resource for decision-makers.

Beyond knowledge translation, ERDERA plays an active and recognised role in policy dialogue, drawing on its long-standing presence, multidisciplinary expertise, and established position within the wider health and research ecosystem. Its policy briefs therefore not only inform discussions but also contribute to shaping policy priorities at local, national, European, and global levels.

In parallel, these briefs highlight the relevance and broader societal impact of ERDERA’s activities, showcasing the value of the research it supports and funds across Europe. They demonstrate how this work contributes to advancing knowledge, strengthening health systems, and ultimately improving outcomes for patients, families, and society as a whole.

Why is this relevant?

Sharing research findings and insights from both clinical practice and patient experience ensures that vital knowledge reaches those who can act on it. Robust evidence enables decision‑makers to understand real needs, set informed priorities, and allocate resources effectively. Ultimately, this leads to better diagnosis, treatment, and care for people living with rare conditions.

ERDERA's impact on policy

ERDERA elevates key research findings to policymakers, clarifies priority needs, and supports the development of coherent, evidence‑based solutions. This contribution plays a critical role in strengthening policy frameworks and enhancing outcomes for patients and health systems.

Shaping policy with evidence-based research

As a co-funded European partnership bringing together 180 public and private partners to advance research and innovation, ERDERA plays an important role at the science–policy interface in rare diseases, health and wider European competitiveness. Policy briefs help explain the relevance of research findings and knowledge to ongoing policy debates, and support ERDERA in articulating its position on proposed policy or regulatory changes, particularly at European level.

Alongside these explanatory briefs, ERDERA’s governance structure also supports the development of specific policy recommendations. In November 2025, following endorsement by the ERDERA Governing Board, the ERDERA Policy Think Tank was established as a neutral platform for evidence-based policy discussion and analysis, aligned with the European legislative calendar to help anticipate policy impacts.

The Think Tank aims to support ERDERA’s objectives by addressing policy issues on which the knowledge and experience of the ERDERA community — including researchers, clinicians, patients and policymakers — together with external stakeholders, can make a meaningful contribution. It is co-led by two chairs, one from the Governing Board and one from the Multistakeholder Advisory Board, and its work is carried out by a Steering Group of nine ERDERA members representing a range of internal stakeholder groups. Its activities are guided by a roadmap covering key policy areas such as data and clinical trials. The Think Tank is expected to hold two workshops each year, bringing together members and external experts to develop recommendations that translate research, knowledge and existing policy tools into concrete action. These recommendations will be submitted to the ERDERA Governing Board for endorsement before being shared with policymakers and other external stakeholders.

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ERDERA Policy Briefs

European Biotech Act 062026

ERDERA Policy Brief: European Biotech Act

The European Rare Diseases Research Alliance (ERDERA), together with the European Reference Networks (ERNs), provides the missing operational layer required to implement the European Biotech Act across the full innovation pathway —from discovery to patient access.
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Other Relevant Policy Publications

BMC Medical Ethics

Ethics and regulatory guidance to address the challenges encountered in research on rare diseases in a multinational dimension: the experience of a European funded programme

This BMC Medical Ethics article examines ethics and regulatory guidance across EJP RD-funded rare disease research projects.
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Evaluating public and patient involvement in interventional research–A newly developed checklist (EPPIIC)

Public and patient involvement (PPI) has been identified as an increasingly desired and, often, required component of trial methodology–leading to higher quality, more accessible and relevant clinical research.
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Strategies for diversity and inclusion in public involvement

Supplement to the briefing notes for researchers
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Involving and engaging ‘seldom listened to’ community voices in clinical translational health research: a social innovation approach

“We are not hard to reach, but we may find it hard to trust”
Publicaciones (1)

EU rare disease action plan- European Added Value Assessment

This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
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The involvement of underrepresented countries in the European rare diseases research alliance: Morocco’s assets

Using Morocco as a case study, this paper shows how underrepresented countries contribute unique genetic insights to rare disease research and why their integration into initiatives like ERDREA is essential for both scientific progress and equity.

Recent updates

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Biotech Act II: European Commission opens Call for Evidence on industrial biotechnology and biomanufacturing

The European Commission has opened a Call for Evidence on the upcoming Biotech Act II — with feedback open until 10 June 2026, inviting stakeholders to submit input that will help shape the initiative before a legislative proposal is prepared.
ERDERA First Policy Think Tank meeting

ERDERA Policy Think Tank meets in Paris to advance data and registry policy for rare diseases

Bringing together policy makers, researchers and data experts to align on how registry data can better support rare disease research and evidence‑based policy across Europe.
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Towards animal-free testing: virtual control groups gain regulatory acceptance

For rare diseases, where research often relies on limited animal models and small development teams, the regulatory acceptance of virtual control groups could help streamline early non‑clinical studies while maintaining a strong focus on patient safety.
Insights frInsights from the ERDERA consultancy service surveyom the ERDERA consultancy service survey

Insights from the ERDERA consultancy service survey

Preliminary results point interest across the rare disease research community, while highlighting underrepresentation of some countries and stakeholders

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