A Vision for an Evidence-Based Rare-Disease Ecosystem
Our vision is to bridge the knowledge gap and connect stakeholders, fostering a more inclusive, data-driven rare disease ecosystem.
Today, data are dispersed across countries, systems, and institutions. This dispersion slows progress, weakens analyses, and limits the ability of organisations to act with confidence. Our ambition is to shorten this distance, to provide a clear, structured, and dependable route from data to results.
Accelerating Evidence-Based Decision-Making
We envision an environment where decisions in research, clinical care, regulation, and policy are systematically supported by reliable knowledge.
By reducing the time required to move from scattered information to validated insight, we help stakeholders act faster, with greater precision and reduced uncertainty.
Boosting Research and Development Efficiency
A more coherent knowledge base improves the entire R&D continuum.
Our vision is to strengthen this continuum by enabling researchers and developers to access accurate, contextualised information without delay.
This efficiency supports better study design, clearer strategic choices, and the ability to progress from hypothesis to impact with fewer obstacles.
Promoting Collaboration and Transparency Across Sectors
Progress in the rare-disease field depends on information that can be shared, understood, and used across scientific, clinical, industrial, and policy domains.
We aim to foster an environment where collaboration is facilitated by transparent, harmonised knowledge, reducing duplication of effort and reinforcing collective intelligence.
Data as a Shared Resource for Progress
At the core of our vision lies a simple principle: data should serve the collective advancement of the rare-disease community. By treating data as a shared resource, rather than a fragmented collection of isolated information, we support faster innovation and the development of outcomes that directly benefit rare-disease patients.
Our long-term vision is a landscape where actionable knowledge circulates efficiently, empowering every stakeholder to contribute meaningfully to research, care, and policy, ultimately improving the lives of those affected by rare diseases.