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Turning rare disease data into actionable knowledge
Orphanet-AISBL is a non-profit organization transforming the world’s most trusted rare disease data into reliable, ready-to-use knowledge to address the needs of all stakeholders active in rare diseases, including pharmaceutical companies, researchers, policymakers, consultancies, CROs, and patient communities.
Built on over 25 years of Orphanet expertise and more than five million expert-curated combined data, we connect and empower all stakeholders to make evidence-based decisions and advocacy, accelerate innovation, and drive measurable impact across the rare disease ecosystem.
Commitment to providing reliable knowledge
The mission of Orphanet-AISBL is aligned with overarching public-interest objectives: strengthening the evidence base available to actors in the rare disease sector, improving the coherence of scientific and policy activities, and ultimately contributing to better outcomes for persons living with rare diseases.
By ensuring the provision of reliable, expert-curated knowledge, Orphanet-AISBL supports the development of informed and transparent decision-making processes at local, national, and international levels.
Orphanet-AISBL remains committed to maintaining its role as a neutral, scientifically rigorous provider of knowledge in service of all players in the rare disease ecosystem.
A mandate framed by scientific rigour
Orphanet-AISBL operates within an institutional framework that prioritises neutrality, transparency, and scientific robustness. Its core function is to expand the impact of the rich Orphanet knowledge base to better serve and support its operational use, e.g., transform data into actionable knowledge.
This transformation process requires Orphanet and its AISBL to follow an established sequence: from raw data, to curated data, to actionable knowledge and generation of evidence for decision-making. Each stage is governed by strict quality requirements designed to guarantee reliability, traceability, and methodological coherence.
By ensuring consistency and standardisation, Orphanet-AISBL contributes to strengthening national and international rare-disease initiatives, supporting evidence-based policymaking, enabling regulatory assessments, and informing the development of research and care strategies.