This category includes universities and other institutions whose primary function is to carry out rare disease research.
- Research and development (R&D)
- Research to identify unmet needs and opportunities in the system, understanding issues
- Methodology development, e.g. innovative trial designs suitable for evaluation of rare disease therapies
- Coordinating and delivering clinical trials
- Provide expert opinion to drive strategy of research landscape.
- Policy influencing
- Education, training and mentoring
- Patient and public involvement and engagement (PPIE) and outreach
- Evidence generation to support regulatory approvals and market access
- Building alliances with other organisational categories
