Spotlight on Rare Diseases

Stats

1. Rare disease definition, 80%, 3.5 million: https://www.raredisease.org.uk/what-is-a-rare-disease/
2. >90%: Pugath Consilium Benchmarking Success report (p.37)

How do medical research charities make a difference for rare diseases?

1. AMRC charities are involved in different activities to make a difference for rare diseases. These key activities were extracted from AMRC's grants database.
2. £142 million, 640 projects, 67 (27) charities, 29%: Awards from current AMRC members active in 2018. Inclusion criteria: award was either funded by a rare disease-focused charity or contained the keyword 'rare' in the title, abstract and/or lay summary. Irrelevant grants (not concerning rare diseases) were excluded.

To learn more about the projects featured in this report, follow these links:

1. Ensuring early diagnosis
2. Establishing a patient registry
3. Building knowledge with innovative tools
4. Engaging at all drug development stages
5. Improving care in the NHS
6. Attracting and supporting leaders
7. Joining forces to drive research forward and http://www.smareachuk.org/
8. Creating a rare disease centre