Rare Disease Watch provides clear, trustworthy information to help families, clinicians, researchers, and advocates make sense of the fast evolving rare disease landscape. This page brings together essential guides, explainers, tools, and reference materials created to support understanding, decision making, and awareness.
How to Read Research
Step-by-step guides designed for patients, families, and advocates who want to better understand scientific studies, trial results, and medical literature.
Learn how to read research →
Clinical Trials Guide
Resources explaining how clinical trials work, how to search trial registries, key questions to ask, and what participation may involve.
View clinical trial resources →
Regulatory Pathways
Clear summaries of EMA, FDA, MHRA, and NICE processes including orphan designations, accelerated approvals, conditional licences, and market access considerations.
Explore regulatory pathways →
Treatment Access Information
Guides covering named-patient programmes, compassionate access routes, and country-specific approaches to accessing rare disease treatments.
Read access guidance →
Rare Disease Statistics
Key facts and figures including prevalence estimates, number of recognised conditions, and global patterns.
View statistics →
Pipeline and Approval Trends
Easy-to-understand data on new drug approvals, emerging therapies, and rare disease research momentum.
See trends →
Charts and Visual Summaries
Clear data visualisations that bring complex information to life.
Browse data visuals →
Funding and Grant Opportunities
A curated list of financial resources for families, researchers, and rare disease organisations.
Explore funding resources →
Policy and Global Frameworks
Summaries of national and international rare disease policies and frameworks, along with links to authoritative documents.
Read policy guides →
Understanding Clinical Trials
Download
How Rare Disease Drugs Are Approved
Download
Questions to Ask Your Clinical Team
Download
A Short Guide to Genetic Testing
Download
Everything in this resource hub is designed to be:
Clear and accessible
Scientifically accurate
Updated regularly
Useful to patients, clinicians, researchers, and advocates
Free to access
