Rare Disease Action Plan
Policy is evolving
Systems are adapting
Momentum is building
This page provides a brief overview of where things stand today.
United Kingdom
A shared framework
Practical national action
The UK Rare Diseases Framework continues through to 2027, supported by action plans across England, Scotland, Wales and Northern Ireland.
Priorities focus on:
- Helping people receive a diagnosis faster
- Increasing awareness of rare diseases among healthcare professionals
- Improving coordination of care
- Expanding access to specialist services and treatments
Key resources
United States
Accelerating research
Advancing treatments
The US approach is shaped by strong federal research programmes and regulatory initiatives designed to support rare disease innovation.
Current developments include:
- Continued expansion of national rare disease action plans, task forces, and research networks (for example the Action plan for rare neurodegenerative diseases)
- Legislative programmes aimed at accelerating development and access to rare disease therapies (for example the RARE Act and ORPHAN Cures Act)
Key resources
European Union
Collaboration across borders
Stronger data foundations
European activities emphasise cross-border specialist collaboration and modernised data infrastructure.
Key resources
Why This Matters
Across the UK, US and EU, the direction of travel is similar:
1Earlier identification of rare diseases
2Better informed clinicians
3Stronger specialist networks
4More structured and responsible use of health data
5Fairer and faster access to innovation and treatments
At My Rare Journey, we believe lived experience and real-world insight are essential to ensuring these ambitions translate into meaningful change for people living with rare diseases.
Behind every framework, strategy or regulation is a person waiting for answers.
Policy and regulation continue to evolve. We will review and refresh this overview quarterly to reflect meaningful developments.
Last updated: March 2026