1. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) Implementing the UK Strategy for Rare Diseases Alastair Kent #raredisease

2. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) 2004 DG Sanco Rare Diseases Task Force 2008 Commission Communication on Rare Diseases 2009 Council of Ministers Recommendation  Unanimous  National plans of Strategies by 2013 2009 Genetic Alliance UK establishes Rare Disease UK as a multi-stakeholder Campaign 2013 UK Strategy for Rare Diseases A Bit of History

3. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) 51 Commitments – 31 Health Related, 20 Research  Empowering Patients and Families  Identifying and Preventing  Diagnosis and Early Intervention  Coordination of Care  The Role of Research First time RD patients and families can have clear expectation as to what to expect The UK Strategy

4. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) UK Strategy, Four Nations, Four NHSs Wales – Plan out for consultation, closing date 23/5/14 England – Statement of Intent published on 26/2/14. Includes rare diseases in review of specialised commissioning N. Ireland – Statement of Intent “imminent” Scotland – Consultation paper promised Implementation

5. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) A strategy without implementation is just fluff. But.....

6. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) Multi-stakeholder  Patients  Four Health Departments  Four NHSs  Public Health England  Health Education England  NIHR  Royal Colleges Monitor Implementation and report to Ministers every two years. Deputy Chief Medical Officer appointed National Lead for Rare Diseases in England UK Rare Disease Forum

7. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) All stakeholders have a part to play A serious health issue 6000+ rare diseases 1 in 17 affected 3.5 million across the UK Too important to leave it to “somebody else” A Shared Responsibility

8. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999) Thank You www.geneticalliance.org.uk www.raredisease.org.uk Join the campaign – make progress irresistible

9. A charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299). A company Limited by Guarantee (Number 05772999)