twitter facebook email
"Working together we can secure the best use of scarce expertise and resources, maximising the health gain for all those with rare conditions" Alastair Kent OBE, Chair of RDUK

Latest Partners

Living with Lowes

HLRCC Family Alliance

LAM Action

Living Beyond a Diagnosis

Cystinosis Foundation UK

MLD Foundation

CDKL5 UK

Ben's Friends

Kirsty's Cause

Bone Cancer Research Trust

PVNH Support & Awareness

PID UK

You are here: Home / UK Rare Disease Strategy

UK Strategy for Rare Diseases

In June 2009, the UK adopted the Council of the European Union’s Recommendation on an action in the field of rare diseases. This document recommends that member states ‘Establish and implement plans or strategies for rare diseases’ and that these should be adopted ‘as soon as possible, preferably by the end of 2013 at the latest’.

Since this Recommendation was adopted by the UK Government in June 2009, RDUK has been working to ensure that the UK develops a strategy that is comprehensive and effective, and accurately reflects the needs of the rare disease community.

As part of this work, on Rare Disease Day 2011 we launched our landmark report ‘Improving Lives, Optimising Resources: A Vision for the UK Rare Disease Strategy’ which outlines the current situation for people living with, or working in the field of, rare diseases in the UK and the actions that are needed to improve on this.

Please follow the links below for more information on a UK Strategy for Rare Diseases:

In November 2013, RDUK was pleased to welcome the UK Strategy for Rare Diseases. 

RDUK (in conjunction with the Department of Health in England and Great Ormond Street Hospital, GOSH) held an event to mark the publication of the UK Strategy. The event was an excellent opportunity to find out more on the UK Strategy and network with those interested in the field of rare disease.  

A number of high-level speakers presented, including:

  • Earl Howe, Minister in charge of rare diseases.
  • Dr Mike Berwick, Deputy Director of NHS England.
  • Alastair Kent OBE, Chair of Rare Disease UK
  • Rosalind Smyth, Professor of Child Health and Director of the Institute of Child Health
  • Baroness Tessa Blackstone, Chair of Great Ormond Street Hospital
  • David Goldblatt, Director of Clinical Research & Development UCL  

  

Earl Howe announced key themes in the UK Strategy, stating that a priority of the Strategy is to empower patients; ensuring that patients are listened to, informed and consulted every step of the way. The Minister confirmed the Government was committed to working closely with patients groups and others to improve services for those affected by rare conditions.    

Josh Tucker / Rare Disease UK

Dr Mike Berwick, Deputy Director of NHS England, presented on the action NHS England would take in order to achieve the 51 commitments in the UK Strategy. He noted that NHS would work with a number of partners, including Rare Disease UK, to hold scoping and engagement events throughout the next three months, where all stakeholders (including patient groups, industry, clinicians and commissioners) will be invited to submit proposals for local changes.   

Patients, clinicians and members of the public will then be asked for their views on the top priorities, before NHS England sets out its plan. The plan is due to be released in July 2014.


From left to right: Baroness Tessa Blackstone, Dr Mike Berwick, Alastair Kent,
David Goldblatt, Rosalind Smyth and Earl Howe

Alastair Kent OBE, Chair of Rare Disease UK, welcomed the UK Strategy for Rare Diseases, stating that patients can now have a clear expectation of what the NHS aspires to provide for them, wherever they live in the UK. He stressed that that the UK Strategy is just the beginning of the process and we must now focus on the implementation.  

Before the presentations Earl Howe had the opportunity to meet with a number of experts based at UCL Institute of Child Health (ICH), including Professor Bobby Gaspar, a pioneer in gene therapy for severe combined immunodeficiencies (SCID). Professor Gaspar introduced the Minister to Nina, a patient on a new clinical trial for SCID led by GOSH.  


Earl Howe with Nina and her parents, Graeme and Aga

The Minister then met with Professor Philip Beales, an expert in medical and molecular genetics, Bardet-Biedl syndrome and related rare conditions, who then accompanied Lord Howe on a tour of the research laboratories at the ICH.


Professor Philip Beales with Earl Howe 

Key features of the Strategy include:

  • a clear personal care plan for every patient that brings together health and care services, with more support for them and their families
  • help for specialised clinical centres to offer the best care and support
  • better education and training for health and social care professionals to help ensure earlier diagnosis and access to treatment
  • promoting the UK as a world leader in research and development to improve the understanding and treatment of rare diseases

To achieve the UK-wide vision for rare diseases, there are 51 recommendations which all four countries of the UK have committed themselves to.

You can read them here: https://www.gov.uk/government/publications/rare-diseases-strategy

On the UK Strategy, Health Minister Lord Howe said:

'For the first time, we are strengthening the links between research and the treatment and care of patients with rare diseases. This is about putting those patients first, with better diagnosis, treatment and support for them and their families.'