Westminster Hall Debate on the UK Strategy for Rare Diseases

Today, MPs will meet in Parliament to debate the implementation of the UK Strategy for Rare Diseases in England. This is a unique opportunity for us to raise awareness of the problems facing patients and families affected by rare conditions in accessing a timely diagnosis, coordinated care and appropriate treatment.

Today, MPs will meet in Parliament to debate the implementation of the UK Strategy for Rare Diseases in England. This is a unique opportunity for us to raise awareness of the problems facing patients and families affected by rare conditions in accessing a timely diagnosis, coordinated care and appropriate treatment.

These issues were highlighted in a report recently published by the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions.

This debate couldn't come at a more crucial time. Just last week, NICE announced its decision to implement an upper funding threshold for its Highly Specialised Technology Programme, the main route used to evaluate medicines for rare disease patients. If implemented, the plans will significantly affect rare disease patients and their ability to access life-changing treatment, delivered at a time when we should be expanding access routes rather than further limiting them.

Why are we having a debate in England?

All four nations in the UK agreed to implement the Strategy by 2020. The departments of health in Scotland, Wales and Northern Ireland have all development implementation plans, however the Department of Health in England has not. This is why the Westminster Hall debate is taking place today, to ensure an implementation plan is developed for England and rare conditions are given the resource and funding they deserve and need.

Why England needs an implementation plan

The All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions has published a report looking at the implementation of the UK Strategy for Rare Diseases in England. The report calls on the Department of Health to take action to improve services for patients and families affected by rare conditions

Over 300 patients and other members of the rare disease community, including family members, clinicians, patient representatives and industry, submitted evidence as part of the APPG's inquiry into the implementation of the UK Strategy for Rare Diseases in England. If implemented, the Strategy would provide those living with rare conditions with the highest possible quality of evidence-based care and treatment, regardless of where they live in the UK.

Key finding: the UK Strategy for Rare Diseases is not being effectively implemented in England.

The departments of health in Scotland, Wales and Northern Ireland have all developed plans to improve services for rare disease patients that reflect their respective health service systems. The Department of Health in England has not coordinated an implementation plan for England.

Many patients in England do not have access to appropriate treatment or information about their condition.

Patients' health and social care is often poorly coordinated, with many struggling to access mental health services needed to help them cope with managing their rare condition.

The UK Strategy for Rare Diseases

The publication of the UK Strategy for Rare Diseases in 2013, represented a landmark for patients with rare diseases. It is the first time all four health departments of the UK have come together to recognise and respond to the needs of all those affected by rare diseases. The aim of the Strategy is to 'ensure no one gets left behind just because they have a rare disease'.

In the UK it is estimated that 3.5 million people will be affected by a rare disease at some point in their lives. Despite the scale of the issue, a significant proportion of these patients do not have access to the right care and treatment.

There are many examples of excellent practice for those with rare diseases throughout the UK, but unfortunately these are more often the exception rather than the rule.

The Strategy contains 51 commitments to ensure that health and social care systems across the four nations provide those living with rare conditions with the highest possible quality of evidence-based care and treatment, regardless of where they live in the UK.

Key Features Of The Strategy

  • A clear personal care plan for every patient that brings together health and care services.
  • Making sure patients, their families and carers have the information they need, are listened to and consulted.
  • Developing better methods of identifying and preventing rare diseases.
  • Improving diagnosis and earlier intervention for those with a rare disease.
  • Better education and training for health and social care professionals.
  • Building on research to improve personalised approaches to healthcare for those with a rare disease.

View the implementation plans for the nations below:

Scotland

Wales

Northern Ireland


Join us in watching the live debate