Government must commit to fully implementing the Rare Diseases Strategy
4 min read
While the Rare Diseases Strategy has been hailed as a breakthrough, poor implementation has caused worsening conditions in certain cases, says Ben Howlett MP.
As Chairman of the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions I have had the privilege of meeting and helping patients, children, their parents and families since 2015. The figures are extraordinary - 1 in 17 people will be affected by a rare disease at some point in their life which equates to approximately 3.5million people in the UK and there are currently between 6000 and 8000 rare diseases identified with patients being affected at all ages.
However, for those that have a known rare disease the picture is slightly better than for those that do not. Many children and adults that I have met do not have a diagnosis, not knowing is both deeply distressing and prevents adequate care being provided fully. This results in a wide range of issues for patients and parents who are often desperate to find an answer. Despite the scale of the issue, unfortunately a significant proportion of these still do not have access to adequate care and treatment.
Given the enormity of the problem in 2013 the Government published their Rare Diseases Strategy. This was heralded by the rare diseases and medical community as a major breakthrough. While symptoms vary from condition to condition, there are a number of issues that patients and families affected by rare diseases face collectively, for example, the difficulties associated with accessing a timely and accurate diagnosis and appropriate coordination of care.
The publication of the UK Strategy for Rare Diseases should have heralded a new era in the treatment and care of rare disease patients in England, Scotland, Wales and Northern Ireland. Containing 51 commitments, the Strategy aims to ensure that health and social care systems across the four nations provide those living with rare conditions with the highest quality of evidence-based care and treatment, regardless of where they live in the UK.
The aim of the UK Strategy is to “ensure no one gets left behind just because they have a rare disease.” Some of the features including setting a clear personal care plan for every patient that brings together health and social care services. Making sure patients, their families and carers have the information they need, are listened to and consulted. Improving diagnosis, intervention and strengthening research to improve personalised approaches to healthcare for those with a rare disease.
The four countries in the UK were given a deadline of 2020 to implement the commitments. While the Department of Health in the devolved nations have all published country-specific implementation plans that reflect their respective health service structures and priorities, the Department of Health in England has not coordinated a plan for England.
Between October 2016 and January 2017, the Rare, Genetic and Undiagnosed Conditions All Party Parliamentary Group conducted an inquiry into the implementation of the UK Strategy for Rare Diseases in England. It held three hearings with the Department of Health and its arm’s length bodies, and more than 300 patients, family members, patient organisations, clinicians and industry representatives also submitted evidence.
Overall the findings were that where the strategy is working well and being implemented effectively, it is doing very well. However, where the strategy is failing to be implemented the problems it was designed to fix are in some cases getting worse or at best failing to get better.
Just last week the Parliamentary Under Secretary of State for Public Health and Innovation Nicola Blackwood MP said that “the UK strategy for rare diseases needs to be translated into an implementation plan, and that is one of my personal commitments.”
However, the evidence we received suggested that the Department of Health does not intend to develop an implementation plan, and believes NHS England should assume responsibility for doing so. NHS England also provided evidence to suggest that it does not intend to develop an implementation plan, and neither has the remit nor the capacity to influence all 51 commitments in the strategy.
Many patients do not have access to appropriate treatment or information about their condition and the barriers to accessing information about a condition begin as soon as a diagnosis is made. The Government must commit to finding a way for either Department of Health to deliver this agenda, or for NHS England to be given direction to do so.
Ben Howett is the Member of Parliament for Bath and the Chairman of the APPG on Rare, Generic and Undiagnosed Diseases.
PoliticsHome Newsletters
Get the inside track on what MPs and Peers are talking about. Sign up to The House's morning email for the latest insight and reaction from Parliamentarians, policy-makers and organisations.
