MPs debate government’s rare diseases strategy

Last week, MPs in the House of Commons debated the government’s Strategy for Rare Diseases, first published in 2013. Ben Howlett, MP for Bath and Chair of the All-Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions, said that this “should have heralded a new era of treatment and care for rare diseases” – but there remains a question mark over the extent to which this has actually happened.


MPs have raised concern that there is no implementation plan for the strategy, due to uncertainty over whether the responsibility for this lies with the Department of Health or NHS England. Both bodies have indicated that they do not intend to develop an implementation plan. This risks undermining the Strategy and making life even more difficult for patients with rare diseases such as Dyskeratosis Congenita.


Patients with rare diseases are often left facing barriers to care, such as difficulties associated with accessing a timely and accurate diagnosis, or having to travel huge distances for treatment. The Strategy aims to overcome some of these barriers through measures such as a personal care plan for every patient, improving access to information, and investing in research into rare and genetic conditions.


As Mr Howlett noted, one in 17 people will be affected by a rare disease at some point in their life, equating to around 3.5 million people across the UK. The Strategy for Rare Diseases pledged to “ensure no one gets left behind just because they have a rare disease”. We at DC Action urge the government to make this a reality, ensuring that the health system provides these patients with the best possible care and treatment.


During the debate, MPs noted that many people with a rare disease struggle to get a diagnosis. Take a look at the ‘Education’ section on our website to find out more about the symptoms of Dyskeratosis Congenita and what can be done to treat them.