Pledge for Patients Reaches UK Parliament

  • Portrait by Quentin Matsys. Michael Baum, emeritus professor of surgery at University College London, suggests that the sitter had the rare condition Paget's bone disease | Wikimedia Commons

170 prospective parliamentary candidates from ten different political parties based across the UK backed the Pledge for Patients campaign. Of these, 40 were elected to Parliament.  This comes as a result of Rare Disease UK’s efforts to help fight for patients with rare illnesses.

There are around 7,000 diseases that are classified as rare, which means that they affect less than 200,000 patients in the US or no more than 5 to 10,000 people living in the European Union.

These diseases are usually passed on through ancestral genes.  They often affect patients early in childhood and are progressive, disabling and life-threatening in nature.  They can have a devastating psychological impact on friends and relatives of young patients who suffer from such conditions.

A few of the better-known examples of such conditions include Duchenne’s muscular dystrophy, cystic fibrosis, mucopolysaccharidoses (MPS) and rare cancers.  A total of 55 million people in the US and the EU suffer from uncommon diseases, which highlights their huge societal impact.  New laws have been passed all over the world to incentivise the creation of new specialist drugs and treatments that will help people cope with and recover from such rare conditions.

95% of patients are still not able to access the most effective treatments and therapies that will help them cope with or manage their conditions.  There is generally a very small number of approvals for orphan drugs so this means the majority of patients will not be able to access the correct treatments.