NHS England has announced plans to establish a national service to treat the ultra-rare kidney condition Atypical Haemolytic Uraemic Syndrome (aHUS), and the administration of Alexion's high-cost Soliris.
A collaboration between Newcastle Upon Tyne Hospitals NHS Foundation Trust and Newcastle University has been awarded a contract by the organisation to oversee the treatment of patients with the condition, which can lead to renal failure.
Around 90 people in England are currently receiving treatment for the aHUS, a chronic, progressive condition causing severe inflammation of blood vessels and the formation of blood clots in the small blood vessels throughout the body.
Up to 70% of patients diagnosed with the disease will progress to end stage renal failure within a year, requiring dialysis. Around 60% of aHUS cases are detected in children and, in 70% of cases, before the child reaches the age of two.
In January last year, the National Institute for Health and Care Excellence approved use of Alexion's Soliris (eculizumab), the only known effective treatment for this condition, by the National Health Service. But the drug doesn't come cheap, with a potential price tag of £340,000 per patient.
As such, the Institute stipulated a number of recommendations attached to its use, including the development of a national database for monitoring the use of the drug, as well as coordination of its prescribing through an expert centre.
Under the deal awarded by NHS England, specialists at Newcastle FT will provide expert diagnostic services, as well as advice to local hospitals on the ongoing treatment of patients with aHUS with Soliris.
The partnership will also help develop national policy and resources, including establishing a register of all patients, counselling services for those for whom treatment is not effective, and further research on the drug's most-effective use, it noted.