Patients with the chronic, ultra-rare and life-threatening blood disease Haemolytic Uraemic Syndrome (aHUS) will no doubt welcome news of continued access to an expensive but ‘game-changing’ therapy on the National Health Service in England.

The National Institute for Health and Care Excellence has now published a Final Appraisal Determination for Alexion’s Soliris (eculizumab), endorsing its use under a new programme designed to assess highly-specialised technologies which may benefit people with very rare diseases at a high price.

The Institute’s Highly Specialised Technologies Evaluation Committee has recommended that Soliris be commissioned for all patients with the disease, securing life-long access to the first and only available treatment for the condition.

Around 200 people in England are affected by aHUS, leaving them at constant risk of sudden and progressive damage to and failure of their vital organs leading to death. In fact, early mortality rates are 10%-15% in the initial, acute phase of the disease, while up to 70% of patients develop end-stage renal failure.

Experts agree that Soliris - a humanised monoclonal antibody - can radically improve the quality of life for people with aHUS, and that the drug is a significant breakthrough in the management of the condition. However, its price-tag ranks amongst the highest in the world. According to the Institute, based on a treatment cost of £340,200 per adult patient in the first year, and assuming a patient cohort of 170, Soliris would cost the NHS £57.8 million in the first year, rising to £82 million in year five.

Cost 'overstated'

However, a spokesman for Alexion told PharmaTimes Digital that the cost has been overstated, because the estimate assumes that every patient with the disease will receive treatment from day one, which is simply not the case. Also, it assumes that all patients are adults, when around 40% are not and so receive lower-dose treatment.

In addition, the Pharmaceutical Price Regulation Scheme (PPRS) ensures that every pound spent on medicines over 2013’s spend will be rebated by the industry, so the end result is net zero cost to the NHS, he argues.

Soliris is a “reasonable use of NHS resources,” given the severity of the disease, lack of any other treatment, and the magnitude of benefit that the drug offers patients, the spokesman told PT. The drug is already available to patients having been approved through the old AGNSS system of assessing low volume, high cost medicines, but NICE’s stamp of approval means that patients now have certainty of access, he noted.