National Health Service cost regulators have approved funding for Alexion’s Soliris (eculizumab) as a treatment for the ultra-rare blood disease Haemolytic Uraemic Syndrome (aHUS), at a potential cost of £340,000 per patient.
The Institute’s Highly Specialised Technologies Evaluation Committee - responsible for evaluating the suitability of very high cost drugs for patients with very rare conditions for national commissioning - has recommended Soliris 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. Soliris can radically improve the quality of life for patients, and the drug is widely considered a significant innovation in the management of the condition.
But the drug is breathtakingly expensive, its price-tag ranking among the highest in the world. The Institute says that, 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.
However, the budget impact would be lower if the potential for adjusting the dose of the drug and stopping treatment was explored, NICE said. “This is reflected in the guidance which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for clinical reasons,” noted chief executive Sir Andrew Dillon.