“In the first draft guidance produced by its new Highly Specialised Technologies programme, NICE has asked Alexion Pharma UK, manufacturer of eculizumab (Soliris) for treating atypical Haemolytic Uraemic Syndrome (aHUS), to explain the high cost of the drug. The draft guidance also asks for advice from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken into account in formulating a recommendation.”
“The net budget impact of eculizumab based on the company's predicted rate of uptake over a 5-year period is confidential. However, to allow consultees and commentators to properly engage in the consultation process, NICE has prepared an illustration of the possible budget impact of eculizumab for aHUS, using information that is available in the public domain. This is based on a treatment cost of £340,200 per adult patient in the first year (based on the acquisition cost of the drug and the recommended dosing for an adult), and assumes a patient cohort of 170, as estimated by NHS England in its interim commissioning policy. If it is assumed that all of these adult patients with aHUS are treated with eculizumab, the budget impact for the first year would be £57.8 million. If an additional 20 new patients are treated the following year (based on a world-wide incidence of 0.4 million; see section 2.2), the budget impact will rise to £62.5 million in year 2, assuming all new patients are treated and all existing patients continue to be treated at the maintenance cost of £327,600 per year. Using the same assumptions, the budget impact will rise to £69 million in year 3 (190 existing and 20 new patients), £75 million in year 4 (210 existing and 20 new patients) and £82 million in year 5 (230 existing and 20 new patients).
The Committee acknowledged that the company's estimate of the incremental cost of eculizumab compared with standard care was considerable and that incremental costs estimated by the ERG were higher still (results are confidential).”