Credit: Globovision
The UK’s National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending eculizumab (Soliris) for funding to treat atypical hemolytic uremic syndrome (aHUS).
However, the agency has a few requirements. Eculizumab use must be coordinated through an expert center.
And monitoring systems must record the number of people with aHUS, the number who receive eculizumab, and the dose and duration of treatment.
NICE is also requiring a national protocol for starting and stopping eculizumab for clinical reasons and a research program with robust methods to evaluate when stopping treatment or dose adjustment might occur.
“[A NICE advisory] committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need,” said NICE Chief Executive Sir Andrew Dillon.
“Eculizumab offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis, and kidney transplantation, as well as other organ damage. The drug is, however, very expensive. The committee felt that the budget impact of eculizumab would be lower if the potential for dose adjustment and stopping treatment was explored.”
“This is reflected in the draft guidance, which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for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. In the meantime, NHS England and the company [developing the drug, Alexion Pharmaceuticals] should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.”
Eculizumab: Dosing, cost, and benefit
Eculizumab is given intravenously in adults as initial treatment at a dose of 900 mg for 4 weeks, then as maintenance treatment at a dose of 1200 mg on week 5, and then every 12 to 16 days. The summary of product characteristics for eculizumab states that “treatment is recommended to continue for a patient’s lifetime, unless discontinuation of treatment is clinically indicated.”
Eculizumab costs £3150 per 30 ml vial (excluding value-added tax). 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 available in the public domain.
NICE’s estimate 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 worldwide incidence of 0.4 per million), 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).
NHS England has indicated that the amount of the budget allocated for highly specialized services in 2013/2014 was £544 million, and the spending on high-cost drugs was £156 million.
The advisory 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 evidence review group were higher still (results are confidential).
The company estimated that eculizumab produced 25.22 additional quality-adjusted life-years (QALYs) per patient compared with standard care. Although the QALYs estimated in the evidence review group’s analysis were markedly lower than those calculated by the company, both analyses produced substantial QALY gains of a magnitude that is rarely seen for any new drug treatment.
NICE has not yet issued final guidance on eculizumab in aHUS. The draft guidance is now with consultees, including the company, healthcare professionals, and patient/carer organizations, who have the opportunity to appeal against the draft recommendations.
Final guidance on the use of eculizumab to treat aHUS is expected in January 2015.
