Credit: Globovision
The UK’s National Institute for Health and Care Excellence (NICE) has issued a final guidance recommending eculizumab (Soliris) as a treatment for atypical hemolytic uremic syndrome (aHUS), but only if certain conditions are met.
The guidance is the first to be produced as part of NICE’s highly specialized technologies program to evaluate treatments for very rare conditions.
aHUS affects around 200 people in England, with 20 to 30 new patients diagnosed with the condition each year.
The condition causes inflammation of blood vessels and thrombus formation throughout the body. So aHUS patients are at constant risk of sudden and progressive damage to, and failure of, vital organs.
“aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families,” said NICE Chief Executive Sir Andrew Dillon.
“[A committee advising NICE] 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. It 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 adjusting the dose of the drug and stopping treatment was explored. 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. In the meantime, NHS England and the company should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.”
The conditions for funding eculizumab are as follows:
- Use of the drug must be coordinated through an expert center.
- Monitoring systems must record the number of people with aHUS, the number who receive eculizumab, and the dose and duration of treatment.
- A national protocol must be developed for starting and stopping eculizumab for clinical reasons.
- A research program is needed with robust methods to evaluate when stopping treatment or dose adjustment might occur.
Eculizumab usage and costs
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 tax).
The net budget impact of eculizumab is confidential. However, 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.
If it is assumed that all of these adults 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 2013/14 was £544 million, and the money spent on high-cost drugs was £156 million.
The committee acknowledged that the estimate of the incremental cost of eculizumab made by Alexion Pharmaceuticals (the company developing eculizumab) compared with standard care was considerable. And incremental costs estimated by the evidence review group were higher still (although results are confidential).
Alexion 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, the advisory committee said both analyses produced substantial QALY gains of a magnitude that is rarely seen for any new drug.
For more information, see the guidance on the NICE website.
