Credit: Bill Branson
The UK’s National Institute for Health and Care Excellence (NICE) has asked the manufacturer of eculizumab (Soliris) to explain the high cost of the drug.
Research has suggested that eculizumab can be effective against atypical hemolytic uremic syndrome (aHUS), a rare disease that often proves difficult to treat.
So the National Health Service (NHS) has made eculizumab available for these patients on an interim basis, pending NICE appraisal.
However, an advisory committee for NICE has estimated that routine use of eculizumab would cost the NHS about £58 million in the first year, and costs would exceed £80 million in 5 years.
Therefore, in its draft guidance for eculizumab, the committee has asked the drug’s manufacturer, Alexion Pharma, to explain its costs.
“[The committee has] asked for clarification from the company on aspects of the manufacturing, research, and development costs of a medicinal product for the treatment of a very rare condition,” said Sir Andrew Dillon, Chief Executive at NICE.
“It has also asked NHS England for clarification on treatment costs for a highly specialized technology in the context of a highly specialized service. The information provided will be considered at the next meeting of the evaluation committee in April.”
The committee will also consider comments on its draft guidance at the meeting. The guidance is available for public comment until midday on March 25.
About aHUS
Estimated to affect more than 200 people in England, aHUS is a chronic condition that causes severe inflammation of blood vessels and thrombus formation in small blood vessels throughout the body.
Patients with aHUS can experience significant kidney impairment, thrombosis, heart failure, and brain injury. In about 70% of patients, aHUS is associated with an underlying genetic or acquired abnormality of proteins in the complement immune system.
Before eculizumab became available, plasma therapy (infusion and/or exchange) was the main treatment for aHUS. However, not all patients with aHUS respond to plasma therapy. And up to 40% of patients may die or progress to end-stage renal failure and require dialysis with the first clinical aHUS manifestation, despite the use of plasma therapy.
Some patients may be eligible for a kidney or combined kidney-liver transplantation. However, there is a high risk of organ rejection following recurrent disease.
Eculizumab in aHUS: Treatment and cost
Eculizumab inhibits the disease process by blocking pro-thrombotic and pro-inflammatory processes that can lead to cellular damage in small blood vessels throughout the body, renal failure, and damage to other organs.
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 should be continued for the patient’s lifetime, unless discontinuation is clinically indicated.
Eculizumab costs £3150 per 30 mL vial, excluding tax, according to the British National Formulary.
“Alexion insisted that its information about the overall cost of eculizumab be kept confidential, and so NICE is unable to share these details of the Alexion submission with stakeholders,” Dillon said.
However, to allow consultees and commentators to properly engage in the consultation process, NICE has prepared an estimate of the possible budget impact eculizumab might have, using information 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.
Assuming all of these patients receive 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 million), the budget impact will rise to £62.5 million. That is 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).
