In the first draft guidance produced by its new Highly Specialised Technologies programme, NICE has asked Alexion Pharma UK, manufacturer of eculizumab (Soliris) for treating atypical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 The draft guidance also asks for advice from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken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.

Estimated to affect over 200 people in England, aHUS is a chronic condition that causes severe inflammation of blood vessels and the formation of blood clots in small blood vessels throughout the body. People with aHUS may experience a considerable impact on their daily living and quality of life and can experience significant kidney impairment, thrombosis (blood clots), heart failure and brain injury. In around 70% of patients, aHUS is associated with an underlying genetic or acquired abnormality of proteins in the immune system called complement.

The prognosis for people with aHUS is poor, with early mortality rates ranging from 10% to 15% in the initial, acute phase of the disease. The majority of people with aHUS - up to 70% - develop end stage kidney failure requiring dialysis and transplantation. 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 inhibits the disease process by blocking pro-thrombotic and pro-inflammatory processes which in aHUS can lead to cellular damage in small blood vessels throughout the body, renal failure and damage to other organs.

Commenting on the draft guidance, Sir Andrew Dillon, Chief Executive at NICE, said: “Drugs for very rare conditions that affect just a few people in the country are inevitably more expensive than for more common diseases. Companies have to recover their costs at much lower volumes.

“Eculizumab is an effective treatment for aHUS. The NHS has already recognised this by making it available on an interim basis, pending the outcome of NICE's appraisal. However, before we can make a confident recommendation for routine use, which we estimate would cost the NHS about £58 million in the first year, rising to over £80 million in 5-years, we need more information.

“Our independent advisory committee has therefore 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. It has also asked NHS England for clarification on treatment costs for a highly specialised technology in the context of a highly specialised service. The information provided will be considered at the next meeting of the Evaluation Committee in April.”

"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. We're disappointed about this decision, for which we have not had an adequate explanation."

NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. Consultees, including the company, healthcare professionals, patient/carer organisations and members of the public are now able to comment on the preliminary recommendations which are available for public consultation until midday on Tuesday, 25 March. Comments received during this consultation will be fully considered by the Committee at its next meeting in April and, following this meeting, the next draft guidance will be issued.

In the meantime, the current interim commissioning arrangements by NHS England in line with the “Clinical Commissioning Policy Statement: Eculizumab for atypical haemolytic uraemic syndrome” will remain in place.

Ends

Notes to Editors

About the draft guidance

1. The draft guidance on eculizumab will be available on the NICE website from 00:01, 4 March 2014.

About eculizumab

2. 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 12-16 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”

3. Eculizumab costs £3150 per 30 ml vial (excluding VAT; ‘British national formulary' [BNF], January 2014).

4. 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).

5. 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).

6. The company estimated that eculizumab produced 25.22 additional QALYs per patient compared with standard care. Although the QALYs estimated in the ERG'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.

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