A drug to treat a very rare blood disorder affecting around 200 people in England has been recommended by the National Institute for Health and Care Excellence in further draft guidance.  

Produced by Alexion, eculizumab (Soliris) treats atypical Haemolytic Uraemic Syndrome (aHUS), an extremely rare but life-threatening disease which causes inflammation of blood vessels and the formation of blood clots throughout the body. People with aHUS are at constant risk of sudden and progressive damage to, and failure of vital organs, particularly the kidneys.

Eculizumab is being evaluated as part of a new programme at NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price.

NICE’s estimates that this drug will cost the NHS up to £58million in the first year, rising to £82million after 5 years. (See notes to editors below for more detail.) It is currently funded by NHS England through interim specialised commissioning arrangements. 

As well as having a significant negative impact on quality of life, the prognosis for people with aHUS is poor, with around 10% to 15% of people with the disease dying in the initial, acute phase. The majority of people with aHUS – up to 70% – develop end stage kidney failure requiring dialysis. One patient in 5 has aHUS affecting organs other than the kidneys, most commonly the brain or heart.

In around 70% of patients, aHUS is associated with an underlying genetic or acquired abnormality of proteins in the immune system. 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Commenting on the draft guidance, NICE Chief Executive Sir Andrew Dillon said: “Eculizumab radically improves the quality of life of the small number of people with aHUS. Until it became available, people with aHUS were at risk of kidney failure needing dialysis, other organ failure, and early death.

“Plasma therapy and dialysis were the main treatments, both of which had a limited impact on managing symptoms and reducing early deaths, but a substantial negative effect on a patient’s quality of life.

“From the available evidence and from the testimony of clinicians and patients, families and carers, it is clear that eculizumab is a significant breakthrough in the management of aHUS. The drug offers people with the disease the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.

“The drug is, however, very expensive. In making its decision the independent Evaluation Committee needed to take into account the total fixed budget for highly specialised services as a whole and how it is allocated.

“The Committee felt that the budget impact of recommending eculizumab for aHUS in relation to the substantial benefits it offers would be lower if the potential for dose adjustment and stopping treatment was taken into account. Therefore the draft guidance recommends that eculizumab is funded only if important conditions are met. These include coordinating the use of eculizumab through an expert centre and putting in place systems for monitoring how many people are diagnosed with aHUS, how many receive the drug, at what dose and for how long. The programme also needs to develop protocols for starting and stopping treatment with eculizumab for clinical reasons and introduce a research programme to collect data to evaluate when stopping treatment or adjusting the dose of the drug might occur.”

Given that the budget impact of eculizumab for treating aHUS will be considerable, the draft guidance also recommends that NHS England and the company should consider what opportunities might exist to reduce the overall cost of Eculizumab to the NHS.

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 Thursday, 25 September. Comments received during this consultation will be fully considered by the Committee at its next meeting in October 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

For more information call the NICE press office on 0300 323 0142 or out of hours on 07775 583 813.

Notes to Editors

About the draft guidance

1. The draft guidance on eculizumab will be available on the NICE website from 00:01, 4 September 2014. Embargoed copies of the draft guidance are available from the NICE press office on request.

About eculizumab

1. 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.” (“See section 4.4 Special warnings and precautions for use of Soliris Summary of Product Characteristics)
2. Eculizumab costs £3150 per 30 ml vial (excluding VAT; ‘British national formulary’ [BNF], January 2014).
3. 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 per 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).
4. The Committee noted that NHS England has indicated that the amount of the budget allocated for highly specialised services in 2013/14 was £544 million and the spend on high cost drugs was £156 million.
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.