National Institute for Health and Clinical Excellence (NICE)
First NICE highly specialised technologies guidance recommends eculizumab (Soliris) for treating very rare life-threatening blood disorder
NICE has yesterday issued final guidance recommending eculizumab (Soliris, Alexion) for funding for treating atypical Haemolytic Uraemic Syndrome (aHUS). The guidance is the first to be produced as part of NICE’s highly specialised technologies programme to evaluate treatments for very rare conditions.
aHUS is a life-threatening disease affecting around 200 people in England, with 20–30 new patients diagnosed with the condition each year. It 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.
“aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families. We are therefore pleased to be able to recommend eculizumab for funding” said NICE Chief Executive Sir Andrew Dillon.
“The 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. 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 guidance states that the following arrangements need to be in place:
- coordination of eculizumab use through an expert centre
- monitoring systems to record the number of people with a diagnosis of aHUS and the number who have eculizumab, and the dose and duration of treatment
- a national protocol for starting and stopping eculizumab for clinical reasons
- a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.
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 guidance
- The guidance on eculizumab is available on the NICE website
- 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.
- 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)
- Eculizumab costs £3150 per 30 ml vial (excluding VAT; ‘British national formulary’ [BNF], January 2014).
- 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).
- 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.
- 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).
- 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.
About the NICE highly specialised technologies (HST) programme
- NICE took over responsibility for evaluating the suitability of very high cost drugs for patients with very rare conditions for national commissioning from April 1 2013. Before that date the Advisory Group for National Specialised Services (AGNSS) was responsible for advising ministers which highly specialised services, products and health technologies should be, or no longer be, nationally commissioned.
- The recommendation(s) contained within NICE HST guidance to the NHS will be subject to the funding direction as per NICE technology appraisal guidance. Therefore, in developing how a highly specialised service is to be directly commissioned, NHS England is subject to the same requirements as the rest of the NHS regarding a technology for which NICE has issued guidance through the programmes in which the funding direction applies.
- Topics evaluated by the NICE HST programme are subject to a formal referral from the Department of Health to NICE.
- The interim statement on the process and methods used to develop NICE HST guidance is available on the NICE website.
 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 (http://www.england.nhs.uk/ourwork/commissioning/spec-services/).
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for driving improvement and excellence in the health and social care system. We develop guidance, standards and information on high-quality health and social care. We also advise on ways to promote healthy living and prevent ill health.
Our aim is to help practitioners deliver the best possible care and give people the most effective treatments, which are based on the most up-to-date evidence and provide value for money, in order to reduce inequalities and variation.
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