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.
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.
Commenting on the draft guidance, NICE Chief Executive Sir Andrew Dillon said: "The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a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.
"Eculizumab 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 dose adjustment and stopping treatment was explored. This is reflected in the draft guidance which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for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."
NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. The draft guidance is now with consultees, including the company, healthcare professionals and patient/carer organisations who now have the opportunity to appeal against the draft recommendations.
Until NICE issues its final guidance 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.
Final guidance on the use of eculizumab to treat aHUS is expected in January 2015.
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About the draft guidance
- The draft guidance on eculizumab will be available on the NICE website from 00:01, 27 November 2014.
- 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.
 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/).
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