NICE Approves of Roche's MabThera for Rare Autoimmune Disorder
NICE says yes to treatment for rare autoimmune disease in draft guidance
NICE has today (3 October) published draft guidance recommending rituximab (MabThera, Roche Products) as an option for treating some adults with the rare autoimmune condition, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
Rituximab is licensed for inducing remission in adults with a type of vasculitis (inflammation of the blood vessels) called anti-neutrophil cytoplasmic antibody [ANCA]-associated vasculitisi (severely active granulomatosis with polyangiitis [GPA] and microscopic polyangiitis). NICE has recommended rituximab in combination with glucocorticoidsii as an option for this patient group, only if further cyclophosphamideiii treatment would exceed the maximum cumulative cyclophosphamide dose.
ANCA-associated vasculitis is an inflammatory autoimmune disease affecting blood vessel walls. It can affect many organs and leads to tissue breakdown and damage. GPA and microscopic polyangiitis are both types of ANCA-associated vasculitis that affect small blood vessels. ANCA-associated vasculitis usually affects the lungs, kidneys, ears, nose or sinuses. Depending on the organs involved it can cause bleeding, rash, or deafness. It is estimated that around 1200 people are diagnosed with ANCA-associated vasculitis each year in England and Wales, and it is most common in those aged between 60 and 70 years.
The aim of treatment is initially to induce remission, then to maintain remission and treat relapse when necessary. With adequate ongoing care, most people with ANCA-associated vasculitis will have a good quality of life and normal life expectancy.
NICE consulted on the first draft guidance in June 2013, and asked for further information from the manufacturer. NICE is now publishing a second draft guidance document to allow consultees to comment on the information submitted by the manufacturer.
Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: "ANCA-associated vasculitis is a rare but serious autoimmune disease and causes the white blood cells to attack the walls of small vessels in different tissues and organs of the body. NICE is, therefore, pleased to recommend rituximab as an option for some people with this condition in draft guidance."
For more information, please call the NICE press office on 0845 003 7782, and (out of hours) on 07775 583 813 or email email@example.com
The draft guidance (appraisal consultation document / ACD) can be found from 00:01hrs on Thursday 3 October on the NICE website
NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its guidance on a technology it replaces local recommendations across the country.
Final guidance is expected to be published in March 2014.
Notes to Editors
References and explanation of terms
i. ANCA-associated vasculitis is an umbrella term for several related conditions, including microscopic polyangiitis (MPA) and granulomatosis with polyangiitis (GPA). ANCA stands for anti-neutrophil cytoplasmic antibody. ANCAs are autoantibodies that affect neutrophils (a type of white blood cell). The disease mechanisms that cause damage in ANCA-associated vasculitis are not fully understood, but it is thought that neutrophils have an important role. After being activated by ANCAs, neutrophils attack the walls of small vessels in different tissues and organs of the body. This causes vasculitis.
ii. Glucocorticoidsare a type of steroid hormone. Glucocorticoid drugsare commonly used to reduce inflammation and suppress the immune system.
iii. Cyclophosphamide belongs to a class of drugs known as alkylating agents, which are used to treat some types of cancer. It can also suppress the immune system. Induction treatment with cyclophosphamide is the standard of care for patients with severe ANCA-associated vasculitis. Cyclophosphamide has the potential to cause serious short term side effects including bladder irritation (cystitis) and bone marrow suppression (low white cell count) leading to infection. Over a longer period of time, cyclophosphamide can cause infertility in both men and women.
About the draft guidance
1. The draft guidance is available from 00:01hrs on Thursday 3 October on the NICE website.
2. Closing date for comments is Thursday 24 October 2013. The next appraisal committee meeting is Wednesday 27 November 2013
3. Final guidance is expected to be published in March 2014.
4. Rituximab (MabThera, Roche Products) is a genetically engineered chimeric (mouse/human) monoclonal antibody that depletes B cells by targeting cells bearing the CD20 surface marker.
5. Rituximab is priced at £174.63 per 10 ml vial and £873.15 per 50 ml vial (excluding VAT; British national formulary [BNF] edition 66). The manufacturer's estimate of the average cost of a course of treatment is £4889.64 (based on 1.79 m2 body surface area and no vial sharing). Costs may vary in different settings because of negotiated procurement discounts.
6. A patient access scheme has not been submitted by the manufacturer.
7. People currently receiving rituximab as an induction treatment for ANCA-associated vasculitis, who do not fulfil the criteria in section 1.1 of the preliminary recommendations, should have the option to continue therapy until they and their clinicians consider it appropriate to stop.
8. The Committee concluded that the most plausible treatment sequence for patients who can receive cyclophosphamide was 2 cycles of cyclophosphamide followed by 1 cycle of rituximab. The Committee noted that 2 cycles of intravenous cyclophosphamide would provide a cumulative dose of 23 g on average, which is below the maximum recommended dose of 25 g.
9. The Committee agreed that the most plausible ICER on which to base its decision for people with ANCA-associated vasculitis who can have cyclophosphamide was £12,100 per QALY gained for the comparison of 2 courses of cyclophosphamide followed by 1 course of rituximab with 2 courses of cyclophosphamide. It concluded that rituximab is a cost-effective use of NHS resources for people with severe ANCA-associated vasculitis who can have cyclophosphamide, only if further treatment with cyclophosphamide would exceed the maximum cumulative dose.
10. The Committee decided there was insufficient evidence to determine whether, for patients who cannot receive cyclophosphamide, rituximab was a cost-effective use of NHS resources. Given the uncertainty about the appropriate comparator and the clinical effectiveness of treatments for this subgroup, the Committee was unable to identify the most plausible ICER for patients who cannot receive cyclophosphamide. Therefore, the Committee could not make a recommendation on the use of rituximab for patients with ANCA-associated vasculitis who cannot receive cyclophosphamide.
Related published NICE guidance
There is no related guidance for this technology at the time of consultation.
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.
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This page was last updated: 02 October 2013
Posted: October 2013