The National Institute for Health and Care Excellence has published draft guidance in which it says it is "minded not to recommend" Roche's MabThera as a treatment for a rare autoimmune disease.

NICE has been examining MabThera (rituximab)'s use in its licensed indication for inducing remission, in combination with glucocorticoids, in adults with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, an inflammatory autoimmune disease affecting blood vessel walls.

Around 1,200 people are diagnosed with ANCA-associated vasculitis each year in England and Wales, most commonly in people aged 60-70. This rare but serious autoimmune disease causes the white blood cells to attack the walls of small vessels in different tissues and organs of the body, usually affecting the lungs, kidneys, ears, nose or sinus.

Depending on the organs involved it can cause bleeding, rash or deafness, and 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, but without treatment, it can be fatal, says NICE.

"Unfortunately, [NICE's] independent committee was minded not to recommend rituximab for this condition because of gaps and uncertainties in the evidence submitted by the manufacturer. The next step is for the manufacturer to respond to the committee's comments and submit further information as requested," said Professor Carole Longson, director of NICE's health technology evaluation centre.

NICE is requesting further clarification from Roche, including: - the definition of severe disease and the subgroup of people for whom avoiding treatment with cyclophosphamide is desirable; - further data to show ritiximab's benefits in the longer term; and - a revised economic model which represents the management of severe ANCA-associated vasculitis in the UK, including current comparators and routine clinical practice.

The drug is priced at £174,63 per 10ml vial and £873.15 per 50ml vial, excluding VAT, and the company estimates the average cost of a course of treatment at £4,889.64. NICE’s independent committee included that none of the incremental cost-effectiveness ratios (ICERs) presented by Roche and the Evidence Review Group (ERG) provided an accurate cost-effectiveness estimate for rituximab in this indication.

NICE points out that a patient access scheme has not been submitted by the manufacturer. The closing date for comments is August 12, and the Institute expects to publish final guidance in November