In a final appraisal determination released August 12, NICE turned down use of Novartis' asthma therapy Xolair (omalizumab) in children aged 6 to 11 years, a decision that angered patient charities and again raised the issue of there being a "postcode lottery" for access to medicines in the U.K.

NICE's guidance applies only to the National Health Service in England and Wales, but not to the NHS in Scotland. Scotland's own health technology assessment body, the Scottish Medicines Consortium, recommended Xolair for use in younger children in April.

So a patient's postcode, which is similar to U.S. zipcode, would indicate whether they could receive the product. However, it is only a rough marker for access - the important factor for drug access would be whether the patient was being treated by doctors working for NHS Scotland or by doctors working for the NHS in England and Wales.

NICE concluded in its final appraisal determination that there was little extra benefit associated with the use of Xolair compared with existing treatments, in children aged 6-11 years with severe persistent allergic asthma. "There is no proven reduction in hospitalization rates, accident and emergency visits, unscheduled doctor visits or total emergency visits for children in this age group treated with omalizumab," said the deputy chief executive of NICE, Gillian Leng. The final appraisal determination therefore stands by the rejection of Xolair use in younger patients made in the initial appraisal determination two months ago ('The Pink Sheet' DAILY, June 3, 2010).

NICE's analysis found that omalizumab was only useful in reducing the rate of clinically significant exacerbations in children who were having three or more exacerbations per year. But in those patients, NICE calculates that Xolair would be associated with a cost of £82,600 ($129,000) per quality adjusted life year (QALY), much higher than the £30,000 or so usually considered to be the upper limit for NHS use.

In the UK, Xolair costs £256.15 per 150 mg vial, and the annual cost of treatment ranges from £1,655 to £26,640, depending on the doses used. NICE calculated an incremental cost effectiveness ratio (ICER) for all patients of £65,900 to £91,200 per QALY.

Is the Decision Unfair?

Xolair is already recommended by NICE for use in children aged 12 years and above, and so rejection of its use in younger patients would appear to be based on age and therefore unfair, a finding that would be grounds for an appeal.

However, the institute has anticipated that criticism. "Our recommendation is not based on the age of patients, but rather on the fact that the evidence implies the use of omalizumab is not cost-effective in this younger age group," it argues.

Nonetheless, NICE's appraisal committee said it was unhappy with having several guidances covering the same product, and would press for NICE to develop a single guidance.

Novartis said that it was disappointed with the final appraisal determination, particularly as Xolair was only expected to be used in a very small number of patients, around 100, and should be considered to be an ultra-orphan drug with a low budgetary impact. At this time, however, it was not saying if it would mount an appeal against the final appraisal determination - the guidance is scheduled to become final next month.

Shifting the Fight

The patient charity, Asthma UK, said that parents whose children have taken Xolair had reported dramatic improvements in their children's asthma and quality of life.

"For parents of children with severe asthma aged 6-11 who have already trialled Xolair and had their lives transformed, this news will come as a massive blow. It's vital that these children do not have the treatment withdrawn by primary care trusts as a result of the NICE decision, as this would be completely unjust."

Asthma UK said it would continue to fight for omalizumab to be made available to people of all ages with severe asthma throughout the UK, although it indicated that it was not currently considering an appeal against the final appraisal determination.

- John Davis (j.davis@elsevier.com)

NICE Rejects Novartis’ Xolair Use In Younger Children

More from Archive

More from Pink Sheet