A few months ago, we noticed news from England that Britain’s national health service (NHS) rejected use of Xolair for after the National Institute for Clinical Excellence (NICE) said it was too expensive. NICE also said that Xolair, known generically as omalizumab, provided “little additional benefit over existing drugs for the treatment of severe persistent allergic asthma in most children aged 6-11”.
We decided to send the news for comment to Dr. Janet Sullivan, chief medical officer for Hudson Health Plan, New York State’s top-rated health plan for Medicaid and Child Health Plus, since it seemed to be the most directly comparable to the UK’s NHS of any payer we know. We also showed it to our own panel of experts—Dr. Chiaramonte and Dr. Ehrlich. Their comments follow Dr. Sullivan’s.
We do cover Xolair, but we require prior approval. The drug is VERY expensive, and we want to be sure it is used appropriately. I recently approved a case that we had sent out to an allergist for review. The reviewer recommended we NOT approve Xolair because the child’s IgE levels were higher than those for which the drug was tested, and was therefore “off-label.” Because the child had significant morbidity from asthma, I discussed the case with the referring allergist and we agreed on a few months’ trial.
I am not familiar with the studies that were reviewed by NICE but their criteria for effectiveness are stronger than ours. The pharmaceutical lobby in the United States is so powerful that new drugs are not required to demonstrate benefit in comparison to existing treatments and the cost of a treatment is not considered in the FDA approval process. A new drug can be FDA approved even if it is less effective than an existing treatment and costs ten times more. In my opinion, this policy benefits drug companies more than it benefits patients.
On the other hand, limitations on the clinical setting for which a drug is approved can be arbitrary. When a drug is approved for a certain age range, or IgE level, it is because those were the conditions that were tested. It doesn’t necessarily mean that the drug is unsafe or ineffective for people in a different age range; it just hasn’t been tested.
When appropriately used, a very expensive drug like Xolair can be both a good medical and economic choice if it improves the patient’s quality of life and prevents repeated ER visits and hospital admissions. That is why we review each case.
Dr. Janet Sullivan–Chief Medical Officer, Hudson Health Plan, Tarrytown, NY
Xolair is not approved by the FDA for use in children less than 13 years of age. Successful multi-center clinical trials are required before the FDA will grant approval. Because of its cost [approximately $1,000/per treatment], Xolair is limited to severe persistent asthmatics who have required systemic steroids in adults. This would be the case if approval is obtained for children.
I analyzed twenty adult patients who met the criteria for Xolair, using the standardized Asthma Control Test [ACT.} An ACT score of a completely well patient is between 20-25 points; below 20 points indicates asthma of increasing severity as the score lessens. The twenty patients studied had scores between 8-10 points. The ten asthmatics who did not get Xolair because they lacked HMO approval remained at their original ACT scores. The ten who received Xolair had ACT scores of 18-20 points within four months of treatment. If Xolair is used in children improvement is expected to follow the adult pattern of occurrence within four months.
Dr. Larry Chiaramonte
I find the NHS decision absolutely ridiculous. The studies which already have been done in the US in children six to eleven have shown Xolair to be not only effective but also money saving. The severe asthmatics might end up costing between $5,000 and $10,000 per visit in hospitalization thus approaching one-year cost of Xolair. This doesn’t even include the cost of medication on an outpatient basis. I look forward to FDA approval, which should be in about 12 months. I follow UK medical choices because I worked at a prestigious London hospital for a time many years ago and still have friends who practice there. Based on that experience and interest, I know a UK political story when I see one.
Dr. Paul Ehrlich