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NICE turns down bone disorder drug for NHS

Published on 03/12/15 at 10:23am

NICE has published draft guidance as part of its highly specialised technologies programme, not recommending Alexion Pharma’s Strensiq (asfotase alfa) for treating paediatric-onset hypophosphatasia.

It is estimated that around seven people in England are diagnosed with paediatric-onset hypophosphatasia each year: a serious, inherited, progressive and life-threatening genetic disorder that disrupts the process in which minerals such as calcium and phosphorus are deposited in developing bones.

The current mainstay of treatment is supportive care, which aims to monitor and alleviate symptoms to decrease morbidity and mortality and improve quality of life. The total cost per person per year of treatment with asfotase alfa is £366,912.

Meindert Boysen, technology appraisals programme director at NICE, justified the decision, saying that although the drug could provide considerable clinical benefit in certain patients, its price was too steep to justify use on the NHS.

“Based on the evidence presented by the company, as well as the testimony of clinical experts and patient representatives, the Committee concluded that asfotase alfa improved the probability of survival in perinatal- and infantile-onset hypophosphatasia compared with best supportive care,” he explains.

“The Committee concluded that, although asfotase alfa is an important development in the treatment of paediatric-onset hypophosphatasia with the potential to provide major benefits for some people with the condition, it does not represent value for money for the NHS.”

Asfotase alfa is the first therapy that specifically targets the underlying cause of hypophosphatasia. It is a targeted enzyme replacement therapy designed to restore the regulation of metabolic processes in the bones and teeth, and to reduce complications of dysregulated bone mineral metabolism.

NICE has not yet issued final guidance to the NHS, meaning its draft verdict could be altered following consultation considered by the Committee at its next meeting on 20 January 2016.

The draft guidance did however recommend that people whose treatment with asfotase alfa started within the NHS before the final guidance is published should be able to continue treatment until they and their NHS clinician consider it appropriate to stop.

Yasmita Kumar

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