NICE Turn Down Bone Disorder Drug for NHS

Image and video hosting by TinyPicAs part of its highly specialised technologies programme, NICE have published draft guidance turning down Alexion Pharma’s Strensiq (asfotase alfa) which is used in the treatment for paediatric-onset hypophosphatasia.

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

The current foundation of treatment is supportive care, in which patients are monitored and their symptoms alleviated to decrease morbidity/mortality rates and improve quality of life. The total cost per person per year for treatment with asfotase alfa is £366,912.

“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,” explained Meindert Boysen, technology appraisals programme director at NICE.

“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 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. It is the first therapy that specifically targets the underlying cause of hypophosphatasia.

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The draft guidance did however recommend that people whose treatment with asfotase alfa started within the NHS before the final guidance is published should be able to continue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.


Industry News