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Orphan drugs

NICE rejects £360,000 therapy for rare paediatric bone disorder

Asfotase alfa, a novel treatment for a rare childhood bone disorder known as paediatric-onset hypophosphatasia has been provisionally rejected for NHS funding by NICE, which costs £366,912 per person per year. In the image, a new-born baby

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Paediatric-onset hypophosphatasia, a genetic disorder that affects some seven newborns and infants per year in England, is often fatal

A treatment for a rare childhood bone disorder has been provisionally rejected for NHS funding by the National Institute for Health and Care Excellence (NICE), which ruled the drug does not represent value for money.

NICE’s evaluation committee agreed that asfotase alfa (Strensiq, Alexion Pharma UK) had the potential to provide “major benefits” for some people with paediatric-onset hypophosphatasia. The condition is a genetic disorder that disrupts the deposition of minerals such as calcium and phosphorus in developing bones. Around seven newborns and infants a year in England are diagnosed with the disorder and it is often fatal. Asfotase alfa “improved the probability of survival” (compared with usual care) among newborns and infants with the condition.

In draft guidance, published on 3 December 2015, NICE concluded that the drug, which costs £366,912 per patient per year (for an average weight of 19.3 kg), “does not represent value for money for the NHS”, despite its potential benefits. The committee was unable to estimate from the clinical evidence the exact magnitude of longer-term benefits for patients.

The targeted enzyme replacement therapy, the first to target the causes of hypophosphatasia, is designed to restore the regulation of metabolic processes in bones and teeth and reduce complications of dysregulated bone mineral metabolism.

It was assessed as part of NICE’s highly specialised technologies programme, which only considers drugs for very rare conditions. Only one appraisal has so far been published through this programme – eculizumab for treating atypical haemolytic uraemic syndrome.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2015.20200215

Readers' comments (1)

  • I'm in total shock that this is even being discussed! This drug has helped my friends boy live a normal life, without it he wouldn't even be here today. How can you put this added stress onto those families needing this to survive each day. What if it was your child, or a friends or a neighbours who needed this life saving drug !!!!

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  • Asfotase alfa, a novel treatment for a rare childhood bone disorder known as paediatric-onset hypophosphatasia has been provisionally rejected for NHS funding by NICE, which costs £366,912 per person per year. In the image, a new-born baby

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