Kuvan: High court case challenges NICE’s appraisal

A case that is due to be heard in the High Court on 30 January will challenge how the UK National Institute for Health and Clinical Excellence planned to review the orphan drug sapropterin dihydrochloride (marketed as Kuvan), a treatment for the rare genetic disorder phenylketonuria (PKU).

The judicial review revolves around 11 year old Cait Cotter, who has the disorder and whose claim is being funded through legal aid.

The fact that NICE chose to use its standard technology protocol to appraise sapropterin for use on the NHS made approval less likely, said Cotter’s lawyer Peter Todd, leaving patients like his client unable to get the treatment on the NHS. He claims that NICE should instead have used the highly specialised technology evaluation, which was introduced in 2013 for appraisal of drugs for very rare conditions, …