Posted by Michael Wonder on 16 Jun 2018
Rare disease therapy Crysvita to be barred from NHS
15 June 2018 - Children and young people with X-linked hypophosphataemia are unlikely to get routine access to Kyowa Kirin’s Crysvita on the NHS.
In draft guidelines, the NICE has rejected NHS funding for the drug, after concluding that the most likely cost-effectiveness estimates are much higher than would normally be considered value for money for highly specialised technologies.
There are around 250 children and young people in England with X-linked hypophosphataemia, an inherited genetic disorder that causes low levels of phosphate in the blood. This leads to soft, weak bones, and children with the condition usually have bowed or bent legs, short stature, bone pain and delayed walking, and may also have dental problems and hearing loss.
