Burosumab for the treatment of X-linked Hypophosphataemia (XLH) rejected by NICE in draft guidelines – have your say to help make a difference

Following the recent evaluation meeting, the National Institute for Health and Care Excellence (NICE) has issued draft guidelines rejecting NHS funding for the drug burosumab for children and young people with X-linked Hypophosphataemia (XLH), 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 XLH, an Inherited Metabolic 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.

 Current treatment mainly consists of vitamin D supplementation and oral phosphate, and is aimed at improving growth, lessening the severity of symptoms and preventing skeletal abnormalities.

 Burosumab is the first treatment to target the underlying pathophysiology of XLH. An injection given every two weeks from the age of one until the skeleton stops growing, aims to increase reabsorption of phosphate from the kidney and, through vitamin D production, improve intestinal absorption of calcium and phosphate.

 However, while evidence suggests the drug is more clinically effective than conventional therapy and provides clinical benefits in children aged between one and 12 years, NICE summise that the data is “limited and uncertain”.

 There is a window of opportunity for patients, family members, carers and the public to comment on this draft recommendation. The deadline for comments is 6th July 2018. The evaluation committee is interested in receiving comments on the following:

 a) How do symptoms (both physical and psychological) and treatments, including any surgery, you or your child experienced in childhood affect you or your child in adult life?

 b) For children on standard treatment (phosphate and calcitriol), what are the side effects of taking current treatment? How is that a burden for the child and your family?

 c) If you have a child who is 1-12 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.

 d) If you have a child who is 13-17 years old and on burosumab, please explain how this treatment is currently helping in the short-term and how you expect that it will help in the long-term.

To have your say on this draft guidance, and read the previous committee papers, please register on the NICE website here: https://www.nice.org.uk/guidance/indevelopment/gid-hst10016/consultation/html-content 

It is important that your stories and experiences are heard in this process so please have your say. Together our voices are stronger.