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    • Has all of the relevant evidence been taken into account?

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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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Draft guidance

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1 Recommendations

1.1

Burosumab is not recommended, within its marketing authorisation, for treating X‑linked hypophosphataemia (XLH) in adults.

1.2

This recommendation is not intended to affect treatment with burosumab that was started in the NHS before this guidance was published. Adults having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

Usual treatment for XLH is oral phosphate and active vitamin D. Burosumab is used in the NHS for treating XLH in people under 18; this evaluation is for treating XLH in adults.

Clinical trial evidence shows that burosumab increases the level of phosphate in the blood more effectively than placebo. This evidence also suggests that people having burosumab may have less pain and fatigue, and improved physical functioning compared with placebo in the short term, but this is uncertain.

There are uncertainties in the assumptions used in the economic model, particularly about the long-term effects of burosumab on how long people live, fracture rates and the quality of life of people with XLH and their carers. And all of the cost-effectiveness estimates are above the range normally considered an acceptable use of NHS resources. So, burosumab is not recommended.