Latest news regarding NICE’s Topic Selection of using burosumab in XLH adults (September 2022)


You may already be aware that XLH UK have been working hard to represent patient views to NICE since March 2021. We have also liaised closely with select expert clinicians, so that NICE has reliable information with which to make the most appropriate decision regarding through which pathway burosumab should be assessed when treating adults with XLH in England.


NICE’s Topic Selection committee has decided that NICE should assess the effectiveness of using burosumab in XLH adults through the ‘Single Technology Appraisal’ (STA) process.

This itself presents a new set of challenges given STA committees are typically used to assessing treatments for disorders that affect large populations and treatments for other, more common diseases such as cancer, and therefore may be less familiar with lifelong multi-system disorders such as XLH.

The STA process is expected to commence by March 2023, at which point NICE will seek in-depth evidence from industry, expert clinicians, XLH UK and Metabolic Support UK.

We have been assured by the Pharmaceutical company Kyowa Kirin that those adults on the Early Access Programme in England will continue to receive treatment at this time.

XLH UK will also be involved in supporting the ‘One Wales Medicines’ process and Scotland’s SMC in the coming months.

How to stay updated

In the coming weeks, we will be sharing more information, including how we are going to collect evidence for this evaluation. However, should you wish to see the latest information from NICE, please see