Finalist
XLH Matters: a Rare Disease Community for XLH
Summary of work
X-linked hypophosphataemia (XLH) is a rare, life-long progressive disease. There are few XLH specialists and limited opportunities to connect and network with colleagues. XLH Matters was launched in EMEA to create a community of XLH experts to address educational needs in XLH, attract more specialists in XLH and share best practice to improve management and patient outcomes. Working with co-chairs to deliver impactful annual meetings, an on-demand platform and practical materials have reportedly improved confidence in diagnosing, treating and MDT collaboration to manage XLH. Engagement in the programme has surpassed expectations – 118 joined the community in 2021 and 213 in 2021 – over 80% increase (vs 50% target) with representation from six specialities and 18 countries. Three months following the launch of the on-demand platform there were 506 visits (153% above target). Surveys and affiliate feedback reported real-world changes in clinical practice as a direct result of the programme. Following a case discussion with the faculty, an attendee called their hospital during the 2022 meeting to stop an unnecessary surgery. Additionally, 12 HCPs have requested access to treatment for their patients, five HCPs now hold regular MDT meetings and four HCPs have taken practical steps to support improved transition.
Judges’ comments
The entry from Sciterion and Kyowa Kirin International was clear and well written. The depth of insights were laudable and it was comprehensive and well implemented. The judges could see the clear positivity of the messages and the real uptake of the programme. The benchmarking was brilliant and it was powerful to see the evidence of efficacy across the different countries.
