Early Access Programs (sometimes called Expanded Access, Pre-approval Access, Compassionate Use, Named Patient or Managed Access Programs) give people with life threatening or seriously debilitating conditions early access to new medicines that do not yet have a marketing authorisation but where there is a clear unmet medical need.
Early access programmes represent a unique opportunity to gather real-world evidence – the first opportunity outside of the clinical trial setting – from a cohort of patients all receiving the same treatment.
Use of Burosumab for Adults In UK (2023)
As the marketing authorisation for the use of burosumab in England, Wales, Scotland, Ireland and Northern Ireland was originally for use in children with XLH, it excluded using burosumab in adults with XLH. However Health Technology Assessments are in progress.
XLH UK and clinical experts from across UK actively maintain a good relationship with health technology assessors, including NICE. When opportunities arise, we advocate for better treatments for the adult XLH population. Our patient stories and surveys help health technology assessors better understand the lived experience of XLH and the impact it has on families, carers and friends.
England: Currently in England, some adults with XLH that met the biochemical and clinical criteria are participating in the Early Access Programme which is prescribed and managed through participating specialist centres across the country. Although the recruitment is closed, the programme is ongoing.
Scotland: In 2023, the Scottish Medicines Consortium (SMC) recommend the use of burosumab in adults. This means that both children and adults can access burosumab from the NHS in Scotland that have a confirmed diagnosis and meet their biochemical and clinical criteria. The recommendation for adults was made through the ultra-orphan pathway, which is used for medicines for very rare diseases. This pathway allows people with XLH to access burosumab while more data is collected to support its effectiveness. The SMC will review this evidence in three years to make a final decision on its routine use.