Experience with burosumab in adults with XLH in the United Kingdom.
Research type
Research Study
Full title
Early access experience with burosumab in adults with X-Linked Hypophosphataemia (XLH) in the United Kingdom
IRAS ID
321609
Contact name
Judith Bubbear
Contact email
Sponsor organisation
Kyowa Kirin Ltd
Clinicaltrials.gov Identifier
54855, CPMS ID
Duration of Study in the UK
0 years, 6 months, days
Research summary
X-linked hypophosphataemia (XLH) is a hereditary disorder characterised by low phosphate levels in the blood (hypophosphataemia). Phosphate is essential to build bones and teeth and maintain their strength, so patients may develop rickets and other bone deformities and growth problems.
Until recently, standard treatment for XLH was oral phosphate and vitamin D. Burosumab was the first drug approved for the treatment of adults with XLH and a real breakthrough for those living with this serious disease. Prior to its approval, Kyowa Kirin made burosumab available free of charge to UK patients, on request, in an Early Access Program (EAP).
The primary goal of an EAP is to provide access to treatment where there is a recognised unmet need. However, as an early opportunity to collect data and generate real-world evidence, EAPs are increasingly valuable, as recognised by regulators, particularly in rare diseases.
In this project, analysis of medical record data collected from patients during the burosumab EAP (at 5 UK NHS hospitals) is proposed. This could provide early insights into the effectiveness, patient reported outcomes and quality of life during treatment with burosumab in NHS clinical practice. These Real World insights would compliment randomised controlled trial evidence and be of interest to the medical community caring for XLH patients.
The aim is for historic data from all the EAP patients to be included, from enrolment until 31 December 2022. Medical record data should be enough for analysis without requiring any study visits for patients. As an observational project, using only historic data, no change will be made to patient care as part of this research.
Through improving the understanding of effectiveness and real XLH patients' experience during burosumab treatment, this project will support improved patient care in future.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
23/EM/0078
Date of REC Opinion
9 May 2023
REC opinion
Further Information Favourable Opinion