Suggested remit: To appraise the clinical and cost effectiveness of burosumab within its marketing authorisation for treating X-linked hypophosphatemia (XLH) in adults
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Process TA
ID number 3822

Provisional Schedule

Expected publication 07 August 2024

Project Team

Project lead Vonda Murray

Email enquiries


Companies sponsors Kyowa Kirin (burosumab)
Others Health Technology Wales
  Department of Health and Social Care
  NHS England
Patient carer groups Metabolic Support UK
Professional groups Royal College of Physicians
Assessment group Centre for Reviews and Dissemination and Centre for Health Economics – York
Associated public health groups None
Comparator companies A H Pharmaceuticals (alphacalcitriol) (confidentiality agreement not signed, not participating)
  Atnahs Pharma UK (calcitriol) (confidentiality agreement not signed, not participating)
  Sigma Pharmaceuticals (alphacalitriol) (confidentiality agreement not signed, not participating)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
  Welsh Government
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
21 June 2024 - 05 July 2024 Final draft guidance
15 February 2024 Declaration of interests
29 November 2023 - 03 January 2024 Draft guidance
08 November 2023 Committee meeting
23 February 2023 Invitation to participate
18 January 2023 Please note that following on from a request received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin in early-February 2023. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission.
11 July 2022 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
11 July 2022 Topic selection. Change decision to selected
08 July 2022 Topic selection
07 January 2022 In progress. Scoping commenced
09 December 2021 The topic was discussed at the Topic Selection Oversight Panel (TSOP) in October 2021. The panel concluded that the topic was suitable for a Technology Appraisal. Following conversations with the company, the scheduling of the evaluation is to be confirmed and stakeholders will be updated in due course.
04 March 2021 (10:00) Scoping workshop
07 January 2021 - 04 February 2021 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

For further information on our processes and methods, please see our CHTE processes and methods manual