Status In progress
Process HST
ID number 1151

Provisional Schedule

Expected publication 24 October 2018

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors Kyowa Kirin
Others Birmingham Children's Hospital NHS Foundation Trust
  Department of Health and Social Care
  NHS England
  Royal Manchester Children’s Hospital
Patient carer groups Children Living with Inherited Metabolic Diseases (CLIMB)
Professional groups British Paediatric and Adolescent Bone Group
  Royal College of Physicians


Comparator companies None
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
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups None


Key events during the development of the guidance:

Date Update
05 September 2018 - 19 September 2018 Final evaluation determination
25 July 2018 Committee meeting: 2
15 June 2018 - 06 July 2018 Evaluation consultation
23 May 2018 Committee meeting: 1
05 December 2017 Invitation to participate
05 December 2017 In progress, In progress

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance