Status In progress
Process HST
ID number 737
Referral date 01 June 2015

Provisional Schedule

1st committee meeting:

20 January 2016

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors Alexion Pharma UK
Others Department of Health
  NHS England
Patient carer groups British Liver Trust
  Children’s Liver Disease Foundation
  Children Living with Inherited Metabolic Diseases
  MPS Society
Professional groups Addenbrooke's Lysosomal Disorders Unit
  Birmingham Children’s Hospital NHS Foundation Trust
  British Inherited Metabolic Disease Group
  London Guy’s Hospital Genetic Centre
  European Lysosomal Storage Disorder Nurses Group
  Mark Holland Metabolic Unit for Adult Inherited Metabolic Disorders, SRFT
  Royal College of Nursing
  Royal College of Pathologists
  Royal College of Physicians
  Willink Unit, Genetic Medicine, CMFT


Evidence review group Kleijnen Systematic Reviews Ltd
  National Institute for Health Research Health Technology Assessment
General commentators Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
Relevant research groups Cochrane Cystic Fibrosis and Genetic Disorders Group


Key events during the development of the guidance:

Date Update
17 August 2017 Committee meeting: 5
18 July 2017 Appeal decision
25 April 2017 Appeal
08 February 2017 - 01 March 2017 Final evaluation determination
20 July 2016 Committee meeting: 3
26 April 2016 - 25 May 2016 Evaluation consultation: 2
22 March 2016 Committee meeting: 2
11 February 2016 - 10 March 2016 Evaluation consultation: 1

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