Status In progress
Process HST
ID number 1643

Provisional Schedule

Committee meeting: 1 06 August 2020
Expected publication 11 November 2020

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors BioMarin Pharmaceuticals (elosulfase alfa)
Others Addenbrooke's Lysosomal Disorders Unit
  Birmingham Children’s Hospital NHS Foundation Trust
  Charles Dent Metabolic Unit, UCLH
  Department of Endocrinology, Queen Elizabeth Hospital, UHBFT
  Department of Health and Social Care
  Great Ormond Street Hospital
  Inherited Metabolic and Lysosomal Disease Service, Cardiff and Vale UHB
  Manchester Foundation NHS Trust
  Mark Holland Metabolic Unit for Adult Inherited Metabolic Disorders, SRFT
  Metabolic Medicine Unit, GOSH
  NHS England
  Royal Free Lysosomal Storage Disorders Unit
  Salford Royal NHS Foundation Trust
  University College London Hospital NHS Foundation Trust
  University Hospital Birmingham NHS Foundation Trust
  Willink Unit, Royal Manchester Children’s Hospital, CMFT
Patient carer groups Carol Ann Foundation/The International Morquio Organisation
  Contact a Family
  Genetic Alliance UK
  Metabolic Support UK
  MPS Society
  Muslim Council of Britain
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association of Genetic Nurses and Counsellors
  Association of Surgeons of Great Britain
  and Ireland
  British Inherited Metabolic Disease Group
  British Society for Genetic Medicine
  National Metabolic Biochemistry Network
  Royal College of Anaesthetists
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics and Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal College of Surgeons
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Genetic Testing Network
  United Kingdom Clinical Pharmacy Association


Associated public health groups Public Health England
  Public Health Wales
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Hospital Information Services – Jehovah’s Witnesses
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  National Services Division (specialised services in Scotland)
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
  Welsh Government
Relevant research groups Cochrane Cystic Fibrosis and Genetic Disorders Group
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research
  Society for the Study of Inborn Errors of Metabolism


Key events during the development of the guidance:

Date Update
13 December 2019 Invitation to participate
06 November 2019 - 27 November 2019 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators
31 October 2019 In progress, Referred

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