Status Proposed
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
  Evelina London Children's Hospital
  Great Ormond Street Hospital
  Guy’s and St Thomas’ NHS Foundation Trust
  NHS England
  Oxford University Hospitals Foundation Trust
  Royal Manchester Children’s Hospital
  Royal Orthopaedic Hospital NHS Trust
  University Dental Hospital of Manchester
Patient carer groups Action for Sick Children
  Children Living with Inherited Metabolic Diseases (CLIMB)
  Contact a Family
  Genetic Alliance UK
  Muslim Council of Britain
  National Children’s Bureau
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association of Genetic Nurses and Counsellors
  British Paediatric and Adolescent Bone Group
  British Society for Genetic Medicine
  British Society for Paediatric Endocrinology and Diabetes
  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 Pharmaceutical Society
  Royal Society of Medicine
  Society for Endocrinology
  UK Clinical Pharmacy Association
  UK Genetic Testing Network


Associated public health groups Public Health England
  Public Health Wales
Comparator companies None
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
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  NHS Alliance
  NHS Commercial Medicines Unit
  NHS Confederation
  NHS National Services Scotland
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Cystic Fibrosis and Genetic Disorders Group
  MRC Clinical Trials Unit
  National Institute for Health Research
  NIHR Musculoskeletal Biomedical Research Unit, University of Oxford (Nuffield Orthopaedic Centre)
  NIHR BioResource for Translational Research in Common and Rare Diseases


Key events during the development of the guidance:

Date Update
05 December 2017 Invitation to participate

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