Status In progress
Process HST
ID number 926

Provisional Schedule

Committee meeting: 2 23 November 2017
Expected publication 28 February 2018

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors GlaxoSmithKline
Others Department of Health
  Great Ormond Street Hospital – Immunology Department
  NHS England
  Welsh Government
Patient carer groups Genetic Alliance UK
  Primary Immunodeficiency UK
Professional groups Royal College of Paediatrics & Child Health
  Royal College of Physicians


Comparator companies Orchard Therapeutics (ex-vivo autologous gene therapy)
  Sigma-Tau Pharmaceuticals (pegylated adenosine deaminase enzyme) CA&U not returned, 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
  Welsh Health Specialised Services Committee
Relevant research groups Cell and Gene Therapy Catapult


Key events during the development of the guidance:

Date Update
23 October 2017 - 13 November 2017 Evaluation consultation: 1
28 September 2017 Committee meeting: 1
08 May 2017 Invitation to participate
14 July 2016 Draft scope documents

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