Suggested remit - To appraise the clinical and cost effectiveness of LentiGlobin within its marketing authorisation for treating transfusion-dependant beta-thalassaemia
Status Proposed
Process STA 2018
ID number 968

Provisional Schedule

Committee meeting 07 April 2020
Expected publication 24 June 2020

Project Team

Project lead Kate Moore

Email enquiries

Evidence Review Group / Assessment Group Centre for Reviews and Dissemination and Centre for Health Economics, University of York


Companies sponsors bluebird bio (Zynteglo)
Others Department of Health and Social Care
  NHS England
  NHS North East Essex CCG
  NHS North Norfolk CCG
  Welsh Government
Patient carer groups Black Health Agency
  Genetic Alliance UK
  Muslim Council of Britain
  South Asian Health Foundation
  Specialised Healthcare Alliance
  UK Thalassaemia Society
Professional groups British Blood Transfusion Society
  British Committee for Standards in Haematology
  British Society for Haematology
  British Society of Bone Marrow Transplantation
  NHS Blood & Transplant
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal college of Surgeons
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Forum on Haemoglobin Disorders
  UK Haemophilia Centre Doctors’ Organisation
  UK National Screening Committee


Associated public health groups Public Health England
  Public Health Wales
Comparator companies Hospira UK (desferrioxamine)
  Novartis Pharmaceuticals (desferrioxamine, deferasirox)
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 Confederation
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups Cell and Gene Therapy Catapult
  Cochrane Haematological Malignancies Group
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research
  National Primary Care Research & Development Centre


Key events during the development of the guidance:

Date Update
01 August 2019 Invitation to participate
14 June 2019 - 12 July 2019 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
15 April 2019 As you will be aware the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of lentiGlobin within its anticipated marketing authorisation for treating transfusion-dependent beta-thalassaemia. The appraisal is expected to start in approximately early August 2019 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately early October 2019. These timings are based on a request from the company to reschedule the initial date set by NICE until after the anticipated marketing authorisation has been received, enabling additional evidence to be considered and to align with when the company hopes to launch the product in England and Wales.
16 May 2018 (10:00) Scoping workshop (London)
21 March 2018 - 20 April 2018 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance