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

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
  Welsh Government
Patient carer groups UK Thalassaemia Society
Professional groups British Society for Haematology
  Royal College of Pathologists
  Royal College of Physicians


Comparator companies Novartis Pharmaceuticals (desferrioxamine, deferasirox)
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
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups Cell and Gene Therapy Catapult


Key events during the development of the guidance:

Date Update
06 April 2020 Topic update: this appraisal has not been defined as therapeutically critical (please follow the link titled rapid guidelines and evidence summaries within the yellow banner at the top of the page for information on recent changes to the way we work). The appraisal will therefore be paused. At this stage, we are unable to provide alternative timelines as to when the appraisal may be re-started but we will provide updates as and when this information becomes clear.
01 August 2019 Invitation to participate
01 August 2019 In progress. 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