Suggested remit: To appraise the clinical and cost effectiveness of CTX001 within its marketing authorisation for treating sickle cell disease.
Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Further information DHSC referral received
Process STA Standard
ID number 4016

Provisional Schedule

Expected publication 17 July 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group Warwick Evidence, Warwick Medical School, University of Warwick


Companies sponsors Vertex (Exagamglogene autotemcel)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Anthony Nolan
  Gene People
  Sickle Cell Society
Professional groups British Society for Haematology
  Royal College of Pathologists
  Royal College of Physicians
  UK Forum on Haemoglobin Disorders
Comparator companies Masters Specialty Pharma (hydroxycarbamide) – confidentiality agreement not signed, not participating
  Medac GmbH (hydroxycarbamide) - confidentiality agreement not signed, not participating
  Neon Laboratories (hydroxycarbamide) - confidentiality agreement not signed, not participating
  Nova Laboratories (hydroxycarbamide) - confidentiality agreement not signed, not participating
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
08 May 2024 Committee meeting: 2
14 March 2024 - 11 April 2024 Draft guidance
14 February 2024 Committee meeting
12 December 2023 We have taken the decision to move the committee discussion for this appraisal to the meeting of the Highly Specialised Technologies Evaluation Committee on 14 February 2024. Please note that this topic will still be considered as a Single Technology Appraisal. This decision was taken to allow both this appraisal and the appraisal of exagamglogene autotemcel for treating transfusion-dependent beta-thalassaemia [ID4015] – the same therapy in a different indication – to be considered by the same committee.
08 June 2023 Invitation to participate
18 January 2023 - 15 February 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 4016
28 January 2022 In progress

For further information on our processes and methods, please see our CHTE processes and methods manual