| 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 | 4015 |
Project Team
| Project lead | Celia Mayers |
Email enquiries
- If you have any queries please email TATeam6@nice.org.uk
| External Assessment Group | Centre for Reviews and Dissemination and Centre for Health Economics, University of York |
Stakeholders
| Companies sponsors | Vertex (exagamglogene autotemcel) |
| Others | Department of Health and Social Care |
| NHS England | |
| Welsh Government | |
| Patient carer groups | Anthony Nolan |
| Gene People | |
| UK Thalassaemia Society | |
| Professional groups | British Society for Haematology |
| Royal College of Physicians | |
| UK Forum on Haemoglobin Disorders | |
| Comparator companies | Accord Health care Limited (deferasirox) – confidentiality agreement not signed, not participating |
| Chiesi Ltd (deferiprone) | |
| Dr Reddy’s Laboratories (UK) Ltd (deferasirox) – confidentiality agreement not signed, not participating | |
| Glenmark Pharmaceuticals Europe Ltd (deferasirox) – confidentiality agreement not signed, not participating | |
| Hospira UK Ltd (desferrioxamine Mesilate) – confidentiality agreement not signed, not participating | |
| Lipomed GmbH (deferiprone) – confidentiality agreement not signed, not participating | |
| MSN Laboratories Europe Limited (deferasirox) – confidentiality agreement not signed, not participating | |
| Mylan (deferasirox) – confidentiality agreement not signed, not participating | |
| Novartis Pharmaceuticals UK Ltd (deferasirox, desferrioxamine Mesilate) – confidentiality agreement not signed, not participating | |
| Sandoz Limited (deferasirox) – confidentiality agreement not signed, not participating | |
| Teva UK (deferasirox) – confidentiality agreement not signed, not participating | |
| Zentiva (deferasirox) – 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 |
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 06 March 2024 | NICE has agreed to pause publication of the draft guidance to allow the company and NHS England to enter into commercial and managed access discussions. Once these discussions have concluded, NICE will provide an update outlining the next steps for this appraisal. The publication date on the website will remain TBC. |
| 14 February 2024 | Committee meeting |
| 07 December 2023 | After very careful consideration, we have taken the decision to postpone the first appraisal committee meeting for this topic. The committee discussion planned for the 14 December 2023 will therefore not be taking place. This delay is necessary to allow the additional time that is needed to ensure the committee has all the evidence needed to inform a full and robust committee discussion leading to the most optimum decision-making. The technology appraisal will be discussed at a 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. |
| 25 April 2023 | Invitation to participate |
| 19 January 2023 - 16 February 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 4015 |
| 28 January 2022 | In progress |
For further information on our processes and methods, please see our CHTE processes and methods manual