Following on from information provided to NICE by the company in August 2021, the appraisal of Betibeglogene autotemcel for treating transfusion-dependent beta-thalassaemia [ID968] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
| Status | Discontinued |
| Technology type | Medicine |
| Decision | Selected |
| Process | TA |
| ID number | 968 |
Email enquiries
- If you have any queries please email scheduling@nice.org.uk
Stakeholders
| Companies sponsors | bluebird bio (betibeglogene autotemcel) |
| Others | Department of Health and Social Care |
| NHS England | |
| Welsh Government | |
| Patient carer groups | Sickle Cell Society |
| UK Thalassaemia Society | |
| Professional groups | British Society for Haematology |
| Royal College of Pathologists | |
| Royal College of Physicians | |
| Comparator companies | Chiesi (deferiprone) |
| 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 |
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 16 December 2022 | Discontinued. Following on from information provided to NICE by the company in August 2021, the appraisal of Betibeglogene autotemcel for treating transfusion-dependent beta-thalassaemia [ID968] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued. |
| 02 December 2021 | As you will be aware, the Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of Betibeglogene autotemcel for treating transfusion-dependent beta-thalassaemia [ID968] For information, the company have advised it is withdrawing its marketing application from the Medicines and Healthcare products Regulatory Agency (MHRA) and does not intend to commercialize the therapy in the UK. Therefore, in collaboration with the company, NICE has suspended this evaluation from its current work programme. As this appraisal has been referred NICE will continue to monitor any development and will update interested parties if the situation changes. |
| 18 August 2021 | This appraisal has been suspended while Bluebird Bio explores how to give patients in Europe access to its gene therapies in light of its forthcoming operational changes. A further update will be provided by late autumn. |
| 18 August 2021 | Suspended. This appraisal has been suspended while Bluebird Bio explores how to give patients in Europe access to its gene therapies in light of its forthcoming operational changes. A further update will be provided by late autumn. |
| 11 August 2021 | Following the company’s recent announcement to withdraw its operations from Europe, next steps for this appraisal are to be confirmed. |
| 14 July 2021 | Committee meeting: 2 |
| 11 February 2021 - 04 March 2021 | Draft guidance |
| 13 January 2021 | Committee meeting: 1 |
| 03 September 2020 | In progress. In Appraisal |
| 05 May 2020 | Schedule affected by COVID-19. Topic update: this appraisal has not been defined as therapeutically critical. 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 an update when this information is available. |
| 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 our processes and methods, please see our CHTE processes and methods manual