Suggested remit: To appraise the clinical and cost effectiveness of doxecitine–doxribtimine within its marketing authorisation for treating thymidine kinase 2 deficiency in people of any age.
- 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
- Process:
- HST Standard
- ID number:
- 6484
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email HST@nice.org.uk
Stakeholders
- Companies sponsors
- UCB Pharma
- Others
- Department of Health and Social Care
- NHS England
- The Mark Holland Metabolic Unit, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
- Patient carer groups
- Metabolic Support UK
- Muscular Dystrophy UK
- The Lily Foundation
- Professional groups
- Royal College of Physicians
- Comparator companies
- None
- General commentators
- All Wales Therapeutics and Toxicology Centre
- British National Formulary
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Welsh Government
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 24 November 2025 | Following a request by the company, UCB Pharma, the evaluation has been paused. The committee meeting planned for 19 March 2026 will not go ahead. NICE will continue discussions with the company and will provide an update in Q1 2026 regarding new timelines. |
| 14 August 2025 | In progress. in progress |
| 14 August 2025 | Invitation to participate |
| 04 August 2025 | Awaiting development. Following consideration by NICE's Prioritisation Board, the evaluation for doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age [ID6484] has been routed to the Highly Specialised Technologies (HST) evaluation programme. Please note that the HST checklist will publish on the NICE website when the Invitation to Participate (ITP) is issued for the evaluation. Additionally, the timelines for the evaluation have been revised slightly. The evaluation will now begin in mid-August 2025 when we will write to you about how you can get involved. Submissions will be due in mid-October 2025. The first committee meeting will be held on 19 March 2026. For any queries relating to the evaluation, please contact HST@nice.org.uk. |
| 15 April 2025 - 16 May 2025 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6484 |
| 16 April 2025 | Referral |
| 15 April 2025 | In progress. Scoping commencing |
| 12 December 2024 | Referral |
| 08 November 2024 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 19 August 2024 | Topic selection |
For further information on our processes and methods, please see our CHTE processes and methods manual