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
Provisional Schedule
- Expected publication:
- 05 June 2026
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email [email protected]
Stakeholders
- Companies sponsors
- UCB Pharma
- Others
- Department of Health and Social Care
- Department of Paediatric Neurology, Manchester University NHS Foundation Trust
- Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust
- NHS England
- NHS Rare Mitochondrial Disorders Service (Newcastle Mitochondrial Service, University College London Hospital NHS Highly Specialised Service for Rare Mitochondrial Disorders & Oxford University Hospitals Rare Mitochondrial Disorders Service)
- The Charles Dent Metabolic Unit, University College London Hospital NHS Foundation Trust
- The Mark Holland Metabolic Unit, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
- Willink Biochemical Genetics Unit, Manchester University NHS Foundation Trust
- Patient carer groups
- Beacon
- Gene People
- Genetic Alliance UK
- Metabolic Support UK
- Muscular Dystrophy UK
- South Asian Health Foundation
- Specialised Healthcare Alliance
- The Lily Foundation
- Professional groups
- Association of British Neurologists
- Association of Genetic Nurses & Counsellors
- Association of Paediatric Chartered Physiotherapists
- British Inherited Metabolic Disorder Group
- British Myology Society
- British Paediatric Neurology Association
- British Society for Gene and Cell Therapy
- British Society for Genetic Medicine
- Neonatal and Paediatric Pharmacists Group
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Paediatrics & Child Health
- Royal College of Pathologists
- Royal College of Physicians
- Royal Pharmaceutical Society
- Royal Society of Medicine
- The Chartered Society of Physiotherapy
- UK Clinical Pharmacy Association
- United Kingdom National Screening Committee
- Associated public health groups
- Public Health Wales
- UK Health Security Agency
- Comparator companies
- None
- General commentators
- All Wales Inherited Metabolic Disease Service Cardiff
- All Wales Therapeutics and Toxicology Centre
- Allied Health Professionals Federation
- Board of Community Health Councils in Wales
- British National Formulary
- Care Quality Commission
- Cell and Gene Therapy Catapult
- Department of Health - Northern Ireland
- Healthcare Improvement Scotland
- Medicines and Healthcare products Regulatory Agency
- National Association of Primary Care
- National Pharmacy Association
- NHS Confederation
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Welsh Government
- Relevant research groups
- Cochrane Cystic Fibrosis & Genetic Disorders Group
- Genomics England
- MRC Clinical Trials Unit
- MRC Mitochondrial Biology Unit
- National Institute for Health Research
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 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 [email protected]. |
| 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