Suggested remit: To appraise the clinical and cost effectiveness of leriglitazone within its marketing authorisation for treating adrenoleukodystrophy.
- 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
- Rationale:
- The topic was considered by the Topic Selection Oversight Panel (TSOP) in September 2023. The panel concluded that the topic should be routed to the Technology Appraisal (TA) work programme. Please see project documents for further details. Following a request from the company, the timelines for the evaluation are to be confirmed.
- Process:
- STA Standard
- ID number:
- 3903
Provisional Schedule
- Committee meeting:
- 19 November 2026
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email RareDiseasesTeam@nice.org.uk
- External Assessment Group:
- Liverpool Reviews and Implementation Group, University of Liverpool
Stakeholders
- Companies sponsors
- Neuraxpharm UK
- Others
- Birmingham Women's and Children's Hospital NHS Foundation Trust
- Central Manchester Foundation Trust, Willink Unit, Genetic Medicine
- Department of Health and Social Care
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Guy's and St Thomas' NHS Foundation Trust
- Health Technology Wales
- Leeds Teaching Hospitals NHS Trust
- NHS England
- Salford Royal Hospital Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust
- University College London Hospital Charles Dent Metabolic Unit
- University Hospital Birmingham Foundation Trust, Department of Endocrinology
- Patient carer groups
- Addison’s Disease Self-Help Group
- Alex The Leukodystrophy Charity
- Anthony Nolan
- Beacon
- Brain and Spine Foundation
- Brain Charity
- Gene People
- Genetic Alliance UK
- Kidney Care UK
- Kidney Research UK
- Metabolic Support UK
- National Kidney Federation
- Neurological Alliance
- South Asian Health Foundation
- Specialised Healthcare Alliance
- Together For Short Lives
- Professional groups
- Association of British Neurologists
- Association of Genetic Nurses & Counsellors
- British Association of Paediatric Nephrology
- British Association of Endocrine and Thyroid Surgeons
- British Association of Urological Nurses
- British Geriatrics Society
- British Inherited Metabolic Disease Group
- British Neuropathological Society
- British Paediatric Neurology Association
- British Society for Gene and Cell Therapy
- British Society for Genetic Medicine
- British Society for Paediatric Endocrinology & Diabetes
- British Society of Blood and Marrow Transplantation
- National Metabolic Biochemistry Network
- National Neuroscience Advisory Group
- Neonatal and Paediatric Pharmacists Group
- NHS Blood and Transplant
- Primary Care and Community Neurology Society
- Royal College of General Practitioners
- Royal College of Nursing
- Royal College of Paediatrics and Child Health
- Royal College of Pathologists
- Royal College of Physicians
- Royal Society of Medicine
- Society for Endocrinology
- UK Clinical Pharmacy Association
- UK Kidney Association
- UK Renal Pharmacy Group
- Associated public health groups
- Public Health Wales
- UK Health Security Agency
- Comparator companies
- None
- General commentators
- All Wales Inherited Metabolic Disease Service
- All Wales Therapeutics and Toxicology Centre
- Allied Health Professionals Federation
- Association of Renal Industries
- 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
- National Services Division
- Neurological Alliance of Scotland
- NHS Confederation
- NHS Wales Joint Commissioning Committee
- Scottish Medicines Consortium
- Wales Neurological Alliance
- Welsh Government
- Relevant research groups
- Brain Research UK
- Cochrane Metabolic and Endocrine Disorders Group
- Cochrane UK
- Genomics England
- MRC Clinical Trials Unit
- National Hospital for Neurology and Neurosurgery
- National Institute for Health Research
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 20 May 2026 | Invitation to participate |
| 27 March 2026 - 28 April 2026 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 31 October 2023 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 31 October 2023 | Topic selection |
| 31 October 2023 | Topic selection |
| 31 October 2023 | In progress |
| 25 July 2023 - 22 August 2023 | Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators: 3903 |
| 25 July 2023 | In progress. Scoping commencing |
| 03 November 2021 (14:00) | Scoping workshop |
| 09 September 2021 - 07 October 2021 | Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators |
| 23 September 2021 | Note added to the project documents |
For further information on our processes and methods, please see our CHTE processes and methods manual