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:
- HST Standard
- ID number:
- 3903
Provisional Schedule
- Committee meeting:
- 19 November 2026
- Expected publication:
- 11 April 2027
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
- Department of Health and Social Care
- Health Technology Wales
- NHS England
- Patient carer groups
- Alex The Leukodystrophy Charity
- Metabolic Support UK
- Professional groups
- Association of British Neurologists
- British Inherited Metabolic Disease Group
- Royal College of Physicians
- Associated public health groups
- None
- 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
- Relevant research groups
- None
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 10 June 2026 | Awaiting development. Project type change |
| 10 June 2026 | In progress. Project type change |
| 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