| 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 |
| Further information | Awaiting development |
| Process | STA Standard |
| ID number | 6145 |
Provisional Schedule
| Committee meeting | 12 June 2024 |
| Expected publication | 23 October 2024 |
Project Team
| Project lead | Celia Mayers |
Email enquiries
- If you have any queries please email HST@nice.org.uk
| External Assessment Group | Centre for Reviews and Dissemination and Centre for Health Economics, University of York |
Stakeholders
| Companies sponsors | BioMarin (cerliponase alfa) |
| Others | Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit |
| Bristol Royal Hospital for Children | |
| Department of Health and Social Care | |
| Newcastle upon Tyne Hospitals NHS Foundation Trust, Great North Children’s Hospital | |
| NHS England | |
| Patient carer groups | Batten Disease Family Association |
| Professional groups | Royal College of Physicians |
| 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 | |
| Medicines and Healthcare products Regulatory Agency | |
| Scottish Medicines Consortium | |
| Welsh Government | |
| Welsh Health Specialised Services Committee | |
| Relevant research groups | National Institute for Health Research |
| Rare Disease Research Partners |
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 31 October 2023 | Invitation to participate |
| 14 September 2023 - 12 October 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 14 September 2023 | In progress. Draft scope issued for consultation. |
| 11 August 2023 | Awaiting development |
For further information on our interim process and methods, please see our health technology evaluations: interim methods and process guide for the proportionate approach to technology appraisals