Suggested remit: To appraise the clinical and cost effectiveness of pegzilarginase within its marketing authorisation as an add-on treatment for arginase-1 deficiency.
- 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:
- The topic routing was discussed at the Topic Selection Oversight Panel (TSOP) in April 2023. The panel concluded that the topic was suitable for Highly Specialised Technologies. Please see project documents for further details. The evaluation is expected to begin with an invitation to participate in September 2023, with a first committee meeting in May 2024.
- Process:
- HST Standard
- ID number:
- 4029
Provisional Schedule
- Committee meeting: 3:
- 23 October 2025
Project Team
- Project lead
- Thomas Feist
Email enquiries
If you have any queries please email [email protected]
Stakeholders
- Companies sponsors
- Immedica
- Others
- Department of Health and Social Care
- NHS England
- Patient carer groups
- Gene People
- Metabolic Support UK
- Professional groups
- Royal College of Physicians
- Comparator companies
- Eurocept International (confidentiality agreement not signed, not participating)
- Immedica (confidentiality agreement not signed, not participating)
- 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
- National Institute for Health Research
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 26 June 2025 - 17 July 2025 | Draft guidance |
| 22 May 2025 | Committee meeting: 2 |
| 06 September 2024 - 27 September 2024 | Evaluation consultation |
| 15 August 2024 | Committee meeting: 1 |
| 15 August 2024 | Declaration of interests |
| 17 January 2024 | Invitation to participate |
| 21 September 2023 | Please note that following on from a request received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin in early-January 2024. These timings are based on a request from the company to reschedule the initial date set by NICE, in order to facilitate a suitably comprehensive and robust submission. |
| 07 September 2023 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 07 September 2023 | Topic selection |
| 07 September 2023 | Topic selection |
| 07 September 2023 | In progress |
| 17 March 2023 (10:00) | Scoping workshop |
| 17 January 2023 - 14 February 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 17 January 2023 | In progress |
For further information on our processes and methods, please see our CHTE processes and methods manual