We are suspending this appraisal to give the Company more time to discuss and agree on a commercial arrangement with NHS England. The incremental cost-effectiveness ratios are above what NICE usually considers acceptable for standard technology appraisals. We are suspending the appraisal while we consider the next steps. We will use the termination process as outlined in section 5.6.8 – 5.6.13 of NICE health technology evaluations: the manual to bring this appraisal to a close if a commercial arrangement is not in place.
| Status | Suspended |
| 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 | STA Standard |
| ID number | 3941 |
Project Team
| Project lead | Thomas Feist |
Email enquiries
- If you have any queries please email TATeam1@nice.org.uk
| External Assessment Group | Warwick Evidence, Warwick Medical School, University of Warwick |
Stakeholders
| Companies sponsors | Mirum Pharma |
| Others | Department of Health and Social Care |
| NHS England | |
| Patient carer groups | Children's Liver Disease Foundation |
| Gene People | |
| Professional groups | Royal College of Physicians |
| General commentators | All Wales Therapeutics and Toxicology Centre |
| British National Formulary (BNF) | |
| Department of Health - Northern Ireland | |
| Healthcare Improvement Scotland | |
| Medicines and Healthcare products Regulatory Agency | |
| Scottish Medicines Consortium | |
| Welsh Government | |
| Welsh Health Specialised Services Committee |
Timeline
Key events during the development of the guidance:
| Date | Update |
|---|---|
| 15 March 2024 | Suspended. We are suspending this appraisal to give the Company more time to discuss and agree on a commercial arrangement with NHS England. The incremental cost-effectiveness ratios are above what NICE usually considers acceptable for standard technology appraisals. We are suspending the appraisal while we consider the next steps. We will use the termination process as outlined in section 5.6.8 – 5.6.13 of NICE health technology evaluations: the manual to bring this appraisal to a close if a commercial arrangement is not in place. |
| 11 September 2023 | Invitation to participate |
| 08 September 2023 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 08 September 2023 | Topic selection |
| 08 September 2023 | In progress |
| 08 September 2023 | Following a challenge from the company, the routing was re-considered by the Topic Selection Oversight Panel (TSOP) in July 2023. The panel concluded that the topic should remain routed to the Technology Appraisal (TA) work programme. Please see project documents for further details. |
| 05 May 2023 (10:00) | Scoping workshop |
| 15 March 2023 - 14 April 2023 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 15 March 2023 | In progress. Rescoping commenced |
| 19 December 2022 | Topic selection |
| 04 November 2022 | The topic was re-considered by the Topic Selection Oversight Panel (TSOP) in September 2022. The panel concluded that the topic should remain routed to the Technology Appraisal (TA) work programme. The timelines for the evaluation are to be confirmed. |
| 08 April 2022 | Following a routing challenge raised by the company, regarding the Topic Selection Oversight Panel’s decision to route maralixibat as a Single Technology Appraisal instead of as a Highly Specialised Technology, the start of this appraisal is therefore delayed. The timelines for this appraisal will be confirmed once NICE has considered this challenge. |
| 14 March 2022 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
| 14 March 2022 | Topic selection |
| 15 December 2021 (10:00) | Scoping workshop |
| 03 November 2021 - 01 December 2021 | Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators |
| 03 November 2021 | In progress. Scoping commenced |
| 01 March 2021 | Awaiting development. Status change linked to Topic Selection Decision being set to Selected |
For further information on our processes and methods, please see our CHTE processes and methods manual