Suggested remit: To appraise the clinical and cost effectiveness of maralixibat within its marketing authorisation for treating cholestatic pruritus in Alagille Syndrome.
 
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
Process STA Standard
ID number 3941

Provisional Schedule

Expected publication 22 January 2025

Project Team

Project lead Jennifer Upton

Email enquiries

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
12 November 2024 Committee meeting
31 July 2024 - 21 August 2024 Draft guidance: 1
09 July 2024 Declaration of interests
14 June 2024 In progress. Ongoing commercial discussions between the company and NHSE for maralixibat for treating cholestatic pruritus in Alagile Syndrome has allowed the appraisal to be resumed and a committee meeting has been scheduled for 9 July 2024.
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