Suggested remit: To appraise the clinical and cost effectiveness of evinacumab within its marketing authorisation for treating homozygous familial hypercholesterolaemia in people aged 12 years and over.
 
Status In progress
Technology type Medicine
Decision Awaiting decision
Process STA Standard
ID number 2704

Provisional Schedule

Committee meeting 02 July 2024
Expected publication 11 September 2024

Project Team

Project lead Celia Mayers

Email enquiries

External Assessment Group BMJ Technology Assessment Group (BMJ-TAG), BMJ

Stakeholders

Companies sponsors Ultragenyx (Evinacumab)
Others Department of Health and Social Care
  NHS England
Patient carer groups Gene People
  HEART UK
Professional groups Royal College of Physicians
Assessment group National Institute for Health Research
Associated public health groups None
Comparator companies Amgen (evolocumab) – confidentiality agreement not signed, not participating
  Amryt Pharmaceuticals DAC (lomitapide) – confidentiality agreement not signed, not participating
  Aurobindo Pharma – Milpharm (ezetimibe) – confidentiality agreement not signed, not participating
  Daiichi Sankyo UK (ezetimibe) – confidentiality agreement not signed, not participating
  Glenmark Pharmaceuticals Europe (ezetimibe) – confidentiality agreement not signed, not participating
  Organon Pharma UK (ezetimibe) – confidentiality agreement not signed, not participating
  Sandoz (ezetimibe) – confidentiality agreement not signed, not participating
  Zentiva (ezetimibe) – confidentiality agreement not signed, not participating
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
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee

Timeline

Key events during the development of the guidance:

Date Update
08 April 2024 NICE previously paused the publication of final draft guidance recommending evinacumab for the treatment of homozygous familial hypercholesterolaemia in people aged 12 and older [ID2704]. NICE has now decided to withdraw the final draft guidance for evinacumab. This is because there is some uncertainty about the dose of lomitapide (the comparator for evinacumab in adults) used in the cost effectiveness analysis of evinacumab, and this raises some uncertainty about the cost effectiveness of evinacumab. Commencing on 8 April 2024, NICE will hold a 28-day stakeholder engagement in which stakeholders are requested to provide information on the dose and efficacy of lomitapide in clinical practice. NICE will then hold a committee meeting to consider the dose of lomitapide and its impact on the cost effectiveness of evinacumab.
29 January 2024 NICE has received a non-appeal request for change on the final draft guidance (FDG) for this appraisal. While the appraisal team consider the request, publication of the final guidance is paused. Final guidance will not publish as planned on 7 February 2024.
14 November 2023 Committee meeting
17 March 2023 Invitation to participate
01 March 2023 Topic selection
11 January 2023 - 08 February 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 2704
11 January 2023 In progress. Scoping commencing
14 December 2020 The Department for Health and Social Care has asked NICE to carry out a Single Technology Appraisal of evinacumab for treating homozygous familial hypercholesterolaemia in people aged 12 and over. Please note that following on from advice received from the company, further information regarding the timelines for this appraisal will be available in due course. In the meantime, NICE will continue to monitor the situation and will provide an update as and when the situation changes.
14 September 2020 (14:00) Scoping workshop
16 July 2020 - 13 August 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

For further information on our processes and methods, please see our CHTE processes and methods manual