Suggested remit: To appraise the clinical and cost effectiveness of etranacogene dezaparvovec within its marketing authorisation for treating moderately severe or severe haemophilia B.
 
Status In progress
Technology type Medicine
Decision Selected
Process TA
ID number 3812

Project Team

Project lead Kate Moore

Email enquiries

External Assessment Group Peninsula Technology Assessment Group (PenTAG), University of Exeter

Stakeholders

Companies sponsors CSL Behring (etranacogene dezaparvovec)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Haemophilia Society
Professional groups Royal College of Physicians
  UK Haemophilia Centre Doctors’ Organisation
Comparator companies Novo Nordisk (eptacog alpha, nonacog beta pegol)
  Pfizer (nonacog alfa)
  Swedish Orphan Biovitrum (eftrenonacog alfa)
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 Health Specialised Services Committee
Relevant research groups National Institute for Health Research

Timeline

Key events during the development of the guidance:

Date Update
13 September 2023 Committee meeting: 2
02 August 2023 - 23 August 2023 Draft guidance
12 July 2023 Committee meeting
01 November 2022 Invitation to participate
01 November 2022 In progress. invitation to participate
16 September 2022 As you will be aware, the Department for Health & Social Care has asked NICE to conduct an appraisal of etranacogene dezaparvovec within its marketing authorisation for treating moderately severe or severe haemophilia B. Please note that following on from advice received from the company this appraisal has been rescheduled to align with latest regulatory expectations. Therefore, we now anticipate that the appraisal will begin during early-November 2022 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately mid-January 2023.
29 March 2022 (14:00) Scoping workshop
24 January 2022 - 21 February 2022 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