Suggested remit: To appraise the clinical and cost effectiveness of fidanacogene elaparvovec within its anticipated marketing authorisation for treating moderately severe to severe haemophilia B.
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 4032

Provisional Schedule

Committee meeting 06 June 2024
Expected publication 14 August 2024

Project Team

Project lead Kate Moore

Email enquiries

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


Companies sponsors Pfizer (fidanacogene elaparvovec)
Others Department of Health and Social Care
  NHS England
Patient carer groups Gene People
  Haemophilia Society
Professional groups Royal College of Physicians
Comparator companies CSL Behring (etranacogene dezaparvovec)
  Novo Nordisk - CAU not returned, not participating
  Pfizer (nonacog alfa)
  Swedish Orphan Biovitrum (eftrenonacog alfa)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Haemophilia Scotland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
25 October 2023 Invitation to participate
17 May 2023 - 15 June 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 4032
17 May 2023 In progress. Scoping commencing
12 August 2022 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