Suggested remit: To appraise the clinical and cost effectiveness of efanesoctocog alfa within its marketing authorisation for treating and preventing bleeding episodes in people of any age with previously treated Haemophilia A.
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 6170

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Kleijnen Systematic Reviews Ltd


Companies sponsors Swedish Orphan Biovitrum
  (efanesoctocog alfa)
Others Department of Health and Social Care
  NHS England
Patient carer groups Gene People
Professional groups Royal College of Pathologists
  Royal College of Physicians
  UK Haemophilia Centre Doctors’ Organisation
Comparator companies Bio Products Laboratory (factor VIII) (confidentiality agreement not signed, not participating)
  Biotest (factor VIII) (confidentiality agreement not signed, not participating)
  CSL Behring UK (factor VIII) (confidentiality agreement signed, participating)
  Novo Nordisk (factor VIII, turoctocog alfa pegol) (confidentiality agreement signed, participating)
  Octopharma (factor VIII, simoctocog alfa) (confidentiality agreement signed, participating)
  Pfizer (moroctocog alfa) (confidentiality agreement signed, participating)
  Roche (emicizumab) (confidentiality agreement signed, participating)
  Takeda UK (octocog alfa, rurioctocog alfa pegol, susoctocog alfa) (confidentiality agreement signed, participating)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  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
17 May 2024 - 10 June 2024 Draft guidance
04 April 2024 Committee meeting
15 March 2024 Efanesoctocog alfa has not yet received regulatory approval, so the committee meeting on 4 April 2024 will be held in private.
31 August 2023 Invitation to participate
27 July 2023 Please note that following on from information received from the company, the timelines for this appraisal have been revised and the appraisal is now anticipated to begin during mid-August 2023, when we will write to you about how you can get involved.
22 May 2023 - 20 June 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6170
13 October 2022 In progress. DHSC referral received
11 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