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 6342

Provisional Schedule

Committee meeting 05 December 2024

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Warwick Evidence, Warwick Medical School, University of Warwick


Companies sponsors Pfizer (marstacimab)
Others Department of Health and Social Care
  NHS England
Patient carer groups Beacon
  Black Health Agency for Equality
  Gene People
  Genetic Alliance UK
  Haemophilia Alliance
  South Asian Health Foundation
  Specialised Healthcare Alliance
  The Haemophilia Society
Professional groups Association of Genetic Nurses & Counsellors
  British Blood Transfusion Society
  British Committee for Standards in Haematology
  British Geriatrics Society
  British Paediatric Allergy, Immunity and Infection Group
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Haematology
  British Society for Immunology
  Haemophilia Chartered Physiotherapists Association
  Haemophilia Nurses Association
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  UK Clinical Pharmacy Association
  UK Forum on Haemoglobin Disorders
  UK Haemophilia Centre Doctors’ Organisation (UKHCDO)
Associated public health groups Public Health Wales
  UK Health Security Agency
Comparator companies Bio Products Laboratory (factor IX, factor VIII)
  CSL Behring (etranacogene dezaparvovec, factor IX, factor VIII)
  Grifols UK (factor IX, factor VIII)
  Novo Nordisk Ltd (factor VIII, eptacog alfa, nonacog beta pegol, turoctocog alfa pegol)
  Octapharma (factor VIII, simoctocog alfa)
  Pfizer (fidanacogene elaparvovec, moroctocog alfa, nonacog alfa)
  Roche products (emicizumab)
  Swedish Orphan Biovitrum (efanesoctocog alfa, efmoroctocog alfa, eftrenonacog alfa)
  Takeda (factor IX, factor VIII, octocog alfa, rurioctocog alfa pegol, susoctocog alfa)
General commentators All Wales Therapeutics and Toxicology Centre
  Allied Health Professionals Federation
  Board of Community Health Councils in Wales
  British National Formulary
  Care Quality Commission
  Cell and Gene Therapy Catapult
  Department of Health, Social Services and Public Safety for Northern Ireland
  Haemophilia Northern Ireland
  Haemophilia Scotland
  Haemophilia Wales
  Healthcare Improvement Scotland
  Hospital Information Services - Jehovah's Witnesses
  Medicines and Healthcare Products Regulatory
  National Association of Primary Care
  National Pharmacy Association
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups Cochrane Cystic Fibrosis & Genetic Disorders Group
  Cochrane Haematology
  Cochrane UK
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research
  NHS Oxford Haemophilia and Thrombosis Centre
  NHS Southern Haemophilia Network


Key events during the development of the guidance:

Date Update
16 May 2024 Invitation to participate
07 March 2024 - 08 April 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6342
07 March 2024 In progress. Scoping commenced.
30 August 2023 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