Suggested remit: To appraise the clinical and cost effectiveness of vanzacaftor–tezacaftor–deutivacaftor within its marketing authorisation for treating cystic fibrosis with 1 or more F508del mutations in the CFTR gene in people 6 years and over.
 
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 Cost Comparison Standard
ID number 6372

Provisional Schedule

Expected publication 21 August 2025

Project Team

Project lead Leena Issa

Email enquiries

External Assessment Group Kleijnen Systematic Reviews Ltd

Stakeholders

Companies sponsors Vertex Pharmaceuticals
Others Department of Health and Social Care
  NHS England
Patient carer groups Cystic Fibrosis Trust
Professional groups Association of Chartered Physiotherapists in Cystic Fibrosis
  British Dietetic Association
  British Paediatric Respiratory Society
  British Thoracic Society
  Cystic Fibrosis Nursing Association
  Neonatal and Paediatric Pharmacists Group (NPPG)
  Royal College of Physicians
  UK Clinical Pharmacy Association
  UK Cystic Fibrosis Medical Association
  UK Cystic Fibrosis Pharmacy Group
  UK Psychosocial Professionals in Cystic Fibrosis Group
Associated public health groups None
Comparator companies Vertex Pharmaceuticals (elexacaftor, lumcaftor and ivacaftor)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health - Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  NHS Wales Joint Commissioning Committee
  Scottish Medicines Consortium
  Welsh Government
Relevant research groups Cochrane Cystic Fibrosis and Genetic Disorders Group

Timeline

Key events during the development of the guidance:

Date Update
23 January 2025 Invitation to participate
17 September 2024 - 15 October 2024 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 6372
17 September 2024 In progress. Scoping commencing
17 September 2024 In progress. Scoping commencing
26 July 2024 Awaiting development. Status change linked to Topic Selection Decision being set to Selected
12 October 2023 Topic selection

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