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 (deutivacaftor, tezacaftor, vanzacaftor)
Others Department of Health and Social Care
  NHS England
Patient carer groups Asthma and Lung UK
  CF Voices
  Cystic Fibrosis Care
  Cystic Fibrosis Trust
  Gene People
  Genetic Alliance
  NARA – The Breathing Charity
  South Asian Health Foundation
  Specialised Healthcare Alliance
Professional groups Association for Respiratory Technology and Physiology
  Association of Chartered Physiotherapists in Cystic Fibrosis
  Association of Genetic Nurses and Counsellors
  Association of Respiratory Nurse Specialists
  British Dietetic Association
  British Geriatrics Society
  British Inherited Metabolic Disease Group
  British Paediatric Respiratory Society
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Medical Genetics
  British Thoracic Society
  Chartered Society of Physiotherapy
  Cystic Fibrosis Nursing Association
  ENT UK
  Interstitial Lung Diseases Interdisciplinary Network (ILD-IN)
  National Heart and Lung Institute
  Neonatal and Paediatric Pharmacists Group (NPPG)
  Primary Care Respiratory Society UK
  Royal College of General Practitioners
  Royal College of Nursing
  Royal College of Paediatrics & Child Health
  Royal College of Pathologists
  Royal College of Physicians
  Royal Pharmaceutical Society
  Royal Society of Medicine
  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 Public Health Wales
  UK Health Security Agency
Comparator companies Vertex Pharmaceuticals (elexacaftor, lumcaftor and ivacaftor)
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
  Department of Health - Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare products Regulatory Agency
  National Association of Primary Care
  National Pharmacy Association
  NHS Confederation
  NHS Wales Joint Commissioning Committee
  Scottish Medicines Consortium
  Welsh Government
Relevant research groups British Association for Lung Research
  CF Health Hub
  Cochrane Airways Group
  Cochrane Cystic Fibrosis and Genetic Disorders Group
  Genomics England
  MRC Clinical Trials Unit
  National Institute for Health Research
  UK Cystic Fibrosis Gene Therapy Consortium

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