Suggested remit: To appraise the clinical and cost effectiveness of elexacaftor in combination with tezacaftor and ivacaftor within its marketing authorisation for treating cystic fibrosis in people with at least one F508del mutation.
Status In progress
Process STA 2018
ID number 1661

Provisional Schedule

Committee meeting 06 October 2020
Expected publication 16 December 2020

Project Team

Project lead Kate Moore

Email enquiries


Companies sponsors Vertex Pharmaceuticals (elexacaftor, tezacaftor and ivacaftor)
Others Department of Health and Social Care
  NHS Enfield CCG
  NHS England
  NHS North Norfolk CCG
  Welsh Government
Patient carer groups British Lung Foundation
  CF Voices
  Contact a Family
  Cystic Fibrosis Care
  Cystic Fibrosis Trust
  Genetic Alliance
  Genetic Disorders UK
  Muslim Council of Britain
  NARA – The Breathing Charity
  South Asian Health Foundation
  Specialised Healthcare Alliance
  Together for Short Lives
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 Geriatrics Society
  British Inherited Metabolic Disease Group
  British Paediatric Respiratory Society
  British Rhinological Society
  British Society for Gene and Cell Therapy
  British Society for Genetic Medicine
  British Society for Human Genetics
  British Thoracic Society
  Chartered Society of Physiotherapy
  Cystic Fibrosis Nurses Association
  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 Pharmacy Group
  UK Cystic Fibrosis Medical Association
  UK Genetic Testing Network
  UK Psychosocial Professionals in Cystic Fibrosis Group


Associated public health groups Public Health England
  Public Health Wales
Comparator companies Accord UK (carbocisteine)
  Aurobindo Pharma – Milpharm (carbocisteine)
  Brown & Burk (carbocisteine)
  Dr Reddy’s (carbocisteine)
  Essential Pharmaceticals (pancreatin)
  Flamingo Pharma (carbocisteine)
  Intrapharm Laboratories (carbocisteine)
  Janssen (pancreatin)
  Merck Serono (pancreatin)
  Mylan (pancreatin)
  Pharmaxis (mannitol dry powder for inhalation)
  Roche Products (dornase alfa)
  Sanofi (carbocisteine)
  Typharm (carbocisteine)
  Zentiva (carbocisteine)
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, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  National Heart and Lung Institute
  National Association of Primary Care
  National Pharmacy Association
  NHS Alliance
  NHS Confederation
  Scottish Medicines Consortium
  Welsh Health Specialised Services Committee
Relevant research groups British Association for Lung Research
  CF Unite
  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


Key events during the development of the guidance:

Date Update
19 February 2020 Invitation to participate
19 February 2020 In progress, Invitation to participate
03 February 2020 (10:00) Scoping workshop (Manchester)
12 December 2019 - 17 January 2020 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance