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.
Following on from information provided to NICE by the company in October 2020, the appraisal of Elexacaftor, tezacaftor and ivacaftor fixed dose combination therapy for treating cystic fibrosis with the F508del mutation [ID1661] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
Status Discontinued
Technology type Medicine
Decision Selected
Process TA
ID number 1661

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
25 January 2023 Discontinued. Following on from information provided to NICE by the company in October 2020, the appraisal of Elexacaftor, tezacaftor and ivacaftor fixed dose combination therapy for treating cystic fibrosis with the F508del mutation [ID1661] was suspended from NICE’s work programme. As no further information has been received from the company the topic has been discontinued.
23 October 2020 The interim data collection agreement for Vertex’s cystic fibrosis modulator therapies has been updated to include elexacaftor, tezacaftor and ivacaftor ( following the announcement of a 4-year deal agreed by NHS England & NHS and Vertex ( . The updated interim data collection agreement enables eligible patients continued access to Vertex’s cystic fibrosis modulator therapies while further data is collected to inform a future NICE technology appraisal. In addition, future therapeutic indications (for ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor or ivacaftor/tezacaftor/elexacaftor) that receive a marketing authorisation from the Medicines and Healthcare products Regulatory Agency or the European Medicines Agency (applicable to the United Kingdom) during the term of the interim data collection agreement will automatically be considered part of the agreement and formalised through a contract addendum. NICE is currently unable to make a recommendation about the use of elexacaftor, tezacaftor and ivacaftor combination therapy in the NHS. In line with the updated interim data collection agreement the company has confirmed that it will provide an evidence submission towards the end of the interim access agreement initiating the NICE appraisal. NICE will contact stakeholders about participating in the new appraisal in due course.
23 October 2020 Suspended. Appraisal suspended
02 July 2020 Following the announcement that NHS patients will be among first in Europe to benefit from access to ivacaftor, tezacaftor and elexacaftor, the appraisal of this triple therapy is now paused. NICE will work with the company and the NHS on continued data collection to inform a future appraisal. New details and timelines for an appraisal will be confirmed in due course.
02 June 2020 Following a request from Vertex Pharmaceuticals, we have had to reschedule this technology appraisal. Vertex has not provided NICE with an evidence submission by the deadline that took into account an expected licensing date towards the end of this calendar year. The appraisal cannot proceed without an evidence submission from the company. Vertex has indicated that it needs more time to allow for further data from the clinical and real world evidence studies to be included. They have committed to providing us with a full evidence submission by January 2021 at the latest. The first committee has been rescheduled to 12 August 2021 on this basis. We appreciate that this is very disappointing for those waiting with great anticipation for the triple therapy to become available, especially in the context of the coronavirus COVID-19 pandemic. Every week that goes by without an evidence submission from the company will lead to delay in the availability of NICE guidance. We will continue to work with Vertex on the evidence we need, and stand ready to receive their submission at the earliest opportunity between now and January 2021.
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 our processes and methods, please see our CHTE processes and methods manual