To appraise the clinical and cost effectiveness of tezacaftor in combination with ivacaftor within its marketing authorisation for treating cystic fibrosis in people with the F508del mutation.
Non submission
Status Suspended
Process STA pre-2018
ID number 1303

Project Team

Project lead Kate Moore

Email enquiries

Evidence Review Group / Assessment Group Kleijnen Systematic Reviews


Companies sponsors Vertex Pharmaceuticals (tezacaftor, ivacaftor)
Others Department of Health and Social Care
  NHS England
  Welsh Government
Patient carer groups Cystic Fibrosis Trust
Professional groups Association of Chartered Physiotherapist in Cystic Fibrosis
  British Thoracic Society
  Cystic Fibrosis Nurses Association
  Royal College of Pathologists
  Royal College of Physicians
  UK Clinical Pharmacy Association
  UK Cystic Fibrosis Medical Association
  UK Cystic Fibrosis Pharmacy Group


Comparator companies AbbVie (no CAU, not participating)
  Allergan UK (no CAU, not participating)
  Essential Pharmaceuticals (no CAU, not participating)
  Janssen (no CAU, not participating)
  Merck Serono (no CAU, not participating)
  Pari Medical (no CAU, not participating)
  Pharmaxis (no CAU, not participating)
  Roche Products (no CAU, not participating)
  Sanofi (no CAU, not participating)
  Teva (no CAU, not participating)
  Zentiva (no CAU, not participating)
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
12 November 2019 November 2019: Tezacaftor and ivacaftor combination therapy is available on the NHS for treating cystic fibrosis. NHS England has agreed an interim access agreement with Vertex Pharmaceuticals, which includes collecting further data through an interim data collection agreement -
09 August 2018 Suspended, Non submission
09 August 2018 The company, Vertex Pharmaceuticals, has not provided an evidence submission for this appraisal. Therefore, we are suspending the appraisal whilst we consider the next steps. Consequently the discussion of this appraisal at the committee meeting on the 8 November 2018 has been cancelled. We will update you on our progress and next steps as soon as possible.
29 May 2018 Invitation to participate
22 May 2018 In progress, In progress
23 November 2017 - 21 December 2017 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

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