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 TA
ID number 3834

Project Team

Project lead Kate Moore

Email enquiries

External Assessment Group BMJ Technology Assessment Group (BMJ-TAG), BMJ


Companies sponsors Vertex Pharmaceuticals (elexacaftor, tezacaftor, lumcaftor and ivacaftor)
Others Department of Health and Social Care
  NHS England
Patient carer groups CF Voices
  Cystic Fibrosis Trust
  Quest for a CF Cure
Professional groups Association of Chartered Physiotherapists in Cystic Fibrosis
  British Dietetic Association
  British Paediatric Respiratory Society
  CF Health Hub / CF DigiCare
  Cystic Fibrosis Nursing Association
  Neonatal and Paediatric Pharmacists Group
  Royal College of Paediatrics & Child Health
  Royal College of Physicians
  UK Cystic Fibrosis Medical Association
  UK Cystic Fibrosis Pharmacy Group
  UK Clinical Pharmacy Association
  UK Psychosocial Professionals in Cystic Fibrosis Group
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department for Health - Northern Ireland
  Healthcare Improvement Scotland
  Medicines and Healthcare Products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research


Key events during the development of the guidance:

Date Update
31 January 2024 Following the technology appraisal committee meeting on 14 December 2023, stakeholders are reminded that access to treatments will continue while the appraisal is ongoing. This will be the case for people starting the treatments as well as those already taking the treatments. NICE has determined with key stakeholders that additional steps are required. We are pausing publication of final draft guidance after the second committee meeting to allow further discussions. The earliest anticipated date of final guidance publication will be updated once timelines are confirmed. Further details can be found here:
19 December 2023 The second technology appraisal committee meeting took place on 14 December 2023. Existing patients and new patients who start treatment while the NICE evaluation is ongoing will continue to have access to the treatments after NICE has issued its final recommendations irrespective of the outcome. NICE will be liaising with key stakeholders to determine the most appropriate next steps and will provide an update in January 2024. Further details can be found here:
14 December 2023 Committee meeting: 2
03 November 2023 - 24 November 2023 Draft guidance
03 November 2023 Access to treatments will continue while the appraisal is ongoing. This will be the case for people starting the treatments as well as those already taking the treatments. Once the appraisal has ended, new patients will only be initiated on treatments that have been recommended in NICE’s final guidance. Existing patients will be unaffected as the flexible commercial mechanism ensures continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation.
12 October 2023 Committee meeting
29 August 2023 Assessment report sent for information
27 January 2023 Invitation to participate
27 January 2023 In progress. Invitation to participate
18 November 2022 - 16 December 2022 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators: 1

For further information on how we select topics for development, please see our page about topic selection