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
If you have any queries please email scheduling@nice.org.uk
Stakeholders
- 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
- Jnetics
- 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
- BDA
- 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
- 4-Front
- 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
Timeline
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 (https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data-collection-agreement) following the announcement of a 4-year deal agreed by NHS England & NHS and Vertex (https://www.england.nhs.uk/2020/06/nhs-patients-among-first-in-europe-to-benefit-from-landmark-deal-for-cystic-fibrosis-treatment/) . 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. https://www.england.nhs.uk/2020/06/nhs-patients-among-first-in-europe-to-benefit-from-landmark-deal-for-cystic-fibrosis-treatment/ |
| 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