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    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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    Are the recommendations sound and a suitable basis for guidance to the NHS?

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Draft guidance consultation

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1 Recommendations

1.1

Ivacaftor–tezacaftor–elexacaftor (IVA–TEZ–ELX) plus ivacaftor (IVA) alone is not recommended within its marketing authorisation, as an option for treating cystic fibrosis (CF) in people 6 years and over who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.

1.2

Tezacaftor–ivacaftor (TEZ–IVA) plus IVA alone is not recommended, within its marketing authorisation, for treating CF in people 6 years and over who have:

  • 2 copies of the CFTR gene with F508del mutations or

  • a copy of the CFTR gene with an F508del mutation and a copy of the CFTR gene with 1 of the mutations listed in section 2.2.

1.3

Lumacaftor–ivacaftor (LUM–IVA) is not recommended, within its marketing authorisation, for treating CF in people 1 year and over who have 2 copies of the CFTR gene with F508del mutations.

1.4

These recommendations are not intended to affect treatment with IVA–TEZ–ELX, TEZ–IVA or LUM–IVA that was started in the NHS before this guidance was published. People having treatment outside these recommendations may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by the clinician, the child or young person, and their parents or carers.

Why the committee made these recommendations

CF causes a range of challenging symptoms that affect the lungs, digestive system and liver. People with CF have a shortened life expectancy and a greatly reduced quality of life. Usual treatment aims to manage the symptoms and includes several intensive treatments and physical therapies. Treatment is very physically demanding and time consuming for people with CF and their families and carers.

Clinical trial evidence shows that IVA–TEZ–ELX improves lung function, growth and weight gain and reduces the number of lung infections more than standard treatment. It is likely that these benefits last while people are on treatment.

Clinical trial evidence shows that TEZ–IVA and LUM–IVA also improve lung function, growth and weight gain and reduce the number of lung infections more than standard treatment. But the short and long-term improvements are smaller than with IVA–TEZ–ELX.

Even when considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates for IVA–TEZ–ELX, LUM–IVA and TEZ–IVA are above the range that NICE considers an acceptable use of NHS resources. So, they are not recommended.