Vanzacaftor-tezacaftor-deutivacaftor for treating cystic fibrosis with 1 or more F508del mutations in the CFTR gene in people 6 years and over

Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) can be used as an option to treat cystic fibrosis in people 6 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vnz-Tez-Diva can only be used if the company provides it according to the commercial arrangement .

Use the least expensive option of the suitable treatments (including Vnz-Tez-Diva and ivacaftor-tezacaftor-elexacaftor [Iva-Tez-Elx]), having discussed the advantages and disadvantages of the available treatments with the person with the condition. Take account of administration costs, dosages, price per dose and commercial arrangements.

This recommendation is not intended to affect treatment with Vnz-Tez-Diva that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers. What this means in practice Vnz-Tez-Diva must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Vnz-Tez-Diva must be funded in England within 30 days of final publication of this guidance. There is enough evidence to show that Vnz-Tez-Diva provides benefits and value for money, so it can be used routinely across the NHS in this population. NICE has produced tools and resources to support the implementation of this guidance . Usual treatment for cystic fibrosis with at least 1 F508del mutation in the CFTR gene is Iva-Tez-Elx, which is licensed for people 2 years and over. Vnz-Tez-Diva works in a similar way and is licensed for people 6 years and over. Clinical trial evidence shows that Vnz-Tez-Diva is as effective as Iva-Tez-Elx in people 12 years and over at improving lung function, growth and weight gain and reducing the number of lung infections. It has not been directly compared with Iva-Tez-Elx in people aged 6 to 11 years but is likely to work as well in these people. A cost comparison of Vnz-Tez-Diva and Iva-Tez-Elx in people 6 years and over suggests that the costs are similar. But Iva-Tez-Elx is licensed for people with at least 1 F508del mutation and no other responsive mutation. So, Vnz-Tez-Diva can only be used in people 6 years and over who have at least 1 F508del mutation in the CFTR gene. For all evidence see the committee papers . For more information on NICE's evaluation of Iva–Tez–Elx, see the committee discussion section in NICE's technology appraisal guidance on ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis .

Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva; Alyftrek, Vertex) is indicated for 'the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene'.

The dosage schedule for Vnz-Tez-Diva is available in the summary of product characteristics for: • Vnz 10 mg, Tez 50 mg and Diva 125 mg tablets • Vnz 4 mg, Tez 20 mg and Diva 50 mg tablets . Vnz 10 mg, Tez 50 mg and Diva 125 mg tablets Vnz 4 mg, Tez 20 mg and Diva 50 mg tablets .

The list price for Vnz-Tez-Diva (excluding VAT; company submission) is £16,110.00 per: • 84-tablet pack of Vnz 10 mg, Tez 50 mg and Diva 125 mg • 56-tablet pack of Vnz 4 mg, Tez 20 mg and Diva 50 mg. 84-tablet pack of Vnz 10 mg, Tez 50 mg and Diva 125 mg 56-tablet pack of Vnz 4 mg, Tez 20 mg and Diva 50 mg.

The company has a commercial arrangement with NHS England. As part of this agreement, Vnz-Tez-Diva is available to the NHS with a discount. The size of the discount is commercial in confidence.

For information, Vertex did not disclose its Carbon Reduction Plan for UK carbon emissions.

Section 7 of the National Institute for Health and Care Excellence (Constitution and Functions) and the Health and Social Care Information Centre (Functions) Regulations 2013 requires integrated care boards, NHS England and, with respect to their public health functions, local authorities to comply with the recommendations in this evaluation within 90 days of its date of publication. Because vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) has been recommended through the cost-comparison process , NHS England and integrated care boards have agreed to provide funding to implement this guidance 30 days after publication. NHS England has agreed a confidential commercial agreement that enables access to Vnz-Tez-Diva from the date of the final draft guidance.

The Welsh ministers have issued directions to the NHS in Wales on implementing NICE technology appraisal guidance. When a NICE technology appraisal guidance recommends the use of a drug or treatment, or other technology, the NHS in Wales must usually provide funding and resources for it within 60 days of the first publication of the final draft guidance.

When NICE recommends a treatment 'as an option', the NHS must make sure it is available within the period set out in the paragraphs above. This means that, if a patient has cystic fibrosis with at least 1 F508del mutation in the CFTR gene and the healthcare professional responsible for their care thinks that Vnz-Tez-Diva is the right treatment, it should be available for use, in line with NICE's recommendations.

Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) can be used as an option to treat cystic fibrosis in people 6 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vnz-Tez-Diva can only be used if the company provides it according to the commercial arrangement .

Use the least expensive option of the suitable treatments (including Vnz-Tez-Diva and ivacaftor-tezacaftor-elexacaftor [Iva-Tez-Elx]), having discussed the advantages and disadvantages of the available treatments with the person with the condition. Take account of administration costs, dosages, price per dose and commercial arrangements.

This recommendation is not intended to affect treatment with Vnz-Tez-Diva that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers. What this means in practice Vnz-Tez-Diva must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Vnz-Tez-Diva must be funded in England within 30 days of final publication of this guidance. There is enough evidence to show that Vnz-Tez-Diva provides benefits and value for money, so it can be used routinely across the NHS in this population. NICE has produced tools and resources to support the implementation of this guidance . Usual treatment for cystic fibrosis with at least 1 F508del mutation in the CFTR gene is Iva-Tez-Elx, which is licensed for people 2 years and over. Vnz-Tez-Diva works in a similar way and is licensed for people 6 years and over. Clinical trial evidence shows that Vnz-Tez-Diva is as effective as Iva-Tez-Elx in people 12 years and over at improving lung function, growth and weight gain and reducing the number of lung infections. It has not been directly compared with Iva-Tez-Elx in people aged 6 to 11 years but is likely to work as well in these people. A cost comparison of Vnz-Tez-Diva and Iva-Tez-Elx in people 6 years and over suggests that the costs are similar. But Iva-Tez-Elx is licensed for people with at least 1 F508del mutation and no other responsive mutation. So, Vnz-Tez-Diva can only be used in people 6 years and over who have at least 1 F508del mutation in the CFTR gene. For all evidence see the committee papers . For more information on NICE's evaluation of Iva–Tez–Elx, see the committee discussion section in NICE's technology appraisal guidance on ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis .

Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) can be used as an option to treat cystic fibrosis in people 6 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vnz-Tez-Diva can only be used if the company provides it according to the commercial arrangement .

Use the least expensive option of the suitable treatments (including Vnz-Tez-Diva and ivacaftor-tezacaftor-elexacaftor [Iva-Tez-Elx]), having discussed the advantages and disadvantages of the available treatments with the person with the condition. Take account of administration costs, dosages, price per dose and commercial arrangements.

This recommendation is not intended to affect treatment with Vnz-Tez-Diva that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers. What this means in practice Vnz-Tez-Diva must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Vnz-Tez-Diva must be funded in England within 30 days of final publication of this guidance. There is enough evidence to show that Vnz-Tez-Diva provides benefits and value for money, so it can be used routinely across the NHS in this population. NICE has produced tools and resources to support the implementation of this guidance . Usual treatment for cystic fibrosis with at least 1 F508del mutation in the CFTR gene is Iva-Tez-Elx, which is licensed for people 2 years and over. Vnz-Tez-Diva works in a similar way and is licensed for people 6 years and over. Clinical trial evidence shows that Vnz-Tez-Diva is as effective as Iva-Tez-Elx in people 12 years and over at improving lung function, growth and weight gain and reducing the number of lung infections. It has not been directly compared with Iva-Tez-Elx in people aged 6 to 11 years but is likely to work as well in these people. A cost comparison of Vnz-Tez-Diva and Iva-Tez-Elx in people 6 years and over suggests that the costs are similar. But Iva-Tez-Elx is licensed for people with at least 1 F508del mutation and no other responsive mutation. So, Vnz-Tez-Diva can only be used in people 6 years and over who have at least 1 F508del mutation in the CFTR gene. For all evidence see the committee papers . For more information on NICE's evaluation of Iva–Tez–Elx, see the committee discussion section in NICE's technology appraisal guidance on ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis .