- Date 18 November 2020
- Documents : 9
- Economic analysis
- History
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Reason for request
Key points
Favourable opinion for reimbursement in the treatment of cystic fibrosis (CF) in patients aged 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.
What therapeutic improvement ?
Therapeutic improvement in management.
Role in the care pathway ?
The management of cystic fibrosis patients requires the intervention of a multidisciplinary team (cystic fibrosis resource and expertise centres, primary care physicians, specialist centres, paramedical team with physiotherapist and nurse). Treatment is symptomatic and life-long. It is based on complementary interventions, in particular respiratory and nutritional management and patient education.
In patients homozygous for the F508del mutation of the CFTR gene, CFTR protein modulators have been shown to be effective: ORKAMBI (lumacaftor/ivacaftor) and SYMKEVI (tezacaftor/ivacaftor) in combination with KALYDECO (ivacaftor).
In patients heterozygous for the F508del mutation with a minimal function (MF) mutation, before the arrival of KAFTRIO (ivacaftor/tezacaftor/elexacaftor), the care pathway did not include any CFTR modulators.
Role of the combination in the care pathway
KAFTRIO (ivacaftor/ tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) is a long-term treatment that should be prescribed from the outset in patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.
In patients heterozygous for the F508del mutation in the CFTR gene with a minimal function (MF) mutation, in the absence of a therapeutic alternative and considering the robust demonstration of its efficacy, KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) constitutes the reference treatment.
In the treatment of patients who are homozygous for the F508del mutation in the CFTR gene, given the substantial clinical benefit demonstrated compared to SYMKEVI (tezacaftor/ivacaftor), KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) is the first-line treatment.
Clinical Benefit
| Substantial |
The Committee deems that the clinical benefit of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) is substantial in the treatment of patients with cystic fibrosis aged 12 years and older who are heterozygous for the F508del mutation in the CFTR gene with a minimal function (MF) mutation. The Committee deems that the clinical benefit of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) is substantial in the treatment of patients with cystic fibrosis aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. |
Clinical Added Value
| important |
Considering :
and despite :
the Committee considers that KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with KALYDECO (ivacaftor) provides a substantial clinical added value (CAV II) in the therapeutic management of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.
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