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New HTA Decisions in France
December 2021
Drug name
KAFTRIO®/KALYDECO® (ivacaftor / tezacaftor / elexacaftor)
Vertex Pharmaceuticals
Decision date
Therapeutic area
Respiratory conditions
Therapeutic sub area
Cystic fibrosis
Official notice date
Reimbursement %
Decision (SMR)
Decision (ASMR)
Minor improvement (IV)
KAFTRIO®/KALYDECO® is indicated for the treatment of patients with cystic fibrosis aged 12 years and older who have at least one F508del mutation in the CFTR gene.
Decision Type
Extension of indication
Considering the seriousness of the condition, the good benefit-risk profile of KAFTRIO®/KALYDECO®, the alternatives available, the important place of KAFTRIO®/KALYDECO® in the therapeutic strategy, and the likely impact of KAFTRIO®/KALYDECO® on public health, the committee considered the actual benefit of KAFTRIO®/KALYDECO® to be ‘important’ in the indication of the Marketing Authorisation and proposed a reimbursement rate of 65%. Furthermore, given the benefit of KAFTRIO®/KALYDECO® in terms of FEV1 and sweat chloride level, its favourable safety profile, the partially covered medical need, and the lack of impact of triple therapy on quality of life, the committee considered that KAFTRIO®/KALYDECO® provides a minor improvement in actual benefit (ASMR IV) in this indication. It was concluded that the target population for KAFTRIO®/KALYDECO® is estimated to be 530 patients.