[A19-13] Lumacaftor/ivacaftor (cystic fibrosis in children aged 2 to 5 years) - Benefit assessment according to §35a Social Code Book V
Last updated 15.05.2019
Project no.: A19-13
Commission: Commission awarded on 11.02.2019 by the Federal Joint Committee (G-BA).
Report type: Dossier assessment
Status: Commission completed
Department/Division: Drug Assessment
Topic: Children's and adolescents' health
Cystic fibrosis in patients aged 2 to 5 years who are homozygous for the F508del mutation in the CFTR gene
Single-arm study without very large (dramatic) effects, no data on comparator therapy, transfer from studies in older children unproductive; added benefit not proven
After completion of the assessment by IQWiG the Federal Joint Committee (G-BA) conducts a commenting procedure. This may provide supplementary information and as a result lead to a modified benefit assessment. Further information and the decision on the early benefit assessment can be found on the relevant page of the G-BA website.