[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
Application field:
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.
| Project no. | Title | Status |
|---|---|---|
| A18-08 | Lumacaftor/ivacaftor (cystic fibrosis) - Benefit assessment according to §35a Social Code Book V | Commission completed |
| G15-14 | Lumacaftor/ivacaftor - Assessment according to §35a (para. 1., sentence 10) Social Code Book V | Commission completed |
| A21-122 | Lumacaftor/ivacaftor (cystic fibrosis, homozygous F508del mutation in the CFTR gene, 2-5 years) - Benefit assessment according to §35a Social Code Book V (expiry of the limitation period) | Commission completed |
| A23-72 | Lumacaftor/ivacaftor (cystic fibrosis, 1 to < 2 years, F508del mutation, homozygous) – Benefit assessment according to §35a Social Code Book V | Commission completed |