Lumacaftor/ivacaftor (Orkambi®) by Cystic fibrosis

Based on the GVS criteria, lumacaftor/ivacaftor (Orkambi®) can not be replaced by another drug in the GVS.  The assessment resulted in a therapeutic added value of lumacaftor/ivacaftor compared to best supportive care for the treatment of Cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene.

This means that the drug fulfils the health care package criterion effectiveness

This report is a summary of recommendations by the National Health Care Institute. The original text is in Dutch.