This Health Technology Assessment (HTA) describes the scientific evidence on the efficacy and safety of lumacaftor + ivacaftor for cystic fibrosis (CF) due to the homozygous F508del mutation and mild to moderate lung involvement in pediatric patients.
This health technology assessment report was generated in response to a request from the Institutional Advisory Commission on Rare and Orphan Diseases of the Ministry of Health.
It was prepared by the Public Health Evidence Analysis and Generation Unit of the (National Institute of Health).
This publication belongs to the compendium Publicaciones – RENETSA