[Lumacaftor/Ivacaftor therapy in cystic fibrosis]

Klappenbach R, García Martí S, Pichon-Riviere A, Augustovski F, Alcaraz A, Bardach A, Ciapponi A.
Record ID 32018001494
Original Title: Lumacaftor/Ivacaftor en fibrosis quística
Authors' recommendations: CONCLUSIONS High-quality evidence in patients 12 years old or older with cystic fibrosis homozygous for Phe508 mutation and mild to moderate lung disease shows that treatment with lumacaftor/ivacaftor significantly improves the number of exacerbations and mildly improves the rate of the predicted FEV1, respiratory symptoms, and body mass index after 24 weeks. It is not known whether these benefits are maintained at long term. There is uncertainty on the incidence of adverse events related to the drug due to the inconsistency between clinical trials and observational studies. Moderate-quality evidence in children 6 to 11 years old with cystic fibrosis homozygous for Phe508 mutation and mild lung disease shows that after 24 weeks, the use of lumacaftor/ivacaptor does not improve clinical outcomes like the number of exacerbations, respiratory symptoms or body mass index, but it mildly improves the predicted rate of FEV1 and the rate of lung clearance. The clinical practice guideline consulted that mentions this technology recommends it for both indications although it recognizes it prescription might be influenced by the health sponsor coverage and the cost for the patient. There is no consensus on the coverage of this drug in high-income countries. The sponsors who do not cover it and the economic evaluations from high-income countries do not meet cost-effectiveness criteria. Latin America’s health systems do not cover or mention it. Argentina’s National Administration of Drugs, Food and Medical Technology has approved the use of this technology only for children over 12 years old.
Project Status: Completed
Year Published: 2018
URL for published report: https://www.iecs.org.ar/home-ets/
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Cystic Fibrosis
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Aminophenols
  • Quinolones
  • Drug Therapy, Combination
Organisation Name: Institute for Clinical Effectiveness and Health Policy
Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name: info@iecs.org.ar
Contact Email: info@iecs.org.ar
Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.