[Elexacaftor/tezacaftor/ivacaftor in cystic fibrosis]

Perelli L, Bardach A, Olivera G, Alfie V, Argento F, García Martí S, Ciapponi A, Augustovski F, Alcaraz A, Pichon Riviere A
Record ID 32018002319
Spanish
Original Title: Elexacaftor/tezacaftor/ivacaftor en fibrosis quística
Authors' recomendations: Moderate-quality evidence suggests that, in patients 12 years old or older with cystic fibrosis bearing the homozygous F508 mutation, treatment with elexacaftor/tezacaftor/ivacaftor, when compared with tezacaftor/ivacaftor, decreases respiratory exacerbations, improves the predicted VEF1 ratio and improves the patient’s quality of live at 4 weeks with an acceptable safety profile. Moderate-quality evidence suggests that, in patients 12 years old or older, with cystic fibrosis bearing the heterozygous F508-minimal function mutation, treatment with elexacaftor/tezacaftor/ivacaftor, when compared to placebo, improves the predicted VEF1 ratio, decreases respiratory exacerbations, and improves the patient’s quality of life at 24 weeks, with an acceptable safety profile. However, whether this benefits persist at long-term is unknown. No evidence was found against other comparators, or in children under 12 years old.Most clinical practice guidelines surveyed, do no mention this therapy since it has recently been approved, although the 2021 Argentine Society of Pediatrics Guideline has a positive opinion on its effectiveness. No Latin American country covers this drug. Health sponsors from the United States, France and Germany cover this drug combination, whereas other high-income countries do not mention this therapeutic combination or it is under review.No information is available on the economic aspect of this therapy in Argentina, where it is still not commercially available. It was assessed in the United States and it was determined it was not cost-effective.
Details
Project Status: Completed
Year Published: 2021
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Cystic Fibrosis
  • Drug Therapy, Combination
Contact
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.