Original Title: Elexacaftor/tezacaftor/ivacaftor en fibrosis quística

Authors' recommendations: Moderate-quality evidence suggests that in patients 12 years old or more with cystic fibrosis who are homozygous for the F508del-mutation, treatment with elexacaftor-tezacaftor-ivacaftor improves quality of life and the percentage of predicted VEF1 at four weeks when compared with tezacaftor-ivacaftor. Moderate-quality evidence suggests that in patients 12 years old or more with cystic fibrosis who are homozygous for the F508delminimal function mutation, treatment with elexacaftor-tezacaftor-ivacaftor improves quality of life at eight weeks compared with tezacaftor-ivacaftor and that it decreases respiratory exacerbations and improves quality of life and the percentage of predicted VEF1 at 24 weeks when compared with placebo. In all the scenarios, the safety profile of the intervention is acceptable. In both populations, whether these benefits maintain in time is unknown. Low-quality evidence suggests that the elexacaftor-tezacaftor-ivacaftor combination might improve quality of life and the percentage of predicted VEF1 in patients 6 to 12 years old who are heterozygous for the F508del-mutation. Given its recent approval, most clinical practice guidelines consulted do not mention this therapy. Only the 2021 Argentine Society of Pediatrics guideline includes positive comments on its effectiveness. No information related to coverage of this combination has been found in any of the Latin American countries consulted. Funders from the United States, Canada, France and Germany cover this drug combination. In other high-income countries, this combination is not mentioned or is under assessment. In Argentina, the National Administration of Medicines, Foods and Medical Devices has recently granted marketing authorization to this drug combination manufactured by a national pharmaceutical company (Trixacar®). The one manufactured abroad ((Trikafta®) has not been granted marketing authorization and should be bought under a special regimen. The sale price of the locally manufactured drug is significantly lower than that of the imported one. No bioequivalence and/or exchangeability studies with Trikafta® y Trixacar® have been found. In the country, there are still no economic evaluations carried out on the treatment with elexacaftor-tezacaftor-ivacaftor in any of its sales formulations. The evaluations carried out in the United States and Canada determined that the combination is not cost-effective in these countries if there is not a significant price reduction. Given the difference in the price of the sales formulation available in the country versus tezacaftor-ivacaftor, the cost-effectiveness and potential budget impact were uncertain at the time of this evaluation.

Project Status: Completed

Year Published: 2022

URL for published report: https://www.iecs.org.ar/publicacion/?id=21787

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Argentina

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)