Pharmacoeconomic review report : indication, for the treatment of cystic fibrosis in patients 6 years of age and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene. Lumacaftor/Ivacaftor (Orkambi) (Vertex pharmaceuticals (Canada) Incorporated) / Canadian Agency for Drugs and Technologies in Health.

Format:

Book

Author/Creator:

Canadian Agency for Drugs and Technologies in Health, author, issuing body.

Language:

English

Subjects (All):

Cost effectiveness.

Physical Description:

1 online resource (31 pages) : illustrations

Edition:

Version: Manufacturer comments and redactions.

Place of Publication:

Ottawa (ON) : Canadian Agency for Drugs and Technologies in Health, 2018.

Summary:

Orkambi is a fixed-dose combination tablet containing 200 mg lumacaftor and 125 mg ivacaftor (LUM/IVA). It is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. This is most common CF-causing mutation worldwide and approximately half of all Canadian patients with CF are homozygous for the F508del mutation. LUM/IVA is the first treatment specifically indicated for the treatment of patients who are homozygous for the F508del mutation in the CFTR gene. The manufacturer has requested that LUM/IVA be listed in accordance with the Health Canada-approved indication. The recommended dose is lumacaftor 400 mg/ivacaftor 250 mg every 12 hours. This represents the lower of the dosages considered in both the TRANSPORT AND TRAFFIC trials. At the current marketed price of 170.54 per tablet, the daily cost of treatment per patient with LUM/IVA is 682, or 248,982 annually. CADTH reviewed LUM/IVA in 2015 for the treatment of CF in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. CDEC recommended that LUM/IVA not be reimbursed, based on the clinical findings. The price submitted for LUM/IVA in the original submission is the same as the current submission. The manufacturer submitted a cost-utility analysis to assess the cost-effectiveness of LUM/IVA + standard of care (SoC) compared with SoC alone in patients with CF who are 6 years of age or older and homozygous for the F508del-CFTR mutation.

Notes:

Description based on publisher supplied metadata and other sources.