Pharmacoeconomic Review Report: Obeticholic Acid (Ocaliva): (Intercept Pharma Canada, Inc.): Indication: For the treatment of primary biliary cholangitis

Obeticholic acid (OCA) is a selective farnesoid X receptor agonist, available as 5 mg and 10 mg oral tablets at a unit price of $98.63 (for both 5 mg and 10 mg). The review of OCA is for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA (proposed indication). The recommended starting dosage of OCA is 5 mg once daily in adult patients who have not achieved an adequate biochemical response to an appropriate dosage of UDCA for at least one year or who are intolerant to UDCA. If an adequate reduction in alkaline phosphatase (ALP) or total bilirubin has not been achieved after six months of OCA 5 mg once daily, and the patient is tolerating OCA, the dosage should be increased to 10 mg once daily. The maximum recommended dosage of OCA is 10 mg once daily. The manufacturer submitted a cost-utility analysis assessing OCA in two populations: the UDCA-intolerant population (comparing OCA with no treatment), and the population of patients with an inadequate response to UDCA (UDCA-tolerant; comparing OCA plus UDCA with UDCA alone). The base-case analysis was conducted from the perspective of the Canadian health care system over a lifetime horizon (50 years) with future costs and benefits discounted at 1.5 %. The model consisted of 10 health states with transitions taking place every three months, capturing patient progression over time. The model captured the two components of the natural history of the disease: the PBC-specific liver disease component, representing the progression of PBC based on ALP and bilirubin biomarkers (three health states), and the liver disease clinical outcome component (seven health states), which is entered once patients progress to decompensated cirrhosis or hepatocellular carcinoma. For the OCA groups and UDCA group, results from the pivotal phase III POISE study were used to inform health state transitions for each three-month cycle for the first year. After year 1, PBC-specific health state transitions were calculated based on data from the Global and UK PBC study cohorts. Utility data specific to cholangitis patients were used for PBC-specific health states, and Canadian data were used for liver disease clinical outcome states. Resource use and costs were collected from clinical trials, published literature, expert opinion, and standard Canadian sources.