obeticholic acid (Ocaliva)

following a full submission assessed under the orphan medicine process:

obeticholic acid (Ocaliva®) is accepted for use within NHS Scotland.

Indication under review:  for the treatment ofprimary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid in adults with an inadequate response to ursodeoxycholic acid or as monotherapy in adults unable to tolerate ursodeoxycholic acid.

In a randomised, double-blind, phase III study of patients with early stage primary biliary cholangitis and poor response or intolerance to ursodeoxycholic acid, treatment with obeticholic acid (+/- concomitant ursodeoxycholic acid) was associated with a greater biochemical response rate at 12 months when compared with placebo. 

This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of obeticholic acid. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.