Intervention of Interest:
- Obeticholic Acid (OCALIVA®, Intercept Pharmaceuticals, Inc.)
Obeticholic acid (OCA) is a novel bile acid analogue that has shown positive effects on biochemical markers of liver function in phase II trials and was approved by the FDA for treatment of primary biliary cholangitis (PBC) after failure of ursodeoxycholic Acid (UDCA). Primary biliary cholangitis (PBC), which has until recently been called primary biliary cirrhosis, is a rare, chronic, progressive autoimmune liver disease that affects mainly middle-aged women. In the US, up to 130,000 individuals may have PBC.
In January 2015, obeticholic acid also received a breakthrough designation for treatment of non-alcoholic steatohepatitis (NASH) with concomitant liver fibrosis. Interim findings from the phase III trial are not expected to be available until March 2017; however, clinical interest in potential off-label use of obeticholic acid is likely to be great, given the lack of available treatments with liver-specific effects. NASH is form of nonalcoholic fatty liver disease (NAFLD) that can progress to cirrhosis, liver failure, and cancer. NASH is present in about 30 million adults in the US.
A majority of the Council voted that for patients with PBC who do not have an adequate response to usual care with ursodeoxycholic acid (UDCA), the evidence demonstrates that OCA provides a net health benefit. When voting on the care value of the drug, a measure which considers the clinical effectiveness of the drug, its cost, contextual considerations, and added benefits or disadvantages of the treatment, a majority of the Council found OCA to represent a low care value, citing the high cost of the drug as the primary reason for their vote. Some members, voting for an intermediate value, indicated that the high percentage of patients who do not respond to UDCA, along with possible productivity gains for a patient population that includes predominantly working-age women, represent important contextual considerations that contribute to the overall care value of the drug.
When voting on the clinical effectiveness of OCA for treatment of NASH, the Council voted unanimously that evidence is not adequate to demonstrate a net health benefit based on the preliminary nature of the data.
Date of Review: July 2016
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Below you will find the final documents from the policy paper review process:
NASH & PBC Documents:
PBC Documents Only:
NASH Documents Only:
“Patients with PBC have very limited options when it comes to treatments. We’re pleased that our report was able to shed light on the potential health benefit of OCA for people with PBC who aren’t benefiting from currently available treatments. We hope that our findings on value will contribute to constructive discussions about pricing to ensure that as many patients as possible who can benefit from this drug are able to access it.”