Obeticholic acid (Ocaliva®) for the treatment of primary biliary cholangitis

Zorginstituut Nederland carried out an assessment of whether obeticholic acid (Ocaliva®) is interchangeable with a drug currently included in the Medicine Reimbursement System (GVS). Obeticholic acid (Ocaliva®) is registered (in the form of film-coated tablets with 5 mg active substance) for the treatment of primary biliary cholangitis (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

Zorginstituut's advice

The Zorginstituut feels that obeticholic acid has no demonstrated added value, because of the following reasons:

  • The Zorginstituut feels that there are better cut-off points than those used in the clinical trial. As a consequence, the effect of treatment on mortality and morbidity is uncertain;
  • The published clinical study (the POISE study) involved mainly patients with a good prognosis;
  • The POISE study's short follow-up of the placebo-arm;
  • The insufficient quality of the two new, ad-hoc, surrogate outcome measures that the manufacturer presented in second instance. Unknown is, partly due to the lack of statistical analyses between the study arms, whether 12 months of treatment with obeticholic acid leads to significantly lower risk scores in comparison to placebo.

The Zorginstituut therefore advises the Minister of VWS against including this drug in the insured package because of inadequate evidence regarding the drug's performance in daily practice.

This report is a summary of recommendations by Zorginstituut Nederland. The original text of this excerpt is in Dutch.