Treatment with obeticholic acid (OCA) has shown positive outcomes in patients with primary biliary cholangitis (PBC), according to a recently published study in the American Journal of Gastroenterology.
PBC is characterized by the immunological destruction of bile ducts, which leads to bile accumulation in the liver, causing liver damage. Current treatment alternatives include ursodeoxycholic acid, which reduces bile accumulation.
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OCA received accelerated U.S. Food and Drug Administration (FDA) approval as a second-line therapy for PBC in 2016 based on clinical trial results that showed it significantly reduced biliary acid accumulation (cholestasis) parameters.
Afterward, the COBALT clinical trial aimed to determine OCA’s impact on the incidence of liver failure, cirrhosis, death, and the need for liver transplantation. The study compared a randomized placebo control group and an external control group; this means that the comparator group is not being drawn directly from the study.
“External control (EC) studies are potentially useful in clinical trials of therapeutic agents for rare diseases such as PBC when the inclusion of a placebo control group may be considered unethical, and patient recruitment and retention can be difficult,” the authors wrote.
In this case, the ECs came from the employed Komodo Healthcare Map, a U.S health claims database.
Results showed that the drug was not safe for cirrhotic patients with portal hypertension, and an appropriate contraindication was written in the boxed warning section.
The authors observed no significant differences in disease progression endpoints between patients receiving OCA and those taking placebo. However, they observed potential interference due to crossover to commercial therapy.
After performing statistical correction due to crossover, the authors found that OCA treatment correlated with mild improvements in disease progression endpoints. The comparison of the controlled group with the EC group revealed that OCA was associated with a significantly lower risk of experiencing adverse clinical outcomes.
“The value of real-world evidence in confirming RCTs is not without precedent in the development of treatments for PBC, as illustrated by the real-world confirmation of the clinical benefits of first-line UDCA,” the authors concluded.
