Komodo Health’s RWE technology platform drives two real-world studies that demonstrate Ocalivia improves outcomes for people with primary biliary cholangitis

RWE that Ocaliva improves outcomes for people living with primary biliary cholangitis has been generated in two collaborative studies between Komodo Health and Intercept Pharmaceuticals.

At the annual meeting of the American Association for the Study of Liver Diseases in November 2022, results were announced from two real-world studies on the impact of Ocaliva in the treatment of primary biliary cholangitis (PBC). The two studies by Intercept Pharmaceuticals, named COBALT and HEROES-US, were both powered by Komodo Health’s RWD technology platform to generate RWE that Ocaliva improves outcomes for people living with PBC.

PBC is a rare autoimmune disease of the liver bile ducts that is most prevalent (1 in 10,000) in women over the age of 40. It causes accumulation of bile acid in the bile ducts, resulting in inflammation and scarring that can lead to cirrhosis and the need for a liver transplant, or death. Designated orphan medicine Ocaliva contains the active ingredient obetocholic acid (OCA), a modified form of bile acid that binds to receptors and reduces bile production, preventing build-up.

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COBALT

Intercept first initiated the study COBALT, a randomized, double-blind, placebo-controlled trial designed to assess efficacy and safety of OCA in patients with advanced PBC. COBALT was terminated in December 2021, before it was fully enrolled, due to the inherent challenges with enrolling and maintaining patients in a placebo-controlled study of a rare disease when the study drug is commercially available. Instead, an external control (EC) group was assembled from the Komodo Health US claims database, as a second comparator arm.

The COBALT analysis then assessed the effect of OCA treatment on time to first occurrence of PBC disease progression (hepatic decompensation), liver transplant or death of OCA-treated subjects in the COBALT clinical trial compared to the non-OCA-treated EC group created from the Komodo database. Results showed a 61% reduction with OCA in risk of measured events at any time during follow-up, and 52% reduction for FDA expanded primary endpoints.

“We have long understood the challenges of running placebo-controlled, post-marketing studies, which is why we initiated real-world analyses and leveraged rigorously matched external controls for COBALT to provide additional opportunities to understand the effect of OCA on clinical outcomes in PBC,” said Michelle Berrey, President of R&D and CMO of Intercept. “We believe that the real-world data we have generated to date demonstrate long-term benefits to people with PBC who are receiving OCA, specifically improved event-free survival, which we know to be the most important treatment goal for patients and clinicians.”

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HEROES-US

A second study, HEROES-US, leveraged the Komodo Health database for a fully real-world analysis of the effects of OCA treatment on people living with PBC. This retrospective observational study was designed and executed according to the FDA’s draft guidance on use of RWE to support regulatory decision-making for drug and biological products.

In HEROES-US, Komodo’s Sentinel application was deployed to link data from the Komodo Healthcare Map with other key data sources, including Quest and LabCorp laboratory data and the Social Security Death Index. The study compared clinical outcomes of people with PBC who were treated with OCA with a comparable group of people with PBC who were eligible, but not treated with OCA. A statistically significant and clinically meaningful reduction of 63% in all-cause death, liver transplant, or hospitalization for hepatic decompensation was seen among OCA-treated patients compared to the control group who were not treated with OCA.

Arif Nathoo, Komodo Health Co-Founder and CEO, said: “Komodo launched Sentinel to enable real-world evidence at scale. Intercept’s HEROES study embodies our view of the future, one that is based on using real-time patient insights to improve patient outcomes. These kinds of important studies, which link Komodo’s Healthcare Map to labs, demographics, and multiple other data sources, are finally unlocking an insight-driven transformation across the industry.”