Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients

Study Overview
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Brief Summary
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This is an observational, retrospective cohort study of patients with primary biliary cholangitis (PBC) who failed ursodeoxycholic acid (UDCA) treatment, using a real-world data source, the Komodo Health United States (US) claims database. The study is designed to evaluate the effectiveness of obeticholic acid (OCA). All patients who meet diagnostic criteria for PBC in the database between 01 Jun 2015 and 31 Dec 2021 and who meet all eligibility criteria will be considered for this study.

Official Title
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Replicate Studies Evaluating the Effectiveness of Obeticholic Acid on Hepatic Real-World Outcomes in Patients With Primary Biliary Cholangitis

Conditions
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Primary Biliary Cholangitis

Intervention / Treatment
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* Drug: Obeticholic Acid 5 MG
* Drug: Obeticholic Acid 10 MG
* Drug: Standard of Care: UDCA


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Definite or probable PBC diagnosis Inadequate response or intolerance to UDCA Age ≥18 years at the index date Continuous enrollment and evaluable data for at least 12 months before the index date (inclusive) Key Exclusion Criteria: History or presence of other concomitant liver diseases History of non-skin malignancy or melanoma History of HIV Medical conditions that may cause non-hepatic increases in ALP Patients with laboratory values indicative of hepatic decompensation or significant hepatobiliary injury History of liver transplant Evidence of fenofibrate, or bezafibrate use History or presence of hepatic decompensating events

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| OCA Treatment Group<br>PBC patients with a history of inadequate response or intolerance to UDCA who initiated OCA in the study window (01 Jun 2015 to 31 Dec 2021) | Drug: Obeticholic Acid 5 MG<br>* once daily, oral administration<br>* Other names: Ocaliva;Drug: Obeticholic Acid 10 MG<br>* once daily, oral administration<br>* Other names: Ocaliva;|
| Control Group<br>PBC patients with a history of inadequate response or intolerance to UDCA who were eligible but were not treated with OCA (or off-label fibrates) in the study window (01 Jun 2015 to 31 Dec 2021) | Drug: Standard of Care: UDCA<br>* No study medication is provided by the sponsor or by the investigators. The decision to initiate, continue or discontinue UDCA, or to modify UDCA dosing, is entirely at the discretion of the treating physician as per their standard of care and is in no way influenced by the sponsor or participating institutions. UDCA utilization is recorded and included in the study record.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 1. Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hospitalization for hepatic decompensation. |  | Time from index date to first occurrence of the composite endpoint events, assessed up to 67 months. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Primary Biliary Cholangitis, Primary Biliary Cirrhosis, PBC, Hepatic Impairment, Liver