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| Name | Class |
|---|---|
| Target RWE | INDUSTRY |
| Syneos Health | OTHER |
| Komodo Health, Inc. | INDUSTRY |
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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 were considered for this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OCA-treated | PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated Obeticholic acid (OCA) in the study window. | ||
| Non-OCA Treated | PBC participants with a history of UDCA failure who were eligible but not treated with OCA (or off-label fibrates) in the study window. |
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| Measure | Description | Time Frame |
|---|---|---|
| Risk of the First Event of the Composite Events | The primary analysis outcome was assessed with hazard ratio (HR) comparing hazard of first event of the composite endpoint among OCA-treated participants and SMR-weighted non-OCA-treated PBC participants indexes. It included all-cause death, liver transplant, hospitalization for hepatic decompensation based on first occurrence of: variceal bleed, ascites (including hepatic hydrothorax and spontaneous bacterial peritonitis) and hepatic encephalopathy. OCA-treated indexes were censored 90 days after OCA discontinuation, or if fibrates were initiated. Control indexes were censored if a participant-initiated OCA therapy, initiated fibrate therapy, reinitiated UDCA for participants who had discontinued UDCA for >6 months, or end of study period (31 Dec 2021), whichever came first. The 2.5th and 97.5th percentile of nonparametric bootstrap samples were used to estimate 95% CI for HR and to perform a test of hypothesis. Risk is presented using the number of composite event and components. | Up to 67 months |
| Measure | Description | Time Frame |
|---|---|---|
| Risk of Death | The primary source for death was the Social Security Death Index (SSDI) along with obituary search, which was compared to Komodo Health claims and LabCorp/Quest laboratory data. The secondary objectives were to estimate the effect of OCA treatment versus non-OCA treatment on each component of the composite endpoint, with the same censoring rule applied as in the primary composite endpoint. Risk is presented using the the number of all-cause death within the risk period (prior to censoring). |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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All patients who meet diagnostic criteria in each database between 01 Jun 2015 and 31 Dec 2021 and who meet the eligibility criteria were considered for these studies.
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| Name | Affiliation | Role |
|---|---|---|
| Lynda Szczech, MD | Intercept Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Intercept Pharmaceuticals, Inc | San Diego | California | 92121 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39630028 | Derived | Brookhart MA, Mayne TJ, Coombs C, Breskin A, Ness E, Bessonova L, Chu YJ, Li J, Fried MW, Hansen BE, Kowdley KV, Jones D, Mells G, Trivedi PJ, Hiu S, Kareithi DN, Wason J, Smith R, Seeger JD, Hirschfield GM. Hepatic real-world outcomes with obeticholic acid in primary biliary cholangitis (HEROES): A trial emulation study design. Hepatology. 2025 Jun 1;81(6):1647-1659. doi: 10.1097/HEP.0000000000001174. Epub 2025 Jan 3. |
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Participants were included if, between 01Jun2015 and 31Dec2021, they met criteria for PBC diagnosis: At least 1 inpatient claim with an associated PBC International Classification of Diseases (ICD)-10 code, or at least 2 outpatient claims separated by >1 day with a PBC ICD-9 or ICD-10 code. Patient records (indexes) were selected from a healthcare database. The unit of analysis were indexes, not participants. Analysis was conducted using weighted index methods.
This study was conducted in the United States (US) and data was analyzed from Komodo Health claims database between 01 Jun 2015 to 31 Dec 2021.
| ID | Title | Description |
|---|---|---|
| FG000 | OCA-treated | PBC participants with a history of Ursodeoxycholic acid (UDCA) failure (inadequate response, intolerance, or discontinuation) who initiated Obeticholic acid (OCA) in the study window were included. |
| FG001 | Non-OCA Treated | PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Weighted index population. The SMR weights were used to create a pseudo-population of non-OCA-treated indexes with the same covariate distribution as the OCA-treated participants at the time of OCA initiation and weighted standardized mean differences (SMDs) were computed. This was a real-world study, and it was conducted in a population of patients selected from a databased using weighted index methods" The unit of analysis is Indexes.
| ID | Title | Description |
|---|---|---|
| BG000 | OCA-treated | PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included. |
| BG001 | Non-OCA Treated |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Risk of the First Event of the Composite Events | The primary analysis outcome was assessed with hazard ratio (HR) comparing hazard of first event of the composite endpoint among OCA-treated participants and SMR-weighted non-OCA-treated PBC participants indexes. It included all-cause death, liver transplant, hospitalization for hepatic decompensation based on first occurrence of: variceal bleed, ascites (including hepatic hydrothorax and spontaneous bacterial peritonitis) and hepatic encephalopathy. OCA-treated indexes were censored 90 days after OCA discontinuation, or if fibrates were initiated. Control indexes were censored if a participant-initiated OCA therapy, initiated fibrate therapy, reinitiated UDCA for participants who had discontinued UDCA for >6 months, or end of study period (31 Dec 2021), whichever came first. The 2.5th and 97.5th percentile of nonparametric bootstrap samples were used to estimate 95% CI for HR and to perform a test of hypothesis. Risk is presented using the number of composite event and components. | Weighted index population. The unit of analysis is Indexes. | Posted | Number | Events during the study period | Up to 67 months | Indexes | Indexes |
Up to 67 months
This is an observational real-world data study to evaluate the effectiveness of OCA on hepatic outcomes in participants with PBC and the study did not plan to evaluate safety endpoints. The unit of analysis is Indexes. For all-cause mortality, numbers of all death cases during the whole study period (no censoring applied) are presented.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | OCA-treated | PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included. |
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This is an observational real-world data study to evaluate the effectiveness of OCA on hepatic outcomes in participants with PBC and the study did not plan to evaluate safety endpoints.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Intercept Pharmaceuticals, Inc. | 844-782-4278 | medinfo@interceptpharma.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 24, 2022 | Mar 3, 2024 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
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| Up to 67 months |
| Risk of Liver Transplantation | The primary source for liver transplant was the Organ Transplant Network (OPTN) transplant registry. Risk is presented using the number of liver transplantation. | Up to 67 months |
| Risk of Hospitalization for Hepatic Decompensation | The primary source for hospitalization for hepatic decompensation were Komodo Health claims. Risk is presented using the number of hospitalization for hepatic decompensation. | Up to 67 months |
PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods.
| BG002 | Total | Total of all reporting groups |
| Indexes |
|
| Years |
| Indexes |
|
|
| Sex: Female, Male | Count of Units | Indexes | Indexes |
|
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants | Participants |
|
|
| Region | Count of Units | Indexes | Indexes |
|
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | OCA-treated | PBC participants with a history of UDCA failure (inadequate response, intolerance, or discontinuation) who initiated OCA in the study window were included. |
| OG001 | Non-OCA Treated | PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods. |
|
|
|
| Secondary | Risk of Death | The primary source for death was the Social Security Death Index (SSDI) along with obituary search, which was compared to Komodo Health claims and LabCorp/Quest laboratory data. The secondary objectives were to estimate the effect of OCA treatment versus non-OCA treatment on each component of the composite endpoint, with the same censoring rule applied as in the primary composite endpoint. Risk is presented using the the number of all-cause death within the risk period (prior to censoring). | Weighted index population. The unit of analysis is Indexes. | Posted | Number | Events during the study period | Up to 67 months | Indexes | Indexes |
|
|
|
| Secondary | Risk of Liver Transplantation | The primary source for liver transplant was the Organ Transplant Network (OPTN) transplant registry. Risk is presented using the number of liver transplantation. | Weighted index population. The unit of analysis is Indexes. | Posted | Number | Events during the study period | Up to 67 months | Indexes | Indexes |
|
|
|
| Secondary | Risk of Hospitalization for Hepatic Decompensation | The primary source for hospitalization for hepatic decompensation were Komodo Health claims. Risk is presented using the number of hospitalization for hepatic decompensation. | Weighted index population. The unit of analysis is Indexes. | Posted | Number | Events during the study period | Up to 67 months | Indexes | Indexes |
|
|
|
| 12 |
| 403 |
| 0 |
| 0 |
| 0 |
| 0 |
| EG001 | Non-OCA Treated | PBC participants with a history of UDCA failure who were eligible but not treated with OCA for the study. This was a real-world study, and it was conducted in a population of participants selected from a database using weighted index methods. | 17 | 405 | 0 | 0 | 0 | 0 |
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| D004066 |
| Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| Title | Measurements |
|---|---|
|
| West |
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| South |
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| Territory |
|
| Unknown |
|