Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterized by inflammation, progressive damage, and, ultimately, destruction of the interlobular bile ducts; this process is followed by cholestasis, which drives debilitating fatigue and itch, among other symptoms. Without adequate management, PBC culminates in liver fibrosis, cirrhosis, end-stage organ disease, and death. PBC has no cure; the goal of treatment is to halt or slow the progression of liver disease, reduce the need for liver transplantation, lower the risk of mortality, and alleviate symptoms. Ursodeoxycholic acid (UDCA) is the cornerstone of PBC treatment, and Intercept’s Ocaliva (obeticholic acid) is an option for patients who respond inadequately or are intolerant to UDCA. Although these drugs improve outcomes, they do not address the underlying autoimmune defect or meaningfully improve symptoms. This analysis of U.S. patient-level claims data explores the use of UDCA, Ocaliva, and numerous symptomatic treatments in both newly diagnosed and recently treated patients, providing insight into the current treatment paradigm for PBC.
Questions answered
- What patient shares do key therapies and brands garner by line of therapy in newly diagnosed PBC patients? What are the quarterly trends in prescribing among recently treated and newly diagnosed PBC patients?
- How has Ocaliva been integrated into the treatment algorithm, and what is its source of business?
- What percentage of PBC patients receive drug therapy within one year of diagnosis, and how quickly? What percentage of patients progress to later lines of therapy within one year of diagnosis?
- What percentage of PBC patients are treated with monotherapy vs. combination therapy? What are the most commonly used combinations?
- What are the product-level compliance and persistency rates among drug-treated patients?
Markets covered: United States
Key drugs: Ursodeoxycholic acid (UDCA), Ocaliva (obeticholic acid), antiepileptic drugs, antihistamines, fibrates, oral corticosteroids, oral immunosuppressants, serotonin receptor antagonists, selective serotonin reuptake inhibitors (SSRIs), statins
Product description
Treatment Algorithms: Claims Data Analysis provides detailed, quantitative analysis of the treatment journey and brand usage across lines of therapy and overall using real-world, patient-level claims data so that marketers can accurately assess their source of business, benchmark usage against competitors, and quantify areas of opportunity for their marketed or emerging brand.
Key feature
Dashboard featuring interactive visuals, easy navigation, and expanded analyses
Vidhi Khosla
Vidhi Khosla, M.B.A., M.Sc., Healthcare Research & Data Analyst, Infectious, Niche, and Rare Diseases. Ms. Khosla has several years of experience providing consulting, research, and analytic support in the pharmaceutical and healthcare domains and has worked in numerous therapy areas, including neurology, women’s health, metabolic disorders, and rare diseases. Prior to joining Clarivate, she worked at WNS Global Services and DelveInsight Business Research LLP. She earned her bachelor’s and master’s (gold medalist) degrees in biochemistry from Delhi University and Kurukshetra University, respectively, and an M.B.A. from Symbiosis International University.
Bethany A. Kiernan, Ph.D.
Bethany A. Kiernan, Ph.D., Director, Healthcare Research & Data Analytics, Infectious, Niche, and Rare Diseases. Dr. Kiernan manages and supports a team of analysts and managers producing both syndicated and custom market research analyses in a range of niche / rare indications as well as infectious diseases. Dr. Kiernan has extensive experience in market forecasting and broad knowledge across a range of CNS disorders, rare diseases, and infectious diseases. Her previous experience includes eight years of scientific research conducted at Tufts University School of Medicine, Case Western Reserve University, and the College of the Holy Cross. She earned her doctorate in neurosciences from Case Western Reserve University.