Primary Biliary Cholangitis – Unmet Need – Unmet Need – Primary Biliary Cholangitis (US/EU)

Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterized by inflammation, progressive damage, and destruction of the interlobular bile ducts, followed by cholestasis, which drives debilitating fatigue and itch. 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, and lower the risk of mortality. Ursodeoxycholic acid (UDCA) is the mainstay of treatment, and Intercept’s Ocaliva (obeticholic acid) is an option for patients who respond inadequately or are intolerant to UDCA, despite the risk of serious liver injury. Recently, PPAR agonists (Ipsen’s Iqirvo [elafibranor] and Livdelzi [Gilead’s seladelpar]) were approved for PBC. Although some of these drugs slow disease progression, addressing the underlying cause or showing meaningful improvement of symptoms remains a challenge. This analysis provides quantitative insights into key treatment drivers and gaps and opportunities in current unmet needs treatment, based on the insights of surveyed gastroenterologist and hepatologists.

Questions answered

• What are the key treatment drivers and goals in PBC treatment? How well do current therapies perform on these goals?
• What drug attributes are key influencers, and which of these have limited impact and which are hidden opportunities?
• Which unmet needs do physicians consider most crucial in the treatment of PBC?
• What trade-offs across different clinical attributes and prices are acceptable to physicians for a hypothetical PBC drug?

Markets covered: United States, France, Germany, United Kingdom

Primary research: Survey of 60 U.S. and 30 European gastroenterologists / hepatologists fielded in February 2025

Key drugs: Ursodeoxycholic acid, obeticholic acid, elafibranor, seladelpar, fibrates, oral corticosteroids, oral immunosuppressants

Product description

Unmet Need offers insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities, enabling you to:

• Evaluate clinical and nonclinical product attributes that influence treatment decisions through physicians’ weightings and analysis of stated vs. derived importance.
• Pinpoint areas of high unmet need by assessing current drug performance against key attributes and treatment drivers.
• Analyze market scenarios for different target product profiles using the TPP Simulator.

Key feature

Target Product Profile (TPP) simulator tool allows for customizable market simulations based on conjoint analysis. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.