Primary Biliary Cholangitis | Unmet Need | US/EU | 2024
Primary biliary cholangitis (PBC) is a rare autoimmune liver disease characterized by inflammation, progressive damage, and destruction of the interlobular bile ducts; this process is followed by cholestasis, which drives debilitating fatigue and itch. 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 mainstay of 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 disease or meaningfully improve symptoms. This analysis provides quantitative insights into key treatment drivers and goals and current unmet needs, based on the insights of surveyed gastroenterologists / 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, which have limited impact, and which are hidden opportunities?
- Which unmet needs do gastroenterologists / hepatologists consider most important in the treatment of PBC?
- Based on conjoint analysis and TPP simulation, what trade-offs across different clinical attributes and prices are acceptable to U.S. and European physicians for a hypothetical PBC drug?
Markets covered: United States, France, Germany, United Kingdom
Primary research: Survey of 61 U.S. and 30 European gastroenterologists / hepatologists fielded in March 2024
Key drugs: Ursodeoxycholic acid, Ocaliva, antiepileptics, antihistamines, fibrates, oral corticosteroids, SSRIs, statins, oral immunosuppressants, serotonin receptor antagonists
Product description
Unmet Need supports clinical development decisions by identifying key attributes and assessing areas of unmet need for a specific disease or subpopulation. Based on surveys with U.S. and European physicians, this report provides insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities. One market scenario is profiled in detail by Clarivate experts, and additional customized market scenarios can be evaluated with the corresponding TPP Simulator.
Key feature
The Target Product Profile (TPP) Simulator tool allows for customizable market simulations based on conjoint analysis that depicts how physicians make decisions based on actual behavior rather than opinion. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.
