Primary Biliary Cholangitis (PBC)


TARGET-PBC represents a unique opportunity in the study of rare diseases. By fostering collaborations between academic and community centers, TARGET-PBC will reveal the true natural history of the disease and the response to available therapies in various subgroups, many of which have been underrepresented in phase 3 trials. TARGET-PBC will answer crucial questions involving the safety and efficacy of approved and off-label therapies used in the real world and will identify at-risk populations in need of novel therapies. Cynthia Levy, MD

In the United States alone, research has shown that approximately 65 out of every 100,000 women, as well as 12 out of every 100,000 men, have PBC. The exact cause of this disease is still unknown, according to the U.S. Department of Health and Human Services, and most individuals have no symptoms when first diagnosed. 

As incredible progress continues to be made in the PBC research field, the TARGET-PBC study was launched to further contribute to the field by generating real-world evidence and disease insights. TARGET-PBC is a 5-year longitudinal, observational study of patients with PBC designed to specifically address key clinical questions that conventional trials have been unable to answer to date. The study also aims to further enhance the understanding of this disease and support the identification of suitable treatment strategies.

Partners can query the TARGET-PBC database comprised of up to 8 years of longitudinal patient journeys and access biospecimen samples linked to validated clinical data to conduct further research and genomics analysis.

Study Goals:

  • Evaluate PBC treatment regimens being used in clinical practice

  • Examine populations under-represented in phase II-III clinical trials

  • Examine biochemical response and its association with long-term outcomes

  • Estimate adverse event frequency and severity and describe management practices


October 2016


Enrollment Goal:

Up to 1,500

TARGET-PBC Steering Committee

Cynthia Levy, MD (Chair)
Assistant Director of Schiff Center for Liver Diseases & Program Director of the Transplant Hepatology Fellowship, Miami, FL

Christopher Bowlus, MD
Division Chief of Gastroenterology and Hepatology, Sacramento, CA

Elizabeth Carey, MD
Associate Professor of Medicine, Hepatology, Phoenix, AZ

Marlyn Mayo, MD
Associate Professor, Gastroenterology, Dallas, TX

W. Ray Kim, MD
Professor of Medicine, Gastroenterology and Hepatology, Stanford, CA

Interested in launching a new disease registry?

Target RWE has developed a turnkey solution to rapidly activate disease agnostic registries to support partners.