---
title: >-
  A Real-World Observational Cohort of Patients with Primary Biliary
  Cholangitis: TARGET-PBC Study Design and Rationale.
description: >-
  Explore this publication on real-world evidence: A Real-World Observational
  Cohort of Patients with Primary Biliary Cholangitis: TARGET-PBC Study Design
  and…
date: '2018-01-01'
category: Publications
tags:
  - Hepatology Communications
  - Abstract / Manuscript
  - R&D
  - Hepatology
  - PBC
canonical_url: >-
  https://www.pedestalhealth.com/resources/publications/hepatology-communications/a-real-world-observational-cohort-of-patients-with-primary/
source: Pedestal Health
license: © 2026 Pedestal Health. All rights reserved.
slug: a-real-world-observational-cohort-of-patients-with-primary
id: 6eyYBQnl0PHVkguvKFau0H
contentType: article
---

## Challenge

Drug development for PBC second-line therapies and anti-pruritic agents lacked a longitudinal, prospective real-world cohort with systematic PRO collection, deep clinical data, and biospecimen infrastructure spanning the full PBC disease spectrum in a diverse US population.

## Solution

The TARGET-PBC study was designed as a multi-site, longitudinal observational cohort of PBC patients at US academic and community sites, incorporating medical record abstraction, PBC-specific patient-reported outcome instruments, and a biospecimen repository.

## Impact

Establishing the TARGET-PBC registry created the foundational real-world infrastructure for all subsequent PBC natural history, treatment effectiveness, and patient burden studies, directly enabling partnerships with Intercept, GSK, and other sponsors developing second-line PBC therapies.

### [Julie M. Crawford](/people/julie-crawford/)