The PBC Ireland patient registry: study protocol for a national platform on primary biliary cholangitis.

Abstract

Background: Primary Biliary Cholangitis (PBC) is a chronic, progressive liver disease. This paper outlines how a PBC patient registry was developed to address the gaps in evidence, care and policy affecting PBC patients in Ireland.

Objectives: The PBC patient registry is designed to collect patient-reported data regarding medical history, pruritus, fatigue and quality of life of PBC patients living in Ireland. This data can be used to identify care and treatment gaps and ensure that the PBC patient voice is included in new treatment decisions and healthcare policy. This real-world data will support further scientific and clinical research, drive patient-led advocacy efforts and facilitate collaboration with the liver disease communities globally.

Design: A patient-led, observational, registry-based study of PBC patients in Ireland.

Methods and analysis: Participants must have a PBC diagnosis and be 18 years of age or older. PROMs (patient-reported outcome measures) were administered through a secure web-based system. After providing electronic informed consent, participants completed online data collection forms. These included demographic information, medical history, standard of care and validated PROMs for fatigue, pruritus and quality of life. This was followed by an anonymous survey to collect usability and comprehensiveness metadata.

Ethics: The protocol was approved by TIER IRB Services, protocol ID: 5250715 (July 18th, 2025), which determined the study to be exempt as an observational, minimal-risk, non-interventional research activity involving anonymised patient-reported data.

Discussion: At the time of publication, 52 participants were registered in the patient registry, of which 40 completed all data collection forms. The results of the post-completion survey suggest high satisfaction across the domains of usability, comprehension, relevance, privacy/confidentiality and overall experience. The PBC patient registry shows that web-based PROMs can be used to collect real-world evidence from patients. Participants reported that the system was easy to use and comprehensive, confirming the usability and effectiveness of this approach. It also provides a starting point to identify healthcare and treatment gaps and facilitates the inclusion of PBC patients' voices in national and international health policy decisions that affect them.

Trial registration: Not applicable.