EASL-AASLD Delphi consensus statement on surrogate endpoints and real-world evidence in primary biliary cholangitis - PubMed
3 hours ago
- #Regulatory Approval
- #Primary Biliary Cholangitis
- #Delphi Consensus
- Conditional approval of three second-line therapies in PBC faces challenges for full regulatory approval due to reliance on unvalidated biomarkers, limited integration of real-world evidence, and lack of standardized patient-reported outcomes.
- EASL and AASLD convened an international multi-stakeholder panel using a modified Delphi process to review evidence, identify research gaps, and create a framework aligned with regulatory expectations.
- 62 panellists, including clinicians, regulators, industry partners, and patient representatives, participated in online meetings, an in-person conference, and two Delphi voting rounds with high response rates.
- The Delphi process resulted in consensus on 16 statements and 42 recommendations across three thematic domains, providing a pragmatic framework to support new therapy development and approval in PBC.