Consensus conference: surrogate end-points and real world evidence in PBC
One-day multi-stakeholder consensus conference with patronage from the main scientific societies in hepatology that aims to integrate the current knowledge in endpoints for therapy management in PBC, the role of real world evidence, and to provide a new framework for ongoing and future clinical trials.
The primary goals of this consensus conference are:
- to identify the key scientific, clinical, and regulatory needs to advance the development of reliable surrogate endpoints;
- to reach a clear consensus on how to use real world evidence to design confirmatory clinical trials in PBC.
Marco Carbone
Christophe Corpechot
Claudia Couto
Bettina E. Hansen
Gideon Hirschfield
David Jones
Cynthia Levy
Ana Lleo
Alejandra Villamil
José Willemse
Review current trial design and end points, and the need for agreement on best study designs.
Review the role of real world evidence in PBC and how to design and use prospective data
collection. Analyze the possibility of collaborative data sets; how can they be used agnostic to
a specific drug.
Understand the views of providers, regulatory agencies and patients about criteria for
defining clinical benefit and potential regulatory approval.
Develop a consensus statement based on multi-stakeholder input about best approaches to
ensuring a diverse range of therapies for patients.
Methodology
Pre-conference: Define working groups and leads for each group; define literature review for each group and summary of data with voting questions.
During the Conference: present findings of groups and allow for open discussion.
In principle, the panel would explore achieving a community consensus agreement, although in special circumstances a majority decision might prevail. In these cases, a minority report will be included in the document.
A final document capturing potential changes will be prepared for submission to Journal of Hepatology by the end of 2025.
Target audience
Health care providers (physicians, nurses, physician associates)
Statisticians
Real world data experts
Patients and patient representatives
Government/Regulatory Representatives
Industry
Pharmacists
Fellows/Trainees
