I was honoured to be asked by PSC Patients Europe to represent PSC patients on 03 December 2018 at the ‘European Medicines Agency stakeholder interaction on the development of medicinal products for chronic non-infectious liver diseases (PBC, PSC, NASH)’ to share what is important when developing treatments for PSC.

In this important meeting, I gave an overview of the impact of PSC on patients, based on information provided to us in our Research and Treatment Surveys (2016), and their implications on clinical trial design.

Brief Summary

The patient community calls for collaboration: we would like to see researchers, academics, patients and industry working together and collaborating to develop and validate endpoints for PSC and move forward clinical trial design.

The PSC patient community is ready and willing to work with all stakeholders in a positive collaborative way. We are well-organised, trained and have expertise within our networks to support and add value to research. 

People with PSC are suffering with the risk of early death and poor quality of life. There are no medical treatment options so time is of the essence.  We urge investigators and sponsors to act now.

Our urgent goal should be to validate appropriate noninvasive endpoints, and let the focus not be solely on surrogates. It is critical always consider quality of life in clinical trials, and include Patient Reported Outcome Measures in endpoints.

Co-ordinated, pooled knowledge and true collaboration will accelerate PSC medicine development.

Ultimately, we want to live as well as possible with PSC, as long as possible.


Martine Walmsley, PSC Support 31 December 2018

Categories: Medicines and Treatments, Representing Patients
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