PSC Support Patient Unmet Needs and Research Implications

PSC patient and Chair of PSC Support, Martine Walmsley, flew to Washington D.C. in March 2016, after being invited to present at an international conference hosted by the American Association for the Study of Liver Diseases (AASLD) and the US Food and Drug Administration (FDA).



You can view the PSC Support presentation on the AASLD website - fast forward to Section III Patient Experience at 24m33s.  The PSC Partners Seeking a Cure presentation is at 1m12s.

Martine said: “This was a key meeting because it brought together leading researchers, patients, regulatory bodies and pharmaceutical companies to discuss current unmet needs and research in what is a complex, progressive disease, with no known cause or effective treatment.

“It is vital for patients that any novel treatment is licensed and made available for use as rapidly as possible, and that requires early collaboration with regulatory authorities like the FDA and EMA to ensure that trial design and endpoints meet the exacting standards required.

“This meeting was a golden opportunity for the PSC field to come together to begin to agree those standards.”


PSC Support Surveys

Martine presented the results of two PSC Support surveys capturing more than 1,300 patient views on unmet needs and attitudes to research and treatments. She said: “When it comes to research, PSC patients are highly motivated – no one wants to know more about the disease than they do."

Thank you to everyone who took the time to complete our surveys on PSC research and PSC treatments. Patients' views informed the following report and will contribute to the direction of future PSC research. 

Download the PSC Support Patient Survey Report


Quality of Life

During her presentation Martine talked about the PSC-specific quality of life instrument for UK patients that PSC Support is developing with Dr Doug Thorburn from UK-PSC (the research group whose work is currently funded by an NIHR Rare Disease - Translational Research Collaboration grant). This attracted a great deal of interest and support from researchers, the FDA/EMA and pharmaceutical companies.

Martine explained: “There’s currently no tool that can adequately assess the quality of life for PSC patients, due in part to its complex nature, so this is a very important project for us. Without the expertise within UK-PSC, we would not be in a position to develop the tool, which will be funded by patient groups and industry.”

UK leading the way

“What was so encouraging to me as a patient was that there was a strong European presence of PSC researchers at this American-hosted conference, particularly British speakers, demonstrating the strength and influence of UK PSC research internationally: Dr Gideon Hirschfield, who leads UK-PSC, Dr Roger Chapman, Professor Dave Jones, Dr Stefan Hubscher.

“This is important because I believe the UK is leading the way both in our approach to PSC research and in patient engagement in PSC research.”


PSC Support presentation slides