The voice for patients in PSC research

PSC Support is leading the way in driving and supporting PSC research

Voice for patients

PSC Support is the voice for PSC patients in research and drug development

PSC Support represents people with PSC nationally and internationally in our work to speed up an effective treatment for people with PSC.

We evidence the views we provide. Of note, two PSC Support surveys capturing more than 1,300 respondent views on unmet needs and attitudes to research and treatments have been instrumental in understanding what people affected by PSC need and want. Our report of the two surveys is called ‘Clinical need in PSC and clinically meaningful change: What is important to patients?’35

In March 2016, PSC Support represented all PSC patients international conference hosted by the American Association for the Study of Liver Diseases (AASLD) and the US Food and Drug Administration (FDA).

In December 2018, PSC Support presented the patient perspective on PSC medicine development with PSC Patients Europe at the 2018 European Medicines Agency multi-stakeholder meeting on PSC, PBC and NASH research.

Thank you to everyone who takes the time to complete our PSC Support surveys. Your views are making a difference.

Key Interim Results from the PSC Support Research and Treatment Surveys

PSC Patients are Engaged

  • PSC patients are interested and eager to participate in research studies
  • Patient-centred research design will help recruitment and retention of participants
  • Patient Reported Outcome Measures should be incorporated into trial design where relevant

Clinically Meaningful Change

  • To patients, clinically meaningful change means:
    • an improvement in quality of life that patient can detect
    • the relief of symptoms (e.g. pain, fatigue, itch) that patient can detect
    • a change in something (e.g. biomarker) directly associated with the disease process that has a convincing ability to:
      • prolong life
      • reduce PSC complications
      • prevent or reduce occurrence of rPSC
      • reduce infection
      • prevent cancer
      • predict (risk of) progression of disease

What is the Most Difficult Part of Living with PSC?

  • When asked to describe the most difficult part of living with PSC, many respondents gave more than one issue. 74% of respondents felt that the emotional impact was the most difficult aspect (n=340) (see Figure 1)
    • 60.4% of the emotional impact responses were around uncertainty about the future
    • 28.2% of the emotional concerns reported in our survey were around helplessness: frustration at having an ‘untreatable disease’, that is not only untreatable, but poorly understood in terms of cause and any kind of prognosis.
    • 11.4% of the emotional concerns reported were about perceived inadequate clinical care or monitoring, and the burden of hospitalisation.

Most difficult aspect of PSC

Figure 1. Reported difficulties and impact of living with PSC


  • When considering the most difficult aspect of PSC, 20% of respondents cited concerns around the social impact of PSC.
    • The social concerns expressed were mostly (38%) around a lack of understanding from others, or rather, the effects of having a mostly invisible illness, and the same proportion noted how difficult it was to stand by and watch someone else suffer or die.
    • 19% of the concerns were around social isolation and 6% were around being a burden to others.

What are the Most Common Symptoms in PSC?

  • When asked to indicate the symptoms they had experienced in the previous four weeks, the most common symptoms indicated by patients (n=317) were: 
    • fatigue (78%)
    • pain (54%)
    • itching (51%) 
  • 5% of respondents indicated that they had had no symptoms in the previous four-week period.
  • More than one third (32%) reported that they had felt depressed. Although this is a self-reported symptom, it demonstrates that PSC comes with a heavy emotional burden for some patients.

What are the Research Priorities for Patients?

  •  Patients and their families provided 530 questions when asked to state 'unanswered questions' in PSC research. By far the most common responses (85%) were fundamental, sense-making questions about PSC (see Figure 2).

Research Priorities

Figure 2. Research Priorities (PSC Support Research Survey 2016)

What do Patients Think About the Use of Invasive Procedures in Research?

  • We asked patients what clinical procedures they had undergone and which they would be prepared to undergo for research. (n=317)
  • Patients’ attitudes are positive about invasive procedures for research, with around half prepared to undergo a colonoscopy (58%), liver biopsy (52%), gastroscopy (52%) or ERCP(55%) (2016 Research Survey, see Figure 3).

Participation in Research and Procedures

Figure 3. Participation in Research and Procedures (PSC Support Research Survey 2016)

A word of caution: biopsies...

  •  In our PSC Support Treatments Survey, we asked patients, 'If you were taking part in a research trial, would you be prepared to have one or more biopsies?(n=618, see Figure 4). This time, we found an even higher proportion (66%) was prepared to undergo biopsy for research and this figure held true even for patients who had previously had a biopsy.
  • PSC patients are informed and alert to the risks and benefits of procedures, and the need for PSC research. Patients want to help research and are aware of the costs to them of having biopsies. It should not be concluded that PSC patients want biopsies as part of their routine care; they don’t. PSC patients are engaged and willing to take part in research but not at any cost.
  • We should be crystal clear about this: no one likes a biopsy or takes one lightly.
    • biopsies should only be part of the study protocol if it is absolutely necessary (or when they are part of existing clinical care), when there is no other way of obtaining the desired information;
    • participants undergoing biopsies should be administered adequate sedation and such procedures be conducted by experienced practitioners only;
    • there should be a convincing and important reason for the use of biopsies in research.

Appetitie for biopsies in research

Figure 4. Participation in Research and Procedures (PSC Support Treatments Survey 2016)

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