Unmet Needs and Research Implications

In March 2016, PSC Support presented the results of two PSC Support surveys capturing more than 1,300 patient views on unmet needs and attitudes to research and treatments at an international conference hosted by the American Association for the Study of Liver Diseases (AASLD) and the US Food and Drug Administration (FDA). Our report of the two surveys is called ‘Clinical need in PSC and clinically meaningful change: What is important to patients?’.

Thank you to everyone who took the time to complete our surveys on PSC research and PSC treatments. 


Key Results from the 2016 PSC Support 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


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.
Figure 1. The Most Difficult Part of Living with PSC
Figure 1. The Most Difficult Part of Living with PSC (PSC Support Research Survey 2016)


  • 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.


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.


Research Priorities

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


Attitudes towards the use of particular 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).
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.


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





PSC Support presentation slides


Download the PSC Support Patient Survey Report 'Clinical need in PSC and Clinically Meaningful Change: What is Important to Patients?'

Walmsley, M.‘Clinical need in PSC and clinically meaningful change: What is important to patients?’ PSC Support (March 2016) https://www.pscsupport.org.uk/unmetneeds