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A really exciting development in PBC is the emergence of new treatments for symptoms. There is real progress being made in itch, fatigue and “brain fog”.
Before treatments can be used in practice, they need to undergo clinical trials to show that they are effective and safe. The results of trials inform the decisions of regulators such as the FDA in America and the MHRA in the UK as to whether the treatments can be used in practice.  Many PBC Foundation members have taken part in clinical trials over the years, contributing to the development of new treatments. Ocaliva, now available on the NHS and around the world was shown to work in clinical trials which were delivered in part in the UK.
Whereas many trials in PBC look at blood tests, when studying treatments for symptoms we need ways to reliably and meaningfully transform the patient experience (the severity of symptoms and their effect on quality of life) to data we can analyse.  Once we know we are measuring this properly, we can more honestly and clearly test the benefit of a new drug. This is a requirement before a new drug can be approved. In practice, this process involves an interview with the study participant before and after the treatment. The impact of symptoms described in the interview are then scored, and the scores before treatment are compared with the scores at the end of the treatment period. This obviously runs alongside peoples’ own assessment of their symptoms. The interviews follow a standard script, to ensure that all areas are covered, but are designed to allow people to express their symptoms in their own way.
The interview-based approach to assessing symptoms (called CGI or Clinical Global Impression) has been used in a number of diseases but is new to PBC. It is a really important step, however, towards getting new symptom treatments into practice. The aim of this survey is to find out what people think about the approach, and whether they would be comfortable with it in practice. We are mindful that these kind of “standard” interviews can be long and even seem repetitive, because all study participants must be interviewed the same way throughout the study. It is possible that standardizing the interviews may best be accomplished by having a single expert (not your own caregiver) interview everyone, or possibly by video or audiotaping the interviews. But before we begin, we need to understand how you would feel about that. This information will be incredibly important as we move forward to better symptom treatments in PBC.

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* 1. The approach involves an interview about symptoms lasting about 30-45 minutes done before and after a trial. Which of the following best describes how you would feel about that?

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* 2. On balance, how would you feel about a clinical trial that needed interviews in this way?

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* 3. There are different options as to WHO does the interview and there are advantages to each. These include a doctor or nurse from your own clinical team who would know you, a doctor or nurse form another team who wouldn’t know you and a trained interviewer who wouldn’t know you.

Which interviewers would you be happy to be interviewed by? PLEASE TICK ALL THAT APPLY

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* 4. There are different options as to HOW we do the interview. The options are to do it in person (i.e. sitting in the same room) or to do it on-line (e.g. “zoom” or another platform). Which of these answers best describes how you feel about an ON-LINE interview? Note that in an on-line interview, the interviewer wouldn’t know your name or anything about you. Please tick one

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* 5. The interview will need to be assessed/scored as part of the trial. One option would be for the person who does the interview to score it. This presents the problem of making sure there is consistency in the scoring between patients and between hospitals. Another approach would be to record the interviews. The approach involves an interview about symptoms lasting about 30-45 minutes done before and after a trial. Which of the following best describes how you would feel about that?

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* 6. On balance, how would you feel about a clinical trial that needed on-line interviews that were recorded for later scoring?

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* 7. If we do use recorded interviews that are then scored later, we will need to record some interviews to help train the people who score (and make sure that their assessments are consistent). To do this, we will need a set of patient interviews that can be shown to a number of doctors and nurses who would see your face but not know your name or anything else about you. Once people had been trained these recordings would be deleted.

In principle would you be prepared to volunteer for a recorded training interview?

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* 8. What is your country of residence?

0 of 8 answered
 

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