Check SCREEN READER MODE to make this survey compatible with screen readers.
PBC Foundation - Clinical Trial Preferences Survey
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.