Assessing symptoms in PBC
Research type
Research Study
Full title
Assessing Symptoms in Primary Biliary Cholangitis: CGI-PBC CGI-S Validation and Implementation in Primary Biliary Cholangitis
IRAS ID
316425
Contact name
David Jones
Contact email
Sponsor organisation
PBC Foundation
Duration of Study in the UK
0 years, 11 months, 31 days
Research summary
Patients with primary biliary cholangitis experience symptoms that can impact on their quality of life. Real progress is being made on understanding why these symptoms arise and how we can potentially treat them better. In order for any future therapy to be licensed for use in patients we need to able to confirm, to the satisfaction of regulatory bodies, that it is effective in improving symptoms. This needs us to have tools that can measure benefit. One approach that is seen an highly desirable by regulators is called "Clinician Global Impact Assessment"; an approach where interviewers discuss symptoms with patients and rate the severity of the impact (this approach runs alongside patient reported impact scoring systems). Whereas we have a well validated patient reporting system (the PBC-40) we currently lack a validated Clinical Global Impact Assessment tool for PBC. The lack of this could limit the development of better therapies.
We have developed an outline tool but, before we can use it in a trial, need to validate it (make sure that different people rating the same interview come up with the same score). The aim of the study outlined here is to do this validation.
In a unique patient involvement model PBC patients who belong to the PBC Foundation (a national patients group) will be invited by the Foundation to take part in on-line interviews that will be rated (or scored) for symptom severity by both the interviewer and a panel of other raters (all clinicians familiar with PBC). The degree of agreement in scores will be the key outcome. Where there is disagreement, the raters will discuss the video and reach a consensus. This will be used to inform the approach to trial use.
The most informative interviews will then be used to develop a training pack for raters.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
22/SW/0163
Date of REC Opinion
7 Nov 2022
REC opinion
Favourable Opinion