PSC Quality of Life Measure

The UK-PSC Quality of Life Measure (UK-PSC-QoL)

Overview

We are developing the first patient-centred, PSC-specific Quality of Life measure for people with PSC, and is a critical tool for improving the wellbeing of people with PSC.

A Quality of Life measure is a questionnaire, and is important because people with PSC experience many quality of life and wellbeing issues, yet there is no validated way to measure them. Without a PSC-specific quality of life measure, we cannot say whether a new drug improves quality of life, or indeed makes quality of life worse. In the absence of a reliable quality of life measure that addresses issues important to people with PSC, researchers have no choice but to use generic questionnaires, or worse, quality of life questionnaires developed for different diseases. This is not good enough.

The steering committee overseeing the entire development comprises: Professor Douglas Thorburn (UCL/Royal Free, London), Professor Shivani Sharma (Aston College/ University of Hertfordshire) PSC Partners, British Liver Trust and PSC Support.

This research is funded by PSC Support and the British Liver Trust and is now in Stage 2 of its development.

Ryan James

Our Quality of Life Measure is being delivered by Ryan James as his PhD fellowship at the University of Hertfordshire. With an undergraduate degree in Psychology, a Masters in Research Methods in Clinical Psychology, and experience as a senior research assistant focusing on health inequalities before commencing the PhD, Ryan is focused on testing the validity of the questionnaire and supporting its translation into multiple languages. The development of the measure is expected to be completed by mid-2027.

History

July 2016

We are developing a measure to assess the quality of life of people living with primary sclerosing cholangitis (PSC), under the supervision of Dr Douglas Thorburn and academics in the Marie Curie Palliative Care Research Department (MCPCRD) at University College London.

This is an exciting, ground-breaking project, aiming to produce the first validated quality of life measure for people living with PSC, with real scope to influence care and treatment. There is a critical need for such a measure ^a, since PSC patients encounter many quality of life related issues, and it is important to develop treatments for PSC that improve quality of life (see recent PSC Support survey results on unmet needs and research implications). Equally, it is important that novel therapies for PSC do not have a negative effect on patients’ quality of life.

PSC Support and the British Liver Trust are therefore developing a PSC-specific quality of life measure for UK patients. The funding for this UK PhD project has been provided by the British Liver Trust, to fund a student to undertake the work in the UK and PSC Support. The initial funding became available in a bequest from Mary and Bill (pictured below) who specified that they wanted their money to be used towards an important PSC study. The principal investigator is Dr Douglas Thorburn, supported by experienced researchers from the UCL Marie Curie Palliative Care Research Department. The UK project is expected to be completed in 2018/9, and the measure may then proceed to international validation, hoping for completion by 2020/1.

Our vision for a Quality of Life measure was presented at the March 2016 AASLD/FDA Conference on Trial Design and Endpoints for Clinical Trials in Adults and Children with primary sclerosing cholangitis and has received international interest from leading researchers and key regulatory authorities (FDA and EMA). The proposed measure will be key in measuring the quality of life for people with PSC and will be suitable for multicentre international clinical trials, to evaluate patients’ responses to new treatments for PSC.

PhD candidate Elena Marcus was appointed July 2016, and began work to develop the measure in October 2016.

Martine Walmsley, Chair of Trustees for PSC Support said, 'We are delighted to have attracted a researcher of Elena’s calibre to join our team. During her interview, Elena demonstrated the passion, ability and experience necessary to ensure the delivery of a truly high-quality PSC QoL measure. This project is critical and we're excited it's moving forward.'

October 2016

PSC Partners joins the team.

November 2017

The first stage the first phase of interviews/focus groups begins.

January 2018

Video released updating on progress of project.

October 2018

Phase 2 completed and Phase 3 began.

October 2019

Elena is now analysing data from the second stage of the study.

We now have a provisional quality of life questionnaire which is ready for international development and validation.

July 2022

After pausing due to the COVID pandemic, a description of the methodology used to develop the PSC quality of life measure was published.

January 2024

Ryan James (University of Hertfordshire) is undertaking the second part of the development of the quality of life measure.

Development Stage 1

Phase 1

The first stage of the Quality of Life Measure was delivered by Dr Elena Marcus.

Quality of life issues relevant to people living with PSC in the UK were identified and a provisional questionnaire was developed.

Nearly 400 issues were identified from PSC patient survey data and a systematic literature review of existing quality of life measures.

The relevance and importance of these issues were explored through interviews and group discussions with people with PSC and with experienced clinicians, and the list of issues was reduced to 89. Thank you to everyone who helped with this phase of the study.

Phase 2

Elena then constructed these issues into 83 questionnaire items for the provisional measure, the UK-PSC-QoL.

Phase 3

Elena investigated the functioning of the provisional questionnaire and its items with people living with PSC in the UK. This included looking at participants’ perceptions around acceptability, clarity, relevance and redundancy of the items and the measure.

Development Stage 2

Publications

Marcus E, Stone P, Thorburn D, Walmsley M, Vivat B. Quality of life (QoL) for people with primary sclerosing cholangitis (PSC): a pragmatic strategy for identifyin g relevant QoL issues for rare disease. Journal of Patient-Reported Outcomes. 2022 Dec;6(1):1-2. https://doi.org/10.1186/s41687-022-00484-5

Marcus E, Stone P, Krooupa AM, Thorburn D, Vivat B. Quality of life in primary sclerosing cholangitis: a systematic review. Health and quality of life outcomes. 2021 Dec;19(1):1-7.

Marcus E, Thorburn D, Stone P, Vivat B. Quality of life (QoL) for people with rare diseases: Recruitment challenges and consequences in a study pilot-testing the UK-PSC-QoL, a provisional QoL tool for people with primary sclerosing cholangitis (PSC) in the UK. 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020). Orphanet J Rare Dis 15, 310 (2020). https://doi.org/10.1186/s13023-020-01550-1

Marcus E. Developing and pre-testing a measure of quality of life for people with primary sclerosing cholangitis (PSC) in the UK (Doctoral dissertation, UCL (University College London)). May 2020.