Exploring participation and engagement in clinical trials in cystic fibrosis (EXPECT)
File(s)
Author(s)
Dobra, Rebecca Anne
Type
Thesis
Abstract
Patient-centred clinical trial design has been shown to improve recruitment and retention, increase participant satisfaction, better meet participants’ psychosocial needs and enable a more diverse cohort of patients to enter trials. This results in optimised time and cost efficiency of research, reduced trial failure rates and ensures research cohorts are maximally representative of the actual clinical population, ultimately allowing safe and effective treatments to reach clinic more quickly. To make trials more patient-centred, the views of invested stakeholders must be sought and incorporated into trial design and delivery.
Cystic fibrosis (CF) is a multisystem genetic disease. Despite substantial shifts in treatment paradigms secondary to novel drugs, so called modulators, CF still carries a significantly reduced life-expectancy. Clinical trials are needed to improve prognosis and quality of life for everyone with this disease.
In my thesis, I present three projects. The first is a systematic literature review which maps the factors influencing participation and engagement in trials in any disease. The review aimed to develop a framework to guide researchers towards themes to consider when conducting trials, and to provide the foundation for later steps of my PhD. The second, a Delphi study, aimed to identify which of the factors identified through the literature review are most relevant to people with CF. The third is a discrete choice experiment, which I used to quantify preferences of people with CF for specific clinical trial features. In the final chapter, I amalgamate the findings to propose key ‘Do’s and Don’ts’ to guide the rational design of patient-centred trials in CF. Incorporating these suggestions into practice and continuing to seek out patient views as the landscape evolves should help to optimise patient-centred trials. This will continue to support the successful design and delivery of CF trials to improve outcomes for this life-limiting disease.
Cystic fibrosis (CF) is a multisystem genetic disease. Despite substantial shifts in treatment paradigms secondary to novel drugs, so called modulators, CF still carries a significantly reduced life-expectancy. Clinical trials are needed to improve prognosis and quality of life for everyone with this disease.
In my thesis, I present three projects. The first is a systematic literature review which maps the factors influencing participation and engagement in trials in any disease. The review aimed to develop a framework to guide researchers towards themes to consider when conducting trials, and to provide the foundation for later steps of my PhD. The second, a Delphi study, aimed to identify which of the factors identified through the literature review are most relevant to people with CF. The third is a discrete choice experiment, which I used to quantify preferences of people with CF for specific clinical trial features. In the final chapter, I amalgamate the findings to propose key ‘Do’s and Don’ts’ to guide the rational design of patient-centred trials in CF. Incorporating these suggestions into practice and continuing to seek out patient views as the landscape evolves should help to optimise patient-centred trials. This will continue to support the successful design and delivery of CF trials to improve outcomes for this life-limiting disease.
Version
Open Access
Date Issued
2022-07
Date Awarded
2023-03
Copyright Statement
Creative Commons Attribution NonCommercial Licence
License URL
Advisor
Davies, Jane C
Madge, Susan
Elborn, Stuart
Publisher Department
National Heart & Lung Institute
Publisher Institution
Imperial College London
Qualification Level
Doctoral
Qualification Name
Doctor of Philosophy (PhD)