Abstract
Introduction:
At present no single symptom appears to be favoured in choosing primary outcomes for dementia with Lewy bodies (DLB) trials, nor are the perspectives of people affected by DLB reflected in their design. The aim of this study is to elicit the preferences of DLB patients and their care partners with respect to the DLB symptoms that they would most like to see improved upon by a potential therapy. We will do so using two complimentary health economic approaches in a single online survey: a best-worst scaling (BWS) exercise and a discrete choice experiment (DCE).
Methods:
Using global voluntary sector networks, we will recruit 100 individuals who either have a diagnosis of DLB or who are a care partner for an individual with DLB. The BWS and DCE will be applied together in a self-administered online survey. Both will be informed by evidence from a scoping review of existing literature and piloted in person with a group of local volunteers.
In the BWS, participants will be presented with a series of choice sets of three symptom domains and asked to determine which domain they consider the most and least important to treat.
In the DCE, participants will be presented with a scenario and asked to choose their preference from one of two hypothetical treatments, each with different characteristics (such as target symptom and side effect profile).
Expected Results:
We will determine the relative importance of DLB symptoms and the potential trade-offs made in benefit-risk decisions related to treatments. Our findings will inform choice of primary and secondary outcomes in DLB trials. They will inform our understanding of the levels of risk and benefit individuals will tolerate, which is important as trials of potentially disease modifying agents, such as amyloid therapies, are pursued.