PREFER NEUROMUSCULAR DISORDERS (NMD)
An online learning to inform patients and assess patient preferences.
PREFER is a 5-year, IMI-funded, project that aims to investigate how and when patient preferences regarding the benefit/risk of drugs can be used in the medical product lifecycle. As part of the project, researchers are evaluating various research questions in clinical patient preference case studies.
One of the case studies being evaluated in PREFER is in the area of neuromuscular disorders (NMD). The NMD case study is led by Newcastle University together with patient organizations in the US, UK, Australia, New Zealand, and Canada. To support this case study, MindBytes developed interactive educational tools to inform patients on several aspects of the study:
- The study objective
- The preference survey methods, which included discrete choice experiment, best-worse scaling, and Q-methodology
High unmet need
Neuromuscular disorders (like myotonic dystrophy type 1 and mitochondrial disorders) are uncommon, serious, debilitating and are weakening the muscles. Due to the progressive nature and limited treatment options, the prognosis is unfortunately poor.
Limited studies
In the NMD field, patient preferences have not yet been studied extensively. However, the nature of these rare diseases and different priorities of stakeholders, make this a sensitive population for patient preference studies.
Compare preference methods
In cooperation with several international patient organizations, this study aims to make the comparison of different preference elicitation methods. This will allow the identification of suitable preference methods, also for populations with possible cognitive limitations.