A study protocol for quantifying patient preferences in neuromuscular disorders: a case study of the IMI PREFER Project.

ABSTRACT: Objectives: Patient preference studies are increasingly used to inform decision-making during the medical product lifecycle but are rarely used to inform early stages of drug development.  The primary aim of this study is to quantify treatment preferences of patients with neuromuscular disorders, which represent serious and debilitating conditions with limited or no treatment options available. Methods: This quantitative patient preferences study was designed as an online survey, with a cross-over design.  This study will target two different diseases from the neuromuscular disorders disease group, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM). Despite having different physio-pathological pathways both DM1 and MM manifest in a clinically similar manner and

SUBMITTER: Jimenez-Moreno AC 

PROVIDER: S-EPMC8356266 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature