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 may benefit from similar treatment options. The sample will be stratified into three subgroups: two patient groups differentiated by age of symptom onset and one caregivers group. Each subgroup will be randomly assigned to complete two of three different preference elicitation methods at two different time points: Q-methodology survey, discrete choice experiment, and best-worst scaling type 2, allowing cross-comparisons of the results across each study time within participants and within elicitation methods. Additional variables such as sociodemographic, clinical and health literacy will be collected to enable analysis of potential heterogeneity. Ethics and Dissemination: This study protocol has undergone ethical review and approval by the Newcastle University R&D Ethics Committee (Ref: 15169/2018). All participants will be invited to give electronic informed consent to take part in the study prior accessing the online survey. All electronic data will be anonymised prior analysis. This study is part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (IMI-PREFER) project, a public-private collaborative research project aiming to develop expert and evidence-based recommendations on how and when patient preferences can be assessed and used to inform medical product decision making.
Highlights
Neuromuscular diseases (NMD) represent uncommon[1], serious and debilitating conditions; all progressive with poor prognosis and with limited or no treatment options available
This study aims to be a large patient preference study targeting rare disease groups through an international collaboration across different patient organizations
People affected by NMD diseases may have limited functional capacity to perform certain daily activities and often require caregiver assistance
Summary
Neuromuscular diseases (NMD) represent uncommon[1], serious and debilitating (i.e. muscle weakness) conditions; all progressive with poor prognosis and with limited or no treatment options available. MM are the result of either inherited or spontaneous mutations in either the mitochondrial genetic material or the nuclear genetic material that result in affected cellular mitochondria[4] In both diseases, different body tissues and organs may be affected in function or during development resulting in a multisystem and heterogeneous phenotype. People affected by NMD diseases may have limited functional capacity to perform certain daily activities and often require caregiver assistance. This adds to the complexity of studying the NMD population given that patients may rely significantly on caregivers for relevant care-related decisions[9,10]
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