In January 2018, MDA partnered with Edge Research, an established research firm with extensive expertise working with patient advocacy organizations, to conduct an objective and thorough study among the neuromuscular disease community. This survey, a first-of-its-kind for MDA, was called ONEVoice and comprised 50 questions that sought to identify the needs of individuals and families impacted by muscular dystrophy, amyotrophic lateral sclerosis (ALS) and related neuromuscular diseases.
The results are in.
More than 3,000 respondents, including 2,430 adult patients and 932 caregivers and family members, shared information about their day-to-day needs, experiences managing disease, treatment benefits and risks, and support and information sources. The exchange revealed a number of critical needs within the community and opportunities to meet them, all of which will inform MDA’s work in 2019 and onward.
Key findings include:
- Genetic testing is under utilized. Nearly a third of adult patients say they have not had their diagnosis confirmed through genetic testing. Genetic testing not only is used to confirm a disease but also can help predict disease course, identify recurrence risk, facilitate enrollment in clinical trials and assist in developing a targeted treatment plan.
- Patients want to participate in clinical trials. Three-quarters of surveyed neuromuscular patients have never participated in a clinical trial, but the interest is high. Nearly 8 in 10 survey respondents say they are interested in finding a clinical trial for which they may be eligible.
- Access to care is a major pain point. More than 3 in 4 respondents have concerns about access to health care and/or health care professionals with expertise in neuromuscular disease.
- Independence and mobility are major concerns for 64 percent of the neuromuscular community and were the top concerns of those surveyed.
Read the full ONEVoice survey report online here.