Finding out what patients with Neuromuscular Disorders prefer!

2020-05-20

PREFER is conducting a patient preference study about what kinds of treatment patients with two hereditary neuromuscular disorders prefer. Together with eleven patient organisations, we are now actively recruiting respondents for an online survey. Anyone over 18 with either myotonic dystrophy type 1, or mitochondrial disorders is welcome to participate!

We are proud to announce the launch of the online survey for the PREFER Neuromuscular Disorders (NMD) Case Study! We ask patients and caregivers about how they do trade-offs between the risks and benefits of different treatments. The results will help us understand how and when to patient’s preferences should be included in medical product decision-making: from development, through approval and reimbursement decisions by authorities, bringing products to market and monitoring safety after.

There is very little research on the preferences of patients with Myotonic Dystrophy and Mitochondrial Disorders. There is no cure. These are progressive and debilitating rare diseases, where patient preference information can give us a better understanding of the kinds of unmet health needs these patients have.

Direct engagement of members from academia, industry and patient organisations has been the biggest drive of this case study. This also makes it a promising example of how we can conduct patient-centric research.

Rare disease affect a small portion of the population. Patients with these conditions are few and spread across the globe. To be able to reach enough patients to get the answers we need, we need international collaboration.  We are very proud to say that 11 patient organisations have joined forces with the PREFER project to support the dissemination and delivery of this study. With their help, we will be able to say something about what patients with myotonic dystrophy type 1, and mitochondrial disorders think is important, and help further research on rare diseases!

The survey is in English, which is why we are engaging with patient organisations from English speaking countries only. We thank them all for their support!

Are you affected by or caring for someone with Myotonic Dystrophy and Mitochondrial Disorders? You might be eligilble for our survey! Have a look at the PREFER survey on Neuromuscular disorders.

By Anna Holm & Josepine Fernow