Any research that’s done with rare disease patients in mind, working to improve their treatment and experience is wonderful. However, when collaboration between both researchers and organizations working to support patients occurs, we make the most progress for individuals living with rare diagnoses. This is especially true when money becomes a barrier to research, and financially stable organizations are able to help.
The Muscular Dystrophy Association recently funded a study by the QVIA Institute for Human Data Science which aimed to uncover gaps in healthcare and indicate ways to improve and accelerate treatment for patients with neuromuscular diagnoses.
The MDA
The Muscular Dystrophy Association is an organization dedicated to helping accelerate research and patient care for over 40 different neuro-muscular diseases including muscular dystrophy. The ultimate goal of the organization is to find more cures for patients.
The MDA has created a data hub called MOVR, or NeuroMuscular ObserVational Research, which documents patient-reported data, data reported from medical professionals, and clinical and genome data. The hope is that this data set will help physician’s determine which patient will respond best to which type of treatment. Additionally, it could accelerate the development of new treatment options. MOVR will be implemented at 50 different MDA centers by 2019.
The QVIA’s study
The MDA’s database has become a crucial component for muscular dystrophy research. However, there was still a need to uncover where the gaps were in patient care. That’s where the QVIA’s study comes into play. With their collaboration with MDA, the goal was to not only identify these gaps, but find ways to fill them quickly. Here are some of their findings:
- Patient care is inconsistent between providers (especially for diseases with small patient populations). We need to identify universal guidelines to ensure all patients receive the best care.
- Technology which allows patients to monitor and report their condition remotely can improve their quality of life. It needs to be considered by more health care providers
- Many patients still struggle to find affordable care options and we need to reevaluate our financial approach to treatment
- Psychological symptoms affect many patients and need to be more thoroughly addressed.
The QVIA study also shows that data collection, like the MDA’s MOVR, is confirming the importance of genetic screening. It could mean earlier intervention and as a result, better treatment outcomes for patients.
With improvements in genetic testing, the development of new therapies, more organized collection of data, and increased collaboration between patient groups and researchers, we’re hopeful for the future of individuals affected by neuromuscular diseases.
“Expectations are high for advancing patient care and bringing disease-modifying therapies across a number of neuromuscular diseases to patients and their families.”
You can read more about the QVIA study and the MDA’s progress here.
