A recently published report on neuromuscular disease care outlines the current challenges and future opportunities in diagnosis and treatment. The report, titled ‘Understanding Neuromuscular Disease Care’, was funded by the Muscular Dystrophy Association. To read about the report’s findings in more detail, you can view the source press release here at MDA’s website. Alternatively, you can find the report in full at the IQVIA Institute for Human Data Science’s website here.
About Neuromuscular Disorders
Neuromuscular disorders are a group of conditions that affect the neuromuscular system, so may cause problems with a person’s muscles, the nerves that control the muscles, or the communication between the nerves and muscles. Amyotrophic lateral sclerosis, muscular dystrophy, and spinal muscular atrophy are all examples of neuromuscular disorders. According to MDA’s article, an estimated 250,000 patients and caregivers are impacted by neuromuscular diseases in the United States.
About the Report
The IQVIA Institute-released report used data, including a survey of ninety healthcare professionals who focused on care for patients with neuromuscular diseases, to inform their conclusions. Below are two of their key findings:
1. More than $46 billion is spent on neuromuscular diseases each year
According to the report, the total annual costs of neuromuscular disorders collectively are estimated to be over $46 billion. Although these conditions are rare, the cost of treatment is often high. Furthermore, many patients have to go through the ‘diagnostic odyssey’ – a long period of expensive and time-consuming testing before reaching a correct diagnosis. However, MDA’s article also emphasises that as genetic testing improves in effectiveness and cost, the time it takes patients to reach a diagnosis may be reduced.
2. Researchers are evaluating more than 190 candidates for neuromuscular diseases
According to the report, the number of molecules being clinically developed has increased by five-fold from about 20 in 2013 to around 100 in 2018. They attribute this in part to a better genetic understanding of neuromuscular diseases, as well as improving technology. Many of these drugs are small molecules, but gene therapies and antisense oligonucleotides also make up a significant proportion.