Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Cost of Illness for Neuromuscular Diseases in the United States

MDA and the Lewin Group have completed a total cost of illness study for three common neuromuscular disorders. This is the first comprehensive study of this kind to be published in the United States, establishing the direct medical and nonmedical costs, as well as the loss of income associated with these diseases. The findings of this study were published in the journal Muscle and Nerve, and the paper and the complete report are available below. This data may affect drug development, as well as social and economic policies.

What is a cost of illness study?

A cost of illness study establishes the costs associated with having a specific condition, both on an individual annual basis and to society as a whole. MDA’s study was a comprehensive study, including both medical and nonmedical costs, and loss of income to families due to caregiving, loss of opportunities for education, etc.

Which diseases were studied?

MDA’s study was designed to study amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), myotonic muscular dystrophy (MMD, or DM) and early- and late-onset spinal muscular atrophy (SMA). These diseases were selected for a number of reasons:

  1. Therapy development is advanced in some of these fields.
  2. Population sizes are large enough and uniform enough for a statistically significant number to be calculated.
  3. We could separate the conditions well enough by medical codes to get useful values for medical costs. 

Unfortunately, the data for SMA did not prove to be useful due to small sample sizes and the inability to distinguish between early- and late-onset disease, in which the costs are very different.

What did the study find?

The per-patient annual costs for ALS, DMD and MMD are as follows:

Disorder Medical Costs Nonmedical Costs Lost Income Total
ALS $31,121 $17,889 $14,628 $63,692
DMD $22,533 $12,939 $15,481 $50,953
MMD (DM) $17,451 $5,157 $9,628 $32,236

The annual costs for ALS, DMD and MMD for U.S. society as a whole, based on the best available statistics on prevalence of the diseases in the U.S., are as follows:

Disorder Total National Cost
ALS $256-$433 million
DMD $362-$488 million
MMD (DM) $448 million

 The total estimated cost of illness to the nation for ALS, DMD and MMD combined is $1.07 to $1.37 billion per year. The authors note that this estimate is likely to be conservative.

How is cost of illness data used?

Different stakeholders will use this data in myriad ways. A family may be interested to see what the average cost is in comparison to their own experience. A researcher may use the number to justify why his or her research should be funded, relative to research into another disease. A drug development company might use it to justify why an insurance company should reimburse a newly developed drug, and organizations like MDA will use it to lobby the government for allocation of more resources to alleviate the costs absorbed by individuals and families living with a particular condition.

Why is this important?

Putting a number to the impact of a disease allows that impact to be compared to other diseases. This is extremely important in competing for limited resources, such as allocation of research dollars, allocation of disability support, etc. These numbers show that the cost of living with ALS, myotonic dystrophy and Duchenne muscular dystrophy are all of a similar magnitude in comparison to diseases such as multiple sclerosis and Parkinson’s disease, which often get more federal attention.

What will MDA do next in this arena?

MDA will use this data in various ways through our advocacy program to lobby for increased allocation of resources to our disease areas. We also will share the information with researchers and companies in order to help them to make a case for further involvement in the area. In addition, more detailed studies are expected to follow up on the data collected here.

What are the caveats behind this data?

This data has a large number of caveats associated with it, which make the numbers less precise than we would like. However, every issue with the data that we have detailed in the paper would make the true cost of these diseases higher than listed, so we see this as a minimal cost — with the true cost of these diseases being much higher. The study also cannot take into account non-financial costs (e.g., emotional impact, etc.).

Can I see the data in detail?

The full report, written by the Lewin group with input from MDA, is available, as well as the summary paper published in Muscle and Nerve. The summary paper is the pre-peer reviewed version of the final article Cost of illness for neuromuscular diseases in the U.S., co-authored by Jane Larkindale, MDA's vice president of research. The final article, published July 8, 2013, is available with a subscription.

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