MDA Study Reveals ‘Cost of Illness’ for ALS, DMD, MMD

MDA has supported a total cost-of-illness study for three common neuromuscular disorders that may affect drug development and social and economic policies

Chronic, debilitating diseases like amyotrophic lateral sclerosis and muscular dystrophy take a toll on families and society that goes beyond medical expenses. Putting a dollar amount on this toll will help guide the drug development industry and policymakers.
Article Highlights:
  • MDA has funded the first-ever study in the U.S. estimating the annual cost of illness in terms of medical costs, nonmedical costs and loss of income for families affected by amyotrophic lateral sclerosis, Duchenne muscular dystrophy and myotonic muscular dystrophy.
  • The study has implications for companies seeking to develop drugs for neuromuscular disorders; for the design of social and economic policies that pertain to families with these disorders; and for individuals seeking insurance or government program coverage of treatments for ALS, DMD or MMD.
  • "This valuable study provides us with a way to translate the adverse effects of our diseases into dollar terms, the universal language of decision-makers and the policy arena," said Jane Larkindale, MDA vice president of research.
  • In a two-minute video, MDA Senior Vice President of Advocacy Annie Kennedy explains the value of the study for individuals.
by Margaret Wahl on August 14, 2013 - 4:00am

"We now have numbers to point to," said Annie Kennedy, MDA's senior vice president of advocacy. "It's not just a back-of-the-envelope calculation anymore. It's real data."

MDA has funded the first comprehensive cost-of-illness study for neuromuscular disorders in the United States, assessing the total cost to families and society per year for amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD) and myotonic muscular dystrophy (MMD, or DM).

Such studies are important for at least two reasons:

  • they inform the drug development industry about current costs of care and how a disease-modifying treatment might change those costs for families, government and insurers; and
  • they provide information to policy makers about the socioeconomic needs of families with chronic, debilitating disorders.

Cost-of-illness studies for neuromuscular disorders have been performed previously, but mostly outside the U.S. and mostly only with respect to direct medical costs. This U.S.-based study took into account medical costs, nonmedical costs and estimates of lost income as a result of someone in the household having a neuromuscular disorder.

The study, published online July 8, 2013, in the journal Muscle & Nerve, was authored by Jane Larkindale, MDA's vice president of research, with other authors from MDA and from the Lewin Group, a health care consulting firm based in Falls Church, Va.

MDA contracted the study to the Lewin group, who guided the research and completed the statistical analyses.

The study could not have been performed without the help of numerous MDA families who took part in focus groups and who completed the survey.

(Note: The initial study also included spinal muscular atrophy (SMA), but the data for that disease were inconclusive and so were not included in the final Muscle & Nerve paper.)

Information came from several sources

The investigators gathered data from three sources:

  • a 55-question survey that could be filled out online or on paper, sent to 600 households in which at least one family member was registered with MDA and noted to have ALS, DMD, MMD (DM) or SMA;
  • a database from a large, national private insurance company with information on 75 million people over a 10-year period; and
  • a Medicare claims database.

Cost of illness findings

From these sources, the researchers estimated the annual cost of ALS, DMD and MMD for families and for the United States as a whole for the following categories:

  • direct medical costs, including expenditures for inpatient and outpatient medical expenses, medications and equipment, including both out-of-pocket and reimbursed costs;
  • nonmedical costs, including expenditures for housing, vehicles, paid care at home, dietary supplements and travel related; and
  • indirect costs, which cover the estimated loss of income related to the illness of at least one family member with ALS, DMD or MMD.

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

Disease Medical Costs Nonmedical Costs Lost Income Total
ALS (amyotrophic lateral sclerosis)
$31,121 $17,889 $14,682 $63,692
Duchenne MD (DMD)
$22,533 $12,939 $15,481 $50,953
Myotonic MD (MMD, or 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 listed in the the table on the right.

Disease 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 very conservative.

The findings also showed that the costs for ALS, DMD and MMD are similar to those for other chronic disabling diseases, such as Parkinson's disease and multiple sclerosis, in the U.S.

Cost drivers

The largest driver of medical costs was outpatient care, and the largest driver of nonmedical costs was the necessity to move to another house or adapt housing, and the expense of paid caregiving.

Loss of income was significantly correlated with the amount of care needed by the person with a neuromuscular disorder.


"The numbers are less precise than we would like," Larkindale said. She noted, however, that having more precise information would likely increase, rather than reduce, the estimated costs of illness for the targeted disorders, making support for their alleviation even more urgent. Larkindale also said the study did not take into account nonfinancial costs, such as the emotional impact of having a chronic disease in the family.

Data have multiple applications

"This valuable study provides us with a way to translate the adverse effects of our diseases into dollar terms, the universal language of decision-makers and the policy arena," Larkindale said.

"It gives us a tool to use in lobbying the government for more investment in neuromuscular disorders, while giving the pharmaceutical industry a tool to help justify reimbursement for neuromuscular disease drugs.

"In addition, Annie Kennedy, MDA's senior vice president of advocacy, will be using these numbers frequently in lobbying for more resources to help meet the social and economic needs of the patient community."

(Watch a two-minute video of Annie Kennedy discussing the cost-of-illness study and what the results mean to individuals.)

Larkindale said she expects the cost-of-illness data to:

  • highlight the value of research into therapies for these diseases to funding sources (government grants, venture groups, etc.);
  • assist in lobbying for allocation of government research dollars;
  • justify reimbursement for potential treatments;
  • provide a basis for policy and planning relative to therapy development;
  • demonstrate the financial impact the diseases have on public programs, such as Medicare and Medicaid; and
  • provide important information for cost-effectiveness and cost-benefit analyses.

Goals: Slow the disease, help families cope

"For all conditions, increased costs are associated with increased care requirements," the authors write.

"ALS patient costs are significantly associated with use of wheelchairs and ventilators, and DM [MMD] patient costs are affected by cognitive impairment. These data suggest that interventions that slow disease progression, reduce care requirements, and keep patients walking for longer could significantly reduce the annual cost of these diseases."

They also note: "The findings help to underscore the importance of research into the causes and treatments of these conditions.

“In addition, the results suggest a possible role for additional policy initiatives to better help individuals and families in terms of treatment and long-term care, disease management, employment, and occupational training to improve quality of life and to alleviate human and economic burden."

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