|"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:
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:
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:
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)
|Duchenne MD (DMD)
|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|
|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.
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.
"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:
"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."