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Burden of Rare Diseases in the USA almost $1 Trillion in 2019

The total economic burden for Rare Diseases in the United States of America for 2019 came to almost $1 Trillion! That is according to the National Economic Burden of Rare Disease Study conducted by the EveryLife Foundation for Rare Diseases.

The study, covering 379 rare diseases, affecting 15.5 million people in the U.S, put the total economic burden for that year at $966 billion — that’s over $600 billion more than the $327 billion estimated for diabetes, which affects some 34 million Americans, in 2017.

The National Economic Burden of Rare Disease Study, a multi-year project supported by 15 pharmaceutical companies and prepared by consultants from the Lewin Group, analysed the cost of rare diseases across three main areas:

- direct medical costs,

- indirect costs (as a result of productivity and other losses)

- non-medical costs and costs not covered by healthcare

“We’re trying to show that even though rare diseases are individually rare, if you combine them together, they are a huge burden on society. They are a public health crisis that policymakers need to pay attention to,” Julia Jenkins, the executive director of the EveryLife Foundation.

What made the study different from others was its attempt to include as much of the rare disease community as possible, rather than focusing on a single disease group. It also was unique in creating a survey to determine costs not shown through traditional Medicare, Medicaid, and private insurance claims.

The survey’s 39 questions covered topics ranging from disease severity, demographics, and caregiver profiles, to employment status, annual earnings, and disability benefits.

Rare disease cost: $966 billion for 2019

The indirect costs of these rare diseases, amounting to $548 billion of that $966 billion total economic burden, were extrapolated from this survey.

Productivity loss was the majority of these costs - from being unemployed or in forced retirement because of a rare disease, or missing work hours or days due to healthcare needs (whether as a patient or caregiver), including arranging appointments or insurance coverage. It also included simply being less effective on the job because of treatment side effects or from sleepless nights due to their condition, thwarting promotion or training opportunities in the work place.

Non-medical and uncovered healthcare costs, such as home modifications, and transport, dietary, and special education needs, together added about $111 billion to this indirect, out-of-pocket burden.

Direct medical costs, which added up things like inpatient and outpatient care, physician visits, prescription medications, hospitalisations, and durable medical equipment, were $418 billion.

“High medical cost is the story that underlies all of it,” Anne Pariser, director of the Office of Rare Diseases Research at the National Institutes of Health said. “What they have to endure, and their high medical and unmet medical needs is why it is so expensive.”

The group of rare diseases with the highest per capita direct medical costs was lysosomal storage diseases in both children and adults. The per-person 2019 cost for this disease area, which includes Batten, Fabry, Pompe and Sanfilippo syndrome, was $132,757 for children and $54,996 for adults.

The endocrine and metabolic disorders group, which includes cystic fibrosis, Cushing’s disease, and porphyria, had the second-highest amount of per-person direct medical costs among children, again because of underlying processes. For adults, disorders of the blood and blood-forming organs, like hemophilia and sickle cell disease, came in second.

Data that ‘adds power’ to patient stories

The study was a long time coming, according to Jenkins, who wanted one that would do for rare diseases what a similar study had done for Alzheimer’s: pump money into research, because the disease is too expensive not to treat.

That Alzheimer’s burden report projected costs of failing to treat 50 years into the future. The community then “came to Congress, and said they’re going to bankrupt the system if we don’t find a cure for Alzheimer’s … and that really made investment in that disease a priority,” she said.

“That’s what we’re trying to do here,” Jenkins added. “We’re trying to show that even though rare diseases are individually rare, if you combine them together, they are a huge burden on society. They are a public health crisis that policymakers need to pay attention to.”

While the 2019 price tag for rare diseases was no surprise to Jenkins or anyone at EveryLife, or to Pariser at the NIH, the prevalence was, Jenkins said — 15.5 million people in the U.S. with 379 diseases, when 30 million is the total estimated number of these patients, and 7,000 that of identified rare diseases.

“We can make the assumption that the burden is actually much greater than what is highlighted in the study,” Jenkins said.

To read the full National Economic Burden of RD report CLICK HERE.

Content taken from Muscular Dystrophy News (Read original article by Hawken Miller here)

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