Analysis Highlights How Rare Diseases Have Broader Fiscal Impact Than Health Costs

Laura Joszt

Rare diseases may affect a small number of people, but they have fiscal impacts beyond just healthcare costs. A new study in Orphanet Journal of Rare Diseases used a public economic framework to identify how hereditary transthyretin-mediated (hATTR) amyloidosis has a public economic burden beyond just health costs in the Netherlands.

hATTR amyloidosis can manifest as sensory, motor, and autonomic neuropathy, as well as cardiomyopathy. The disease can make it difficult for people to work and of the 33.3% of patients with hATTR amyloidosis who are employed, 21.9% report missing work because of their disease and closer to half (40.7%) report some impairment at work.

The authors analyzed the impact of hATTR amyloidosis morbidity, linked to employment activity based on the polyneuropathy disability score, and mortality on government costs in the Netherlands.

“In contrast with the healthcare perspective, the government public economic perspective can have consequences even in death as health events and disability represent unfulfilled lifetime taxes paid, increased social dependency costs and pension receipts,” the authors explained.

They developed a fiscal cost model based on other frameworks that are used to evaluate investments in healthcare technologies and disease burden. The authors estimated that the general population between the ages of 40 and 80 years receives €338,330 (US $373,309) and pays €319,922 (US $352,998) in taxes. They then looked at 4 scenarios. In the first 3 scenarios the patient has hATTR amyloidosis without severe cardiomyopathy. In the fourth scenario the patient has hATTR amyloidosis with severe cardiomyopathy. These fiscal models, the authors noted, can help inform government stakeholders to “how changes in morbidity and mortality influence public accounts.” While hATTR amyloidosis has a small public economic burden because it is a rare disease, such an analysis on a broader public health burden, such as diabetes or obesity, may be more informative.

“Introducing fiscal models in healthcare decision making can introduce new factors to consider in resource allocation decisions,” the authors noted.

For instance, this assessment highlighted the additional public costs beyond just health costs. As a result, investments that delay progression or prevent health events can be fiscally beneficial to governments when considering future taxes and lower disability payments.

“We believe this approach can be applied to a range of different rare diseases and offers a complementary approach to cost-effectiveness analysis and can be used for priority setting,” the authors concluded.

Connolly MP, Panda S, Patris J, Hazenberg BPC. Estimating the fiscal impact of rare diseases using a public economic framework: a case study applied to hereditary transthyretin-mediated (hATTR) amyloidosis. Orphanet J Rare Dis. 2019 14:220.
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