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

October 16, 2019
Laura Joszt, MA
Laura Joszt, MA

Laura is the editorial director of The American Journal of Managed Care® (AJMC®) and all its brands, including The American Journal of Accountable Care®, Evidence-Based Oncology™, and The Center for Biosimilars®. She has been working on AJMC® since 2014 and has been with AJMC®'s parent company, MJH Life Sciences, since 2011. She has an MA in business and economic reporting from New York University.

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 amyloidosis has a public economic burden beyond just health costs in the Netherlands.

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.

  • Scenario 1: This patient has early disease onset and a median progression until an early death at 55 years. This individual pays fewer taxes (—€180,812; US –$199,505) but received more from the government in the form of pensions, disability payments, and healthcare (+€111,695; US +$123,242).
  • Scenario 2: This patient lives 10 years longer, but their earning potential is only slightly higher than that in scenario 1. This individual pays €124,138 (US $136,972) less in taxes, but receives €603,153 (US $665,511) more from the government.
  • Scenario 3: This patient has late disease onset (age 60) and dies at age 70. While the gap between taxes paid for this patient and the general population is the narrowest (€7013; US $7738), this individual is still receiving a substantial amount more from the government compared with the general population (€123,426; US $136,186).
  • Scenario 4: While this patient also has a late disease onset (age 6), diagnosis is late, and they have severe cardiomyopathy. This patient dies within 4 years. As a result, this patient only pays €13,156 (US $14,516) more in taxes but receives €172,459 (US $190,289) less from the government compared with the general population.

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.

Reference

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.