Estimating the fiscal impact of rare diseases using a public economic framework: a case study applied to hereditary transthyretin-mediated (hATTR) amyloidosis

Abstract

Background: A wide range of rare diseases can have fiscal impacts on government finances that extend beyond expected healthcare costs. Conditions preventing people from achieving national lifetime work averages will influence lifetime taxes paid and increase the likelihood of dependence on public income support. Consequently, interventions that influence projected lifetime work activity, morbidity and mortality can have positive and negative fiscal consequences for government. The aim of this study was to apply a public economic framework to a rare disease that takes into consideration a broad range of costs that are relevant to government in relation to transfers received and taxes paid. As a case study we constructed a simulation model to calculate the fiscal life course of an individual with hereditary transthyretin-mediated (hATTR) amyloidosis in The Netherlands. In this lethal disease different progressive disease scenarios occur, including polyneuropathy and/or cardiomyopathy. Results: Due to progressive disability, health care resource use, and early death, hATTR amyloidosis with polyneuropathy receives more transfers from government compared to the general population. In a scenario where a patient is diagnoses with hATTR at age 45, an individual pays €180,812 less in lifetime taxes and receives incrementally €111,695 in transfers from the government, compared to a person without hATTR. Patients suffering from cardiomyopathy die after median 4 years. The health costs of this scenario are therefore lower than that of the other polyneuropathy-based scenarios. Conclusions: The fiscal analysis illustrates how health conditions influence not only health costs, but also the cross-sectorial public economic burden attributed to lost tax revenues and public disability allowances. Due to the progressive nature of hATTR amyloidosis used in this study, public costs including disability increase as the disease progresses with reduced lifetime taxes paid. The results indicate that halting disease progression early in the disease course would generate fiscal benefits beyond health benefits for patients. This analysis highlights the fiscal consequences of diseases and the need for broader perspectives applied to evaluate health conditions. Conventional cost-effectiveness framework used by many health technology assessment agencies have well-documented limitations in the field of rare diseases and fiscal modeling should be a complementary approach to consider.

The Clinical and Economic Burden of Newly Diagnosed Hereditary Transthyretin (ATTRv) Amyloidosis: A Retrospective Analysis of Claims Data

“…Third, this study examines only direct healthcare costs and does not include indirect costs such as reduced work productivity. Moreover, while we did not examine the effect of demographic factors, such as age, on the burden of illness, we would expect to find greater burden in certain populations similar to what has been found in other amyloidosis studies [14,28]. Additionally, costs prior to diagnosis, which may also contribute to burden, were not captured.…”

“…Additionally, the study reported the greatest disease burden for this group [28]. A fiscal cost model found that patients with ATTRv amyloidosis in the Netherlands pay less taxes and receive incrementally more in government intervention, with the conclusion that halting disease progression early would result in economic benefits beyond health benefits [14]. In the simulated disease scenarios, patients with severe cardiomyopathy had less significant health costs due to early mortality compared to patients without severe cardiomyopathy.…”

“…If left untreated, symptoms worsen with death occurring 3--15 years after clinical presentation [1,12]. Delayed diagnosis in amyloidosis is associated with poorer quality of life and productivity, as well as an increased disease burden [1,5,[13][14][15].…”

Val50Met hereditary transthyretin amyloidosis: not just a medical problem, but a psychosocial burden

“…Some had to adapt their schedule because they were unable to handle work-related stress or the fatigue related to the disease. Diseases that impact daily life, especially those that manifest at early ages, can lower lifetime earnings, savings and wealth accumulation, which can influence living standards in all life stages [ 24 ]. The disease was reported to lower the incomes of 29.8% of those surveyed, with its impact on patient income more important for those older than 50 years of age and in advanced stages of the disease, independently of the time since diagnosis.…”

Val50Met Hereditary Transthyretin Amyloidosis: Not Just a Medical Problem, but a Psychosocial Burden

“…Some had to adapt their schedule because they were unable to handle work-related stress or the fatigue related to the disease. Diseases that impact daily life, especially those that manifest at early ages, can lower lifetime earnings, savings and wealth accumulation, which can in uence living standards in all life stages (24). The disease was reported to lower the incomes of 29.8% of those surveyed, with its impact on patient income more important for those older than 50 years of age and in advanced stages of the disease, independently of the time since diagnosis.…”