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

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.

Population access to new vaccines in European countries.

Time from registration to population access to new vaccines can take considerable time in European countries. Reasons might be found in the regulatory framework, decision-making processes or the assessment of vaccines by evaluating bodies. The aim of this study was to determine whether some decision-making processes can explain between-country differences in the time to population access to new vaccination programs. Information gathered from a survey among European National Vaccine Industry Groups was combined with information from official health authorities, vaccine manufacturers and literature published. Firstly, a retrospective survey was conducted to measure access time to new vaccines against three diseases in 17 European countries. Secondly, qualitative information on the country-specific decision-making frameworks for the introduction of new "vaccination programs" was identified in a cross-sectional survey. Spearman's rank correlation coefficients (ρ) were used for data analysis. The median access time to new vaccines was 6.4 years (95% confidence interval: 5.7-7.1 years) post marketing authorization. National assessments underlying immunization policy decisions (recommendation phase) absorbed most of the access time. Correlation analysis suggested that processes with established timelines and clarity in regard to vaccine evaluation criteria used could ameliorate the effectiveness of the decision-making process. In order to reduce the time to access for new, beneficial vaccines, the underlying vaccination recommendation, implementation and funding process needs to be understood and optimized, where necessary.