Abstract
Cost-of-illness (COI) studies quantify the economic burden of a disease, including direct healthcare and non-healthcare costs and productivity losses. Different approaches can be adopted to evaluate the resources associated to a disease and to calculate the total costs. Prevalence-based studies estimate the total costs of a disease during a given period, while incidence-based studies measure lifetime costs from onset until death. Data can be collected from individuals, using a bottom-up approach, or from population statistics, using a top-down approach. Different perspectives are possible, but the broadest and also mostly used is the societal one. Appropriate discounting should be applied for future costs and a sensitivity analyses of main parameters should be performed. The main limitation of COI studies is that they don’t account the outcomes or benefits of possible treatments.
There is a lack of COI studies in the field of rare diseases. A multinational COI study (BURQOL-RD) evaluated recently the burden of 10 rare diseases in Europe, using a prevalence-based method with a bottom-up approach to quantify resources from a societal perspective, which is the mostly used methodology for COI studies in rare diseases; however, several other studies illustrate different approaches to conduct COI analysis in this field, such as incidence-based methods or narrower perspectives.
COI studies are useful to inform policy-makers about the magnitude of a disease. To support correctly the decision-making process, it is necessary to identify the cost-drivers through COI studies with robust design and standardized methodology.
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Linertová, R., García-Pérez, L., Gorostiza, I. (2017). Cost-of-Illness in Rare Diseases. In: Posada de la Paz, M., Taruscio, D., Groft, S. (eds) Rare Diseases Epidemiology: Update and Overview. Advances in Experimental Medicine and Biology, vol 1031. Springer, Cham. https://doi.org/10.1007/978-3-319-67144-4_17
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