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Rare Diseases Epidemiology: Update and Overview pp 283–297Cite as

Cost-of-Illness in Rare Diseases

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Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1031))

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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Author information

Authors and Affiliations

  1. Fundación Canaria de Investigación Sanitaria (FUNCANIS), Santa Cruz de Tenerife, Spain

    Renata Linertová & Lidia García-Pérez

  2. Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC), Madrid, Spain

    Renata Linertová, Lidia García-Pérez & Iñigo Gorostiza

  3. Centro de Investigaciones Biomédicas de Canarias (CIBICAN), Santa Cruz de Tenerife, Spain

    Renata Linertová & Lidia García-Pérez

  4. Basurto University Hospital, Osakidetza, Spain

    Iñigo Gorostiza

Authors
  1. Renata Linertová
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  2. Lidia García-Pérez
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  3. Iñigo Gorostiza
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Correspondence to Renata Linertová .

Editor information

Editors and Affiliations

  1. RDR and CIBERER, Instituto de Salud Carlos III, Madrid, Spain

    Manuel Posada de la Paz

  2. Centro Nazionale Malattie Rare, Istituto Superiore di Sanita, Roma, Italy

    Domenica Taruscio

  3. National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA

    Stephen C. Groft

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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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