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Cost-Effectiveness Methods and Newborn Screening Assessment

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Rare Diseases Epidemiology: Update and Overview

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

Abstract

Nowadays, health funding decisions must be supported by sound arguments in terms of both effectiveness and economic criteria. After more than half a century of newborn screening for rare diseases, the appropriate economic evaluation framework for these interventions is still challenging. The validity of standard methods for economic evaluation heavily relies on the availability of robust evidence, but collection of such evidence is precluded by the rareness of the conditions that may benefit from screening. Furthermore, there are a series of conceptual and methodological limitations that warrant further careful consideration when assessing the cost-effectiveness of newborn screening programs. In this chapter we provide a general overview of current economic evaluation methods and the challenges for their application to newborn screening programs.

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Notes

  1. 1.

    It is worth noting that the term cost-effectiveness analysis is used extensively in the literature as a synonymous to economic evaluation, independently of the outcome measure used in the analysis.

  2. 2.

    www.e-imd.org

  3. 3.

    www.e-hod.org

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Author information

Authors and Affiliations

  1. Departamento de Ingeniería Informática y de Sistemas, Universidad de La Laguna, La Laguna, Spain

    I. Castilla-Rodríguez

  2. Centre for Biomedical Research of the Canary Islands (CIBICAN), Santa Cruz de Tenerife, Spain

    I. Castilla-Rodríguez

  3. Health Services Research on Chronic Patients Network (REDISSEC), Madrid, Spain

    I. Castilla-Rodríguez, L. Vallejo-Torres, J. Mar & P. Serrano-Aguilar

  4. Department of Applied Health Research, University College London, London, UK

    L. Vallejo-Torres

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

    L. Vallejo-Torres & C. Valcárcel-Nazco

  6. Unit of Diagnosis and Treatment of Congenital Metabolic Diseases, Department of Pediatrics, University Clinical Hospital of Santiago de Compostela, Santiago de Compostela, Spain

    M. L. Couce

  7. IDIS, CIBERER, Madrid, Spain

    M. L. Couce

  8. Clinical Management Service, Alto Deba Hospital, Mondragón, Spain

    J. Mar

  9. Evaluation Unit of the Canary Islands Health Service (SESCS), Tenerife, Spain

    P. Serrano-Aguilar

  10. Centre for Biomedical Research of the Canary Islands (CIBICAN), Tenerife, Spain

    P. Serrano-Aguilar

Authors
  1. I. Castilla-Rodríguez
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  2. L. Vallejo-Torres
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  3. M. L. Couce
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  4. C. Valcárcel-Nazco
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  5. J. Mar
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  6. P. Serrano-Aguilar
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Corresponding author

Correspondence to I. Castilla-Rodríguez .

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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Castilla-Rodríguez, I., Vallejo-Torres, L., Couce, M.L., Valcárcel-Nazco, C., Mar, J., Serrano-Aguilar, P. (2017). Cost-Effectiveness Methods and Newborn Screening Assessment. 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_16

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