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Health Technology Assessment and Appraisal of Therapies for Rare Diseases

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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

Innovative rare disease therapies and health technology assessment (HTA) share a lot of similarities. Both represent cases of interaction of epidemiology and health economics. Both are relatively new topics in public health practice. And both pose a lot of challenges to rare disease stakeholders who are currently looking for tools to support the timely access to innovative treatments while putting budget spending in order. This is why optimisation of assessment and appraisal of new rare disease therapies is a fundamental issue in rare disease health policy. Rare disease patients and caregivers expect prolonged life expectancy and improved quality of life and they perceive innovative health technologies as a rightful way to achieve these objectives.

Multi-criteria decision analysis (MCDA) provides a structured, transparent approach to identify preferred alternatives by means of combined calculation of relative importance of different criteria and performance of the alternatives on these criteria. The labyrinth of competing interests and numerous stakeholders involved is why innovative rare disease health technologies make an excellent case study of the integration between HTA and MCDA. This kind of formalisation of decision-making is perceived as fair and legitimate, leading to a balance and agreement. MCDA provides a stage for a debate of policy priorities, health system specifics and societal attitudes, while also addressing the impact of rarity on all criteria and considerations.

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Author information

Authors and Affiliations

  1. Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, Plovdiv, Bulgaria

    Georgi Iskrov, Tsonka Miteva-Katrandzhieva & Rumen Stefanov

  2. Institute for Rare Diseases, Plovdiv, Bulgaria

    Georgi Iskrov, Tsonka Miteva-Katrandzhieva & Rumen Stefanov

Authors
  1. Georgi Iskrov
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  2. Tsonka Miteva-Katrandzhieva
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  3. Rumen Stefanov
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Corresponding author

Correspondence to Georgi Iskrov .

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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Iskrov, G., Miteva-Katrandzhieva, T., Stefanov, R. (2017). Health Technology Assessment and Appraisal of Therapies for 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_13

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