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Orphan Drugs in Oncology

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Book cover Regulatory and Economic Aspects in Oncology

Part of the book series: Recent Results in Cancer Research ((RECENTCANCER,volume 213))

Abstract

Rare diseases represent a group of conditions affecting a very limited number of patients. Low profitability resulting from the small size of target population coupled with difficulties in conducting the research causes the lack of interest from the pharmaceutical industry. In order to promote research and development of medicines for rare diseases, a special ‘orphan’ legislation was introduced in a number of regions. These measures led to a significant increase in the number of approved orphan molecules. The high per patient cost of orphan drugs, as well the rapid growth of orphan drug sector, raised concerns regarding the sustainable funding of therapies for rare diseases. Rare cancers represent the majority of the current orphan drug market and are often associated with very high revenues. This chapter provides a review of orphan legislations and health technology assessment framework, analyses the position of oncology drugs on the orphan drug market and discusses future perspectives.

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Authors and Affiliations

  1. Mental Health and Public Health, Inserm U669, Maison de Solenn, Paris, France

    Daria Korchagina

  2. Public Health Department, Aix-Marseille University, Research Unit EA 3279, Marseille, France

    Szymon Jaroslawski & Mondher Toumi

  3. Laboratory P2S Systemic Health, Laennec Faculty of Medicine, Claude Bernard University Lyon 1, Lyon, France

    Guy Jadot

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  1. Daria Korchagina
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  2. Szymon Jaroslawski
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  3. Guy Jadot
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  4. Mondher Toumi
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Correspondence to Daria Korchagina .

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  1. Institute for Pharmaeconomic Research, Vienna, Austria

    Evelyn Walter

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Korchagina, D., Jaroslawski, S., Jadot, G., Toumi, M. (2019). Orphan Drugs in Oncology. In: Walter, E. (eds) Regulatory and Economic Aspects in Oncology. Recent Results in Cancer Research, vol 213. Springer, Cham. https://doi.org/10.1007/978-3-030-01207-6_8

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  • DOI: https://doi.org/10.1007/978-3-030-01207-6_8

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