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Oncologic Rare Disease Drug Development

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Rare Disease Drug Development

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Abstract

Recent advancements in technology have facilitated progress in attaining a greater understanding of cancers than ever before, transforming what used to be known as histological types of cancer into a large group of rare diseases. Alongside these discoveries, new druggable targets have been revealed, giving rise to a new wave of targeted therapies for new disease subtypes. These, together with the earlier known rare cancer types, form a large pool of rare diseases, for which drugs are developed under the Orphan Drug Act. In this chapter, we will discuss the segmentation of the larger oncology indications based on biomarkers, and the critical role of these biomarkers in the overall success of a drug candidate. We will examine the business opportunity for oncology drug makers focusing on rare cancer indications. We will also touch on the probability of success of drug development in the rare cancer indication space. Examples relevant to each section will outline important cases of drug development within this context.

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Author information

Authors and Affiliations

  1. Syneos Health, Tel Aviv, Israel

    Keren Moss

  2. Syneos Health, Budapest, Hungary

    Jozsef Palatka

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  1. Keren Moss
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  2. Jozsef Palatka
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Correspondence to Keren Moss .

Editor information

Editors and Affiliations

  1. Rare Disease Consortium, Syneos Health Clinical Services, Morrisville, NC, USA

    Raymond A. Huml

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Moss, K., Palatka, J. (2021). Oncologic Rare Disease Drug Development. In: Huml, R.A. (eds) Rare Disease Drug Development. Springer, Cham. https://doi.org/10.1007/978-3-030-78605-2_12

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  • DOI: https://doi.org/10.1007/978-3-030-78605-2_12

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-030-78604-5

  • Online ISBN: 978-3-030-78605-2

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