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Book Sections Year : 2019

Orphan Drug Designations as Valuable Intangible Assets for IPO Investors in Pharma-Biotech Companies

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Abstract

Personalized and precision medicine (PPM)—the targeting of therapies according to an individual’s genetic, environmental, or lifestyle characteristics—is becoming an increasingly important approach in health care treatment and prevention. The advancement of PPM is a challenge in traditional clinical, reimbursement, and regulatory landscapes because it is costly to develop and introduces a wide range of scientific, clinical, ethical, and socioeconomic issues. PPM raises a multitude of economic issues, including how information on accurate diagnosis and treatment success will be disseminated and who will bear the cost; changes to physician training to incorporate genetics, probability and statistics, and economic considerations; questions about whether the benefits of PPM will be confined to developed countries or will diffuse to emerging economies with less developed health care systems; the effects of patient heterogeneity on cost-effectiveness analysis; and opportunities for PPM’s growth beyond treatment of acute illness, such as prevention and reversal of chronic conditions.This volume explores the intersection of the scientific, clinical, and economic factors affecting the development of PPM, including its effects on the drug pipeline, on reimbursement of PPM diagnostics and treatments, and on funding of the requisite underlying research; and it examines recent empirical applications of PPM.
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hal-02152424 , version 1 (28-04-2021)

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Philippe Gorry, Diego Useche. Orphan Drug Designations as Valuable Intangible Assets for IPO Investors in Pharma-Biotech Companies. Berndt, E.R.; Goldman, D.P.; Rowe, J. Economic Dimensions of Personalized and Precision Medicine, University of Chicago Press Books, pp.305-334, 2019, 9780226611068. ⟨10.7208/chicago/9780226611235.003.0011⟩. ⟨hal-02152424⟩
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