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FDA: 2019 Continues Uptick in Orphan Drug Approvals

(Regulatory Focus) Jan 6, 2020 - Similar to 2017 and 2018, the US Food and Drug Administration (FDA) in 2019 approved an increasing number of orphan drugs and biologics, as well as drugs targeted at specific cancers.

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William McGivney, PhD (Posted: January 13, 2020)

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In Oncology, drug/biologic development is clearly in a world of orphan diseases. The FDA defines an orphan disease as one that involves less than 200,000 patients diagnosed or afflicted with the disease in the United States. As such. In general terms, all but the major tumor types can be considered to be orphans. As we progress deeper and deeper into targeted therapies, almost all tumor types and specific indications will be considered to be orphan diseases. The advancement in our knowledge about targets and biomarkers that predict responsiveness of a patient’s cancer will be major determinants of specific diagnoses. As patient populations become smaller and smaller, the pricing pressures on the cost of agents that will be approved for such indications will be great. We will have important increases in the number of targeted therapies in the coming months and years. As such indications will become more "orphan-orphan".

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