The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022

Abraham Viju Ipea Pediatrics, Children’s National Hospital, Washington DC, USAView further author information

Lewis J. Fermaglichb Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USACorrespondence[email protected]
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Selma Kraftb Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USAView further author information

Catherine Measeb Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USAView further author information

Tran Leb Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USAView further author information

Kathleen L. Millerc Division of Science and Public Health Policy, Office of Science and Data Policy, Office of the Assistant Secretary for Planning and Evaluation, US Department of Health and Human Services, Washington DC, USAView further author information

The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022

Table 1. Top 10 rare cancers with the most orphan drug designations (1983–2022).

Table 2. Top 10 orphan drug-designated cancers with the most initial approvals (1983–2022).

Table 3. Classification of oncologic orphan drug designations (and Approvals) by site group and site, 1983—2022 (Designations, n = 2,355) (Approvals, n = 317).

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The Orphan Drug Act and rare cancers: a retrospective analysis of oncologic orphan drug designations and associated approvals from 1983-2022

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Table 1. Top 10 rare cancers with the most orphan drug designations (1983–2022).

Table 2. Top 10 orphan drug-designated cancers with the most initial approvals (1983–2022).

Table 3. Classification of oncologic orphan drug designations (and Approvals) by site group and site, 1983—2022 (Designations, n = 2,355) (Approvals, n = 317).

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