The Orphan Drug Act (ODA) was enacted in 1983 in an effort to incentivize pharmaceutical manufacturers to develop drugs that treat rare diseases.1 From a regulatory perspective, an orphan drug means a drug intended for use in a rare disease or condition, which is any disease affecting less than 200 000 persons in the United States, or more than 200 000 people but for which no reasonable expectation that the cost of developing a drug for such condition will be recovered.1 When the ODA was enacted in 1983, its drafting could not account for the recent technological developments of precision medicine. In a variety of disease states, such as cancer, much has changed: immunohistochemistry, genomics, and proteomics have reclassified many diseases. Next-generation sequencing, targeted therapy, and the pursuit of individualized medicines have altered treatment paradigms.
Nabhan C, Phillips EG, Feinberg BA. Orphan Cancer Drugs in the Era of Precision Medicine. JAMA Oncol. 2018;4(11):1481–1482. doi:10.1001/jamaoncol.2018.3498
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