European journal of clinical pharmacology
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Eur. J. Clin. Pharmacol. · Sep 2019
Regulatory characteristics and pivotal study design of US Food and Drug Administration approval of drugs for major vs. minor cancer.
We aimed to investigate the regulatory approval of drugs for cancers by the US Food and Drug Administration based on the cancer type (major vs. minor), including the use of expedited development programs and duration from Investigational New Drug application (IND) to marketing approval. ⋯ Drugs targeting minor cancers have frequently utilized the expedited development programs; thus, efficiently shortening time to approval. As many of such drugs are approved based on non-comparative pivotal studies, meticulous evaluation and follow-up should be performed for such drugs after their approval.