The development of a new drug is often portrayed as a series of increasingly demanding clinical trials, performed in patients with a single target condition, as the development program advances toward drug approval. However, rarely does drug development involve a single approach to using a drug. Instead, drugs are tested in multiple clinical trials—many pursued after initial drug approval and licensure—involving different clinical indications or drug combinations. This family of trials could be referred to as a drug development “portfolio.” For example, the portfolio of the anticancer drug sorafenib included 203 clinical trials conducted over 13.2 years, spanning 26 different malignancies and 67 drug combinations.1 A portfolio can be enormous if defined in terms of drug classes. For instance, at least 803 trials are testing checkpoint inhibitors for the treatment of different malignancies.2
Kimmelman J, Carlisle B, Gönen M. Drug Development at the Portfolio Level Is Important for Policy, Care Decisions and Human Protections. JAMA. 2017;318(11):1003–1004. doi:10.1001/jama.2017.11502
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