• R Simon, G M Blumenthal, M L Rothenberg, J Sommer, S A Roberts, D K Armstrong, L M LaVange, and R Pazdur.
• National Cancer Institute, Bethesda, Maryland, USA.
• Clin. Pharmacol. Ther. 2015 May 1; 97 (5): 502-7.

AbstractAlthough randomized trials provide the most reliable evidence of a drug's safety and efficacy, there are situations where randomized trials are not possible or ethical. In this article we discuss when and how single-arm trials can be used to support full approval of oncology drugs. These include situations in which an unprecedented effect on tumor response is observed in a setting of high unmet medical need, clinical trial patients have been well characterized, enabling a target population to be clearly defined, experience exists in a sufficient number of patients to allow adequate assessment of the risk:benefit relationship, and a proper historical context can be provided for analysis. We also discuss how response rates might be considered predictive of long-term outcomes or clinically meaningful in and of themselves in certain contexts.© 2015 American Society for Clinical Pharmacology and Therapeutics.

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