Abstract
Although 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.
Original language | English (US) |
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Pages (from-to) | 502-507 |
Number of pages | 6 |
Journal | Clinical pharmacology and therapeutics |
Volume | 97 |
Issue number | 5 |
DOIs | |
State | Published - May 2015 |
ASJC Scopus subject areas
- Pharmacology
- Pharmacology (medical)