Modern Approaches to Clinical Trials Using SAS: Classical, Adaptive, and Bayesian Methods

6.2 Statistical Design Considerations in a Classical Dose-Response Study

6.2.1 Dose-Response Relationship

In most cases, phase II features a learning period when the patient population may be first exposed to the test therapy for evidence of clinical benefit and risk; it is also a period to explore and recommend alternative doses to be tested in the later phase (e.g., phase III) confirmatory clinical trials. In the development of a first-in-class new chemical entity, the first phase IIa study is referred to as a proof-of-concept (PoC) study followed by phase IIb study called dose-ranging studies. A PoC study is often faster and cheaper because it only requires a well-tolerated dose of test therapy plus a control group (e.g., placebo), as appropriate. ...