Practical and Statistical Considerations in Designing an Early Phase II Osteoarthritis Clinical Trial: a Case Study

Naitee Ting
Department of Epidemiology
Biostatistics
Pfizer Inc.
ABSTRACT

After the withdrawal of Vioxx(r) in late 2004, the drug development environment for any new anti-inflammatory agents became more and more challenging. For every new compound developed to treat osteoarthritis (OA), both drug safety and drug efficacy are followed very closely. In a proof of concept (PoC) study designed at early Phase II, it is important to explore both a high dose so that the drug efficacy can be demonstrated, and a low dose to deliver an acceptable amount of efficacy, while maintaining safety. In this article, a true case study is presented to demonstrate how these considerations can be incorporated in an early Phase II clinical trial design. Keywords - Osteoarthritis, Dose Response, Phase II, Proof of Concept