The Phase III Clinical Cancer Trial

Abstract

Phase III clinical trials in oncology represent the final step in a multistage process of evaluating the role of a new drug, combination regimen, or other treatment modality in the clinical cancer setting. In the United States, these trials are often conducted by large cooperative groups with support from the National Cancer Institute (NCI). Some cooperative groups were founded in the 1960s, and many now have world-wide clinical site affiliations. The major cooperative groups conducting phase III trials in adult and pediatric hematological malignancies and solid tumors in the United States are listed in Table 1. Phase III trials compare a new drug or combination to therapy regarded as standard of care in a specific cancer setting. Commercial sponsors often play an active role in the conduct of trials, either by assuming direct responsibility for clinical trial design and implementation or through agreements with cooperative groups. Input on the design of these trials is often sought from regulatory bodies such as the U.S. Food and Drug Administration (USFDA), the European Agency for the Evaluation of Medicinal Products (EMEA), or the Japanese regulatory authorities, owing to the potential regulatory impact of some phase III trials. The design, conduct, and outcome analysis of phase III cancer trials is a complex interaction among academic institutions, cooperative groups, commercial sponsors, and regulatory bodies.