[Phase III trial of new anticancer agents from a medical oncologists viewpoint]

The purpose of a phase III study is to establish the efficacy of a new agent (whether it is a single-agent, a combination of drugs or a combined modality approach) compared with the best conventional treatment. Several cytotoxic agents with promising single-agent activity in non-small cell lung cancer have recently been defined. Among them are Navelbine, taxol, taxotere, CPT-11 and gemcitabine. A phase III study of Navelbine has been completed. A study is currently in progress defining activity for taxol in combination with cisplatin. Several aspects in designing a phase III trial of CPT-11 related to the efficiency were discussed, with particular emphasis on the following points considered particularly important, timely, or relevant to this symposium. Firstly, the value of a clinical trial is determined mostly by the importance of the question addressed. After the question of timing has been resolved, the choice of the test and appropriate control treatments is central. The keys to obtaining a reliable answer are the use of randomized treatment allocation, adequate sample size, good data quality, protocol adherence, sufficient follow-up, avoidance of exclusions, appropriate analysis, and lack of data dredging. Participation of a biostatistician is also indispensable for improving the efficiency of comparative clinical trials.