First attempt of large phase III oncology trial using Japanese new trial evaluation system, the evaluation system of investigational medical care

Background: This is the first attempt to conduct a large-scale phaseIII oncology trial using the Evaluation System of Investigational Medical Care (ESIMeC). Methods: The study design is a randomized phase II / III trial comparing administration routes of carboplatin either intravenously (IV) or intraperitoneally (IP) in combination with weekly administration of paclitaxel for ovarian cancer patients. Target accrual is 746. Both IP carboplatin and weekly paclitaxel have not been approved for national insurance coverage in Japan. Results: Because of the expensive drug cost, it was first assumed impossible to conduct the trial if the study chair is responsible for purchasing the investigational drugs from the limited research grant or the patients have to pay for the investigational drugs without insurance coverage. Therefore, we negotiated with the pharmaceutical companies including generic makers to supply the investigational drugs with free of charge. The duration from initial consultation to the Ministry of Health, Labor, and Welfare to the finial approval to conduct the trial using ESIMeC was 8 months. Conclusion: The ESIMeC appears to be an efficient system as the official evaluation process of investigator-initiated, non-indication directed clinical trials, which manifestly require quality control of the trials. However, cost coverage for the investigational medicine or technique remains as an important issue to be resolved in the future, especially in large phase III oncology trials.