T-10: A Seamless Phase 2/3 Adaptive Design for Clinical Trials with a Continuous Endpoint in Asia

Poster Presenter

Lien-Cheng Chang

Senior Secretary, Department of Intellectual Property and Technology Transfer
Academia Sinica
Taiwan

Objectives

Method

We demonstrate the phase II/III design for continuous endpoint based on pairwise comparisons at the phase II stage when evaluating the efficacy of drugs.

Results

Reducing the costs of clinical trials is absolutely crucial for pharmaceutical development. More efficient and reliable methods are needed to minimize sample size, shorten the period of development duration thus reducing the cost for drug development. We demonstrate the phase II/III design for continuous endpoint based on pairwise comparisons at the phase II stage when evaluating the efficacy of drugs. For the phase II stage, patients are randomly assigned to receive either one of several doses of the test drug or are assigned to the control group. If one or more doses are declared to have a statistically significant superior efficacy over the control group, these doses will be selected for the phase III trial. New patients recruited randomly receive either the selected doses of the test drug or that of the control group. We find the critical value at each stage to determine whether the treatment should continue or be dropped. We integrate the traditional phase II and III trials into a single trial. The data collected from phase II stage will also be included in the final analysis, and thus both sample size reduction and trial time saving may be possible.

Conclusion

This design is important as it could be used in regions with similar ethnicity, e.g. the greater Chinese area. In regions with similar ethnicity the phase II stage can be conducted in one region, whereas the phase III stage can be conducted in all regions. Our design promotes global new drug development and will benefit patients worldwide.