THE 5^th STAT4ONC ANNUAL SYMPOSIUM

SHORT COURSE 2

May 4, 2022
1:30 PM – 5:30 PM CT

Phase II Clinical Development of New Drugs

Naitee Ting, Boehringer-Ingelheim

Qiqi Deng, Moderna

In the process of medicine discovery and development, understanding the dose-response relationship is one of the most important and challenging tasks. It is critical to identify the right range of doses in early stages of medicine development so that Phase III trials can be properly designed to confirm appropriate dose(s) for the patient. Usually in the beginning of Phase II development, there is limited information to help guide trial designs, therefore Phase II clinical trials often consists of objectives for establishing proof of concept (PoC), identifying a set of potentially safe and efficacious doses, and characterizing the dose-response relationship.

Some of the major challenges in designing these Phase II trials include the selection of dose range and frequency, clinical endpoints and/or biomarkers, and the use of control(s). Inappropriate Phase II trial designs may lead to delay of the medicine development program or waste of investment. Specifically, misleading results from poorly designed Phase II trials could force a Phase III program to confirm sub-optimal dose(s), or even stop developing a potentially useful medicine. Therefore, it is critical to consider Phase II trial designs, in the broader context of the entire medicine development plan, to make the best use of all available information, and to engage relevant experts. The first part of presentation will focus on these perspectives.

The second part of the presentation will focus on the recently development on dose finding for oncology field. With FDA's project Optimus, there has been increasing interest in improving dose finding process for cancer treatment. Instead of simply use maximum tolerable doses, dose finding design options which provide additional insight into dose optimization under context of oncology program will be discussed.