Today's oncology drug development is undergoing a radical shift away from the traditional paradigm of dose selection. This approach, aiming to define the maximum tolerated dose, can put unnecessary burden on patients and all too often can result in excess toxicity for those desperately seeking a cure. Doses and schedules of molecularly targeted and immune- and cell-based therapies are often poorly characterized upon entering registrational trials, leading to further inefficiency in the overall process.

The FDA's Oncology Center of Excellence has taken steps to solving this challenge through Project Optimus, for which the draft guidance was recently published in January 2023. It seeks to better inform dose selection and maximize patient outcomes through its move from drug-centered to patient-centered drug development.

This insight brief will discuss strategies for building dose finding and optimization into early clinical development to allow for a more sophisticated analysis of dose and schedule before moving into later phase trials. It will also highlight implications of this new paradigm for sponsors who are developing drugs for oncology patients.