The Growing Complexity Of Conducting Oncology Trials

By Louise Scott, Ph.D.

Early-phase oncology development is undergoing rapid transformation as regulatory expectations, scientific innovation, and precision medicine converge. Project Optimus has pushed sponsors to move beyond toxicity-driven dose selection and instead generate richer PK, PD, and dose-response data earlier in development. This shift, combined with increasingly targeted therapies, has expanded the size and geographic scope of early-phase studies, which now commonly operate across multiple countries to access narrower patient populations.

At the same time, trial designs are becoming more sophisticated: traditional Phase I monotherapy dose escalation is giving way to integrated Phase 1/2 studies that assess safety, dosage, and early signals of efficacy within a unified framework. Sponsors are also adopting adaptive and model-based dose-finding methods, incorporating expansion cohorts, and relying more heavily on biomarker-driven enrollment strategies. These scientific advances bring significant operational complexity, from global sample logistics and expanded safety monitoring to increased vendor coordination, resource demands, and region-specific regulatory expectations.

As diversity plans and multi-regional considerations become core to trial design, early-phase oncology programs must navigate a landscape that offers unprecedented opportunity — alongside a growing set of challenges that shape how new cancer therapies move from first-in-human studies toward clinical impact.