The Future of Clinical Trial Design in Oncology

Clinical trials represent a fulcrum for oncology drug discovery and development to bring safe and effective medicines to patients in a timely manner. Clinical trials have shifted from traditional studies evaluating cytotoxic chemotherapy in largely histology-based populations to become adaptively designed and biomarker-driven evaluations of molecularly targeted agents and immune therapies in selected patient subsets. This review will discuss the scientific, methodological, practical, and patient-focused considerations to transform clinical trials. A call to action is proposed to establish the framework for next-generation clinical trials that strikes an optimal balance of operational efficiency, scientific impact, and value to patients. Significance: The future of cancer clinical trials requires a framework that can efficiently transform scientific discoveries to clinical utility through applications of innovative technologies and dynamic design methodologies. Next-generation clinical trials will offer individualized strategies which ultimately contribute to globalized knowledge and collective learning, through the joint efforts of all key stakeholders including investigators and patients.

Automated summary

Clinical trials have evolved from traditional cytotoxic chemotherapy studies to biomarker-driven evaluations, aiming to provide safer and more effective treatments for patients. The future of cancer clinical trials will require a framework that efficiently transforms scientific discoveries into clinical applications, offering personalized treatment strategies and contributing to global knowledge and collective learning through collaboration among all stakeholders.