Statistics and Data Science Advancements in Drug Development: Perspectives from Academia, Industry, and Regulatory Authority

The design and analysis of contemporary cancer clinical trials present a myriad of challenges that continue to evolve with the advancements in cancer research and treatment. These challenges range from methodological issues, such as the selection of appropriate endpoints and statistical designs, to practical considerations, like patient recruitment and retention. The increasing complexity of trials, driven by the advent of personalized medicine and immunotherapies, further complicates the design and analysis process. This talk will explore these challenges in detail, highlighting the need for innovative strategies and flexible designs to improve the efficiency and effectiveness of cancer clinical trials.  

Multi-Arm Multi-Stage (MAMS) clinical trial designs are seamless Phase 2/3 designs that have multiple treatment arms and concurrent control arm. These designs allow treatment selection at early stage and uses these early stage patients' data in making confirmatory decision at the later stage; thus, saving time and resources. While the designs have been around for some time, they are not popular because of two major challenges. The first challenge is controlling the overall Type I error which is more complicated than traditional two-arm design due to various multiplicity issues. The second challenge is the choice of appropriate endpoint for treatment selection and how that choice may impact the confirmatory outcome. In this presentation, we will discuss these challenges and provide insights on the solutions that one may use to overcome these challenges. Simulation studies will be presented to show benefits of the proposed solutions 

As clinical trials continue to evolve in response to new regulatory standards, such as FDA's guidance on master protocol and dose optimization, there are significant challenges and opportunities for statisticians and data scientists. BeiGene has been at the forefront of adapting its strategies to align with these evolving guidelines. This presentation delves into our comprehensive approach to refining dose optimization processes in early-phase trials, emphasizing the collaboration between regulatory authorities and our internal teams. By sharing our experiences and lessons learned, we aim to highlight best practices and innovative strategies that enhance regulatory compliance while enhancing clinical trial efficiency. This discussion seeks to provide valuable insights that will drive the development of safer and more effective oncology therapies.