DIA/FDA Biostatistics Industry and Regulator Forum

Due to the COVID-19 pandemic, the need to have more flexible approaches for conducting clinical trials became essential. Decentralizing elements of a clinical trial enable information to be generated and captured without traditional clinical trial infrastructures. As an example, endpoints can be captured remotely using digital technologies. By reducing the burden of trial participation on patients and their caregivers, decentralization is likely to facilitate the recruitment, retention, and engagement of a more diverse patient population. However, decentralizing elements of a trial may impact the scientific question(s) the study can answer. Additionally, increased heterogeneity amongst remotely collected data influences sample size requirements and analysis strategies. In this session, through discussion and case studies, we will: i) explore how the ICH E9(R1) estimand framework can evaluate the impact of decentralization on study objectives; ii) discuss statistical challenges with regards to establishing endpoint validity and reliability; and iii) highlight opportunities for leveraging innovative analytical approaches.