WEBINAR
Help Shape the Future of Rare Disease Evidence Generation!
Join this insightful webinar that will tackle one of the most complex challenges in clinical research: designing and analysing trials for rare and ultra-rare diseases. The session will explore innovative trial designs and advanced analytical approaches that can transform evidence generation for these conditions. The discussion will focus on raising awareness of emerging methodologies, fostering collaboration from the very beginning, identifying current challenges in rare disease trials, and working toward shared principles for interpreting ultra-rare disease data.
A key topic of the session will be single-arm trials (SATs). SATs remain widely used in rare disease research and may be accepted by regulators in specific contexts, such as when treatment effects are dramatic or placebo use would be unethical. During the webinar, we will review regulatory and HTA positions on SATs, examine case studies where SATs succeeded and where they fell short, and discuss the challenges they pose. Another key topic will be starting from a single patient and ultimately leading to a US FDA approval, how it is initiated a real-world evidence clinical trial. We will also look at evolving statistical methods and strategies to optimize study design and meet evidence needs.