All Times EDT
Keywords: real world evidence, regulatory decisions, rare disease
For rare disease and some life threatening disease without any treatment, a large sample size confirmatory trial with blinded placebo control may not always be feasible or ethical.Last year, U.S. Food and Drug Administration (FDA) as well as China’s National Medical Products Administration (NMPA) issued a guidance on how to submit documents using RWD and real-world evidence (RWE). The European Medicines Agency also has similar work underway. Regulatory agencies around the world as well as the pharmaceutical industry hold highly consistent interest on utilizing the real-world data(RWD) to support regulatory decision-making and bring life saving medications to patients.
During the discussion, We discuss a recent FDA guidance for using RWE in supporting rare disease drug development, we will share our experience of using history studies as external control groups for single-arm intervention trials as well as some concerns on selection bias, confounding, and unrecognized baseline differences. Lastly, we also want to discuss some future directions and limitations (data reliability and quality) to expand the use of RWD and RWE to support orphan drug approvals.
Here are some inspiration questions for participants to consider before join the round table discussion:
1. Where and when can we use RWE/RWD to support drug development for rare disease? 2. What is the minimum requirements for RWD/RWE to be used as historical control of a registration trial in rare disease area (data quality, consistency with the trial design, severity of the disease, etc)? 3. What are the factors that prevent the sponsor or FDA to use RWE/RWD for drug development and approval? How can the society help close the gap? 4. Tell us about your story of using RWE/RWD to help patients with rare disease.