[ON DEMAND] RWE Expertise: A Regulatory Roadmap for Small-to-Mid Sized Biotech & Pharma Sponsors
Join us for a short focused session with 3 Mini episodes (10Mins/eps.)which will enable attendees to better leverage critical real-world evidence (RWE) in regulatory submissions, with a focus on the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). With extensive expertise in US data and solving complex evidence generation challenges, Dr. Jason Simeone will present recent initiatives intended to foster the use of RWE in regulatory submissions, key contexts for use, and practical advice.
Why Attend: While agencies such as the FDA and EMA have consistently signaled the increasing importance of RWE in regulatory decision-making , small-to-mid-sized sponsors need to be aware of relevant guidelines, different eligible uses for RWE, and the various pitfalls that can endanger their submission. Attendees will gain confidence in the value of RWE, whether they intend to use it as supplementary or even primary evidence in their submission.
EP 1: Decoding FDA Activity in RWE : Highlights and Communication Strategies for Small Sponsors
Content: An overview of the evolving FDA RWE Framework and key guidance, including data standards for submissions containing RWD.
Focus: Understanding the Advancing RWE Program, which offers sponsors the opportunity to discuss RWE proposals with FDA staff before protocol development.
Key Takeaway: Learn how to utilize Type C meetings to foster early communication with regulators and de-risk your submission pathway.
EP 2: Using RWE Across the Product Lifecycle : Overview and Use Cases for Sponsors with Limited Resources
Content: Evidence generation needs differ across the lifecycle, from early-stage development to post-marketing authorization.
Focus: For sponsors with limited resources, we discuss strategies for using RWE to conduct database landscaping, strengthen disease background, and inform clinical trial selection criteria.
Key Takeaway: Discover how RWE can validate trial endpoints or serve as a future external comparator arm when planning single-arm trials.
EP 3: From Theory to Approval: Analyzing Successful and Unsuccessful RWE Usage in FDA Submission
Content: A landscape review of RWE use cases. Learn what to avoid from previous decisions where the inclusion of RWE was unsuccessful while understanding what factors will strengthen the submission
Focus: A "Post-Mortem" analysis of supportive cases, such as Tazemetostat, versus non-supportive cases like Selinexor.
Key Takeaway: Understand the critical factors that strengthen a submission and learn to avoid pitfalls such as immortal time bias, selection bias, and confounding.
Speaker
Jason Simeone
Jason is Managing Director and the US RWE lead at GIPAM. He is a pharmacoepidemiologist with over 17 years of experience in real-world evidence generation, evidence synthesis, and related activities in the United States, Europe, and other regions. Jason has extensive expertise in the assessment of real-world data and routinely executes data landscaping and feasibility studies to identify fit-for-purpose data sources for work being submitted to regulatory agencies, health technology assessment bodies, and other stakeholders. He has also contributed to Duke-Margolis working groups focused on data quality, reliability, and other topics, as well as serving in several relevant special interest groups for the International Society of Pharmacoepidemiology (ISPE). He received his Ph.D. and Master’s degree in Pharmacoepidemiology and Pharmacoeconomics from the University of Rhode Island