SW-4B - Real-world evidence fit for regulatory applications — what does good look like?

Background: Real-world evidence (RWE) is increasingly being used for regulatory decision-making related to biologics, drugs, and medical devices. Multiple health authorities around the globe have issued guidance documents related to RWE in the past few years. These guidance documents largely codify best practices and establish general recommendations for the design and generation of RWE. However, interpretation is still needed to translate these guidelines into practice. Many initiatives have been launched to synthesize existing guidance and develop and evaluate methods, tools, and frameworks that can be used to demonstrate that RWE is consistent with regulatory guidance.

Objectives: 1. Provide an overview of current health authority guidance on RWE for biologics, drugs, and devices;
2. Highlight multiple initiatives aimed at developing tools and approaches for demonstrating the fitness of RWE for regulatory decision-making; and
3. Advance a discussion towards objective evaluation of high-validity evidence that is fit for use in regulatory applications.

Stakeholders involved in generating, reviewing, or using RWE in regulatory and other decision-making applications would benefit from attending this Symposium.

Description: This symposium will offer perspectives from leaders in academia, industry, and regulatory agencies:

• Marie Bradley (US Food and Drug Administration): Health authority perspective on RWE for decision-making
• Heather Colvin (Johnson & Johnson): Overview of evolving regulatory guidance documents
• Dan Riskin (Verantos): Data reliability: implementing accuracy, completeness, and traceability in practice
• Dani Prieto-Alhambra (Oxford and Erasmus MC): European initiatives and public-private partnerships: data, methods, and tools for demonstrating RWE fitness for use

Josh Gagne (Johnson & Johnson) will open the session and outline the need for advancing our understanding of what constitutes RWE that is fit for regulatory decision making (15 minutes). Dr. Bradley will provide a status update on current thinking about RWE for decision-making from a regulatory perspective (15 minutes). Heather Colvin will provide an overview of existing regulatory guidance documents for both pharmaceutical and medical devices (15 minutes). Dr. Riskin will describe US FDA-funded demonstration projects aimed at measuring and optimizing data reliability (15 minutes). Prof. Prieto-Alhambra will discuss recent, ongoing, and upcoming initiatives designed to pilot RWE use cases for medicines, vaccines, and medical device development and regulatory decision-making (15 minutes). The session will include 25 minutes for moderated question and answer and interactive discussion between the speakers and the audience.