Back to: FDA Second Annual Animal Drug User Fee Educational Conference – July 2025
Lesson Content:
Real World Data/Evidence
Emily Smith
Primary Reviewer & Project Manager
Office of New Animal Drug Evaluation
Center for Veterinary Medicine, FDA
Q&A Session
Walt Ellenberg & CVM Q&A Panel Table
Abstract
This webinar details the US Food and Drug Administration’s (FDA) program concerning real world data (RWD) and real world evidence (RWE) for new animal drug approvals, as mandated by the Animal Drug and Animal Generic User Fee Amendments of 2018 and outlined in Guidance 266. RWD refers to data routinely collected from various sources related to animal health, productivity, or veterinary care, distinct from data gathered in a research setting. RWE is the clinical evidence of a new animal drug’s effectiveness derived from analyzing this RWD. While the primary focus is on effectiveness, RWD and RWE can also support safety. The presentation guides stakeholders through key questions for evaluating RWD/RWE’s appropriateness, starting with defining the regulatory purpose, which can range from supporting product development plans to generating substantial evidence of effectiveness. It then explores available data sources, such as health records, pharmacy data, and digital health, and emphasizes assessing the data’s fitness for use, which includes its relevance and reliability. The discussion also covers various study designs that utilize RWD, noting that traditional randomized controlled studies remain the gold standard when feasible. If a study using RWD is appropriate, sponsors must develop a protocol that clearly defines objectives, addresses potential sources of bias and confounding, details data curation processes, and outlines the statistical analysis plan. The FDA strongly recommends early and close communication with the Center for Veterinary Medicine for those considering using RWD and RWE, especially when developing protocols to generate evidence for drug approval.