Ensuring Patient Registries Support Regulatory Decision-Making

Description: Patient registries can be a useful tool in collecting data on the disease that can inform drug development and regulatory decision-making. Registries may provide data on the natural history of a disease, burden of disease and burden of the current treatment standards of care. Registries often provide information on the impact of available treatments, or the lack thereof, for a given disease population. Importantly, registry data serves as an important component for demonstrating evidence of the safety, effectiveness and value of potential treatments and cures in the real-world setting. It is important for all stakeholders, including registry holders, patients, caregivers, and other personnel involved in the creation of patient registries, and those participating in the collection and analysis of registry data, to have the same foundational knowledge, as regulators and drug development sponsors, for how this important data can be used in drug development and regulatory decision making. These stakeholders, often, have similar goals (i.e., assessing how patients feel, function and survive, and the current state of the disease population, etc.) to create treatments/cures where possible for both common and rare diseases.
Both the FDA and EU guidance define registries similarly as an organized system that collects clinical and other data in a standardized format for a population defined by a particular disease, condition, or exposure. Establishing a registry can be extremely important to a rare disease community especially when, there is a small patient population, the disease is not well characterized, there is poor understanding of the natural history of the disease and its progression, and there are no validated biomarkers or clear clinical endpoints. It is imperative that all stakeholder regulatory requirements and are intentional when creating patient registries, so that data collection efforts are not duplicated or wasted, and data from registries can inform regulatory decision-making.