– Written by the Quality and Reusability of Real World Data project within the Real World Evidence Working Group.

View the FDA Guidance here.

In the realm of drug development, the use of real-world evidence (RWE) has emerged as a powerful tool for informing regulatory decisions and advancing medical innovation. With the FDA providing comprehensive guidelines on the use of RWE, sponsors play a pivotal role in ensuring compliance and adherence to regulatory standards throughout the drug development journey. Let’s delve into the key responsibilities sponsors should uphold when leveraging RWE in accordance with FDA regulations.

1. Early Engagement and Transparency: Sponsors must proactively engage with the FDA early in the drug development process. This entails seeking guidance on the appropriateness of using RWE, sharing draft study protocols and statistical analysis plans, and fostering transparency in data collection and reporting practices. By establishing open lines of communication with regulatory authorities, sponsors can address potential challenges and ensure alignment with regulatory expectations from the outset.

2. Data Access and Documentation: Access to high-quality, relevant data is paramount for generating robust RWE. Sponsors must discuss data access expectations with the FDA and ensure comprehensive documentation and submission of RWD for regulatory review. This includes providing detailed descriptions of data sources, methodologies, and analytical approaches employed, as well as ensuring the accuracy, completeness and reliability of the data presented.

3. Study Monitoring and Safety Reporting: Maintaining rigorous study monitoring practices is essential to safeguarding data integrity and ensuring compliance with regulatory requirements. Sponsors must implement robust quality control measures to monitor data accuracy, protocol adherence and human subject protection throughout the duration of the study. Additionally, sponsors are obligated to comply with post-marketing safety reporting regulations, promptly reporting any adverse events identified during the course of non-interventional studies.

4. Compliance with Regulatory Standards: Sponsors bear the responsibility of adhering to regulatory standards and guidelines governing the use of RWE in drug development. This includes compliance with relevant FDA regulations such as 21 CFR Part 312 (Investigational New Drug Application) and 21 CFR Part 11 (Electronic Records; Electronic Signatures), as well as ensuring alignment with applicable post-marketing safety reporting requirements. By maintaining strict adherence to regulatory standards, sponsors can demonstrate the reliability and validity of the RWE generated, thereby facilitating the regulatory approval process.

5. Accountability and Oversight: Sponsors are accountable for all aspects of the study, including study design, conduct, oversight and data management. This encompasses selecting qualified researchers, ensuring adherence to study protocols, maintaining comprehensive study records and facilitating access to relevant data for regulatory review. Sponsors should also establish clear lines of accountability for third-party organisations involved in study-related activities, ensuring that all entities adhere to regulatory standards and fulfil their respective responsibilities.

In conclusion, sponsors play a pivotal role in driving the responsible and ethical use of RWE in drug development. By embracing their responsibilities outlined by the FDA, sponsors can navigate the regulatory landscape with confidence, to ensure the integrity, reliability and compliance of RWE-based studies. Ultimately, this collaborative effort between sponsors and regulatory authorities paves the way for advancements in medical innovation, ultimately benefiting patients and improving healthcare outcomes.