Frequently Asked Questions: 2023 EMA and FDA Guidance
Unraveling the implications and intricacies of recent regulatory guidance: from screenshot requirements to patient-focused drug development and beyond
Bryan McDowell, M.Sc., M.B.A. – VP, eCOA Science and Consulting at Clario
In a recent LinkedIn poll, we asked our followers about their awareness of new eCOA-related regulatory guidance published this year. With over 140 responses, more than half indicated low to fairly low awareness. In a recent webinar, Bryan McDowell, Clario’s VP of eCOA Clinical Science and Consulting, delved into pivotal questions from clinical trial teams. Responses have been edited for clarity.
Q: Are screenshots for eCOA a critical part of IRB submission?
A: There has been a shift in mindset regarding the necessity of screenshots as we transition from paper to electronic records. While there was high demand for screenshots in the past, with various modalities and devices emerging, screenshots have become less important. The need for screenshots varies across countries and institutions. Regulatory authorities and ethics committees may review translations or IT implementations, but these processes shouldn’t rely solely on screenshots. Complex tools and devices make providing screenshots for all scenarios unrealistic. Instead, demonstrating proper implementation processes can alleviate the need for screenshots.
Q: How does the FDA’s Patient-Focused Drug Development guidance Part 4 address the issue of patient burden and how can we avoid missing data? What are the key differences between the FDA and the Patient Focused Guidance series and the FDA’s PRO guidance that came out in 2009?
A: The FDA’s Patient-Focused Drug Development (PFDD) series emphasizes patient input throughout drug development. It focuses on increasing participation convenience and value for patients, therefore lessening patient burden. The PFDD guidance is more comprehensive than the 2009 PRO guidance, offering detailed steps for gathering patient input throughout drug development, not just for labeling claims. When final, the PFDD series is expected to replace the 2009 guidance, while maintaining the principle of patient-centric drug development.
Q: Why is the latest patient document submission so complex?
A: The complexity arises from variations in national regulations across European countries. Although clear principles exist in Good Clinical Practices (GCP), implementation of these principles in national regulations leads to complexity. Patient documents must be categorized as fitting Part 1 or Part 2 of the PFDD, which varies based on different countries’ expectations. Over time, this complexity is expected to stabilize as regulations evolve.
Q: If we record the audit trail, even during a PRO-completion, how will we know if the initial entry was a tapping error or a hesitation from a patient? And what would we do with this data?
A: Audit trails pose challenges when patients make changes during completion. The requirement to document why and when a change occurs is unrealistic for patient-entered data like tapping errors or hesitations. This highlights the impracticality of audit trails for patient inputs made before questionnaire completion.
Q: There’s a feeling that decentralized trials are being pushed too hard and sometimes they’re not appropriate. What is the advice there?
A: The appropriateness of decentralized trials depends entirely on study design and objectives. Patient accessibility, safety, and scientific rigor are crucial. Science should guide trial design, determining whether decentralized elements align with the study’s goals. A flexible approach is key, considering various factors and evaluating if decentralized elements are suitable.
Q: What is the maximum amount of paper that could be used in a clinical trial that would be acceptable by regulators?
A: Minimizing paper is crucial to maintaining data quality. While exceptions exist, advocates discourage using paper, as it can degrade the quality of electronically collected data. In oncology, accurate early-stage data is particularly important, as these studies often support drug approvals.