Enhanced Risk-Based Quality Management (RBQM) Approaches for First-in-Human and/or Small Clinical Trials
Problem Description |
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Early development (ED), including first-in-human (FIH) and small clinical trials (e.g. rare diseases), involves complex, adaptive study designs that can be fast-paced or data-limited. Determining the appropriate balance for applying risk-based approaches and timing their implementation can be challenging, particularly when ensuring meaningful, value-added oversight. In FIH studies, participant safety is paramount, often leading to conservative approaches where many elements are considered critical, limiting effective risk prioritisation. Rare disease studies present additional challenges due to limited data, which restricts the ability to identify and assess emerging risks. This project will focus on defining robust strategies, processes, and best practices for identifying critical data and processes; implementing risk-based data review and monitoring approaches (including tSDV/tSDR); and applying analytical tools for central monitoring, with consideration of methodological limitations. It will also address threshold setting with limited patient data, targeted medical review, and proactive management of highly dynamic data, while aligning with related FIH and small study workstreams. |
Project Leads | |
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Agnes Verhoeven, Johnson & Johnson | |
Alicja Budek Mark, Genmab | |
Alex Pearce, PHUSE Project Coordinator |
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