Safety Physician Shionogi B.V. London, United Kingdom
Background: Signal assessment requires multiple data sources including individual case safety reports, aggregated data from passive surveillance systems or post-marketing safety studies and information from scientific literature. Increasingly real-world data (RWD) is being used to assess potential safety signals more accurately under normal conditions. However, RWD access, collection, and analysis can be time-consuming, while signal assessment must largely be conducted within specific time constraints.
Objectives: To evaluate the current state and potential scope for the rapid use of RWD to contribute to signal assessment.
Methods: A literature search was conducted to understand the current state of RWD use in signal assessment within pre-defined timeframes. Regulatory guidance, with a specific focus on the USA and EU, was reviewed to determine whether a framework or guidance exists on the expedient use of RWD in signal assessment. Scientific initiatives were explored, whether past, ongoing, or planned, in the context of potential rapid use in signal assessment. TransCelerate member companies were invited to participate in a voluntary survey in which current and prospective use of real-world data was explored in the context of signal assessment within time constraints.
Results: Little is published in the literature on rapid use of RWD data to support signal assessment. From the relevant articles identified, rapidity may be facilitated by establishing de-identified databases prior to analysis or by establishing standardised coding practices across data sources to speed up RWD analysis. Scientific initiatives such as FDA Sentinel and Observational Health Data Sciences and Informatics (OHDSI) are facilitating federalised de-identified databases with standardised coding and may represent a potential solution to expediting signal assessment using real-world data.
The TransCelerate member survey highlighted some ongoing barriers experienced by companies in rapidly leveraging real-world data for signal assessment, with commonly understood concepts and processes, a common framework, and clarity on the differences with traditional RWD studies, all being highlighted as areas of interest.
The format of protocols for RWD in rapid signal assessment as well as access to sources of data repositories were identified as key barriers for survey respondents.
Conclusions: RWD can be an important source of safety information, and there is growing use of RWD in rapid signal assessment. However, divergent approaches and a dearth of published literature on the topic is apparent. Understanding and addressing existing barriers and data access challenges to leveraging RWD for rapid signal assessment may offer opportunities for expanded use and increased quality.