(132) A Framework for Rapid Analysis of Real-World Data in Safety Signal Assessment

Background: Real-world data (RWD) can provide important insights for safety signal assessments, but formal RWD studies with fully specified protocols are often resource and time-demanding and seldom feasible within regulatory timelines for signal assessment. TransCelerate BioPharma formed the Rapid Signal Assessment using RWD (RSA-RWD) initiative to identify opportunities to address these challenges. Survey results from 18 TransCelerate member companies identified the potential value of a clearly defined process framework and alternative to full study protocol to support safety management teams conducting RWD analyses in a rapid approach during a signal assessment.

Objectives: To develop a framework and alternative to full protocol for rapid analysis of RWD in safety signal assessment.

Methods: An interdisciplinary team of experts from TransCelerate member companies was formed to develop the RSA-RWD framework. The team conducted a scientific literature review to understand the status of RSA-RWD. Team members further built on their experience within their member companies to identify best practices and potential opportunities to conduct rapid analyses of RWD to support safety signal assessment.
In addition, a Rapid RWD Analysis Plan Template for Signal Assessment (RASA) was developed based on existing standard templates for fully specified study protocols. Essential sections from a full protocol were selected and tailored to design a simplified version of a protocol that can be used for RSA-RWD.

Results: The framework includes an end-to-end process map for conducting RSA-RWD, key considerations for rapidity, a glossary and 4 example use cases. The process is summarized into three main stages including: 1) Problem Formulation, Planning, and Design, which consists of formulating a research question, identifying data sources, and developing an RWD analysis plan; 2) Analysis Execution, which delineates the preparation and execution of the analysis, and 3) Results Interpretation and Communication, which outlines a process of interpreting results and disseminating findings internally and externally. A list of process activities and opportunities to expedite the process is provided for each step. The framework applies the principles of accurate evidence generation, adding value to signal assessment, and maintaining internal and external transparency.
The RASA template offers an alternative to a full protocol to increase efficiency for RSA-RWD whilst maintaining transparency of the analysis.

Conclusions: Both solutions, framework and RASA template, could support safety management teams in pharmaceutical companies or researchers who want to begin or increase the use of RWD to complement safety signal assessments and thereby benefit patient safety.