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A couple of the Working Groups have expanded, with three new projects this quarter:   

• Estimands for RWD/RWE 

• Enhanced Risk-based Quality Management (RBQM) Approaches for First-in-Human and/or Small Clinical Trials 

• Communication of Version Metadata for Open-Source Languages   

Several additional projects are currently being developed for the next quarter.  

We have four more published deliverables, with many more in progress. The published deliverables this quarter are:  

• The Adverse Event Collection Recommendations project within the Safety Analytics Working Group has published a new white paper, Adverse Event Collection Recommendations. This paper provides recommendations for adverse event (AE) collection and associated instructions for Phase II to III clinical trials, building on standardisation efforts by the Clinical Data Interchange Standards Consortium (CDISC) and PHUSE. The primary intent is to reduce variation in AE collection and instructions to improve the site experience and the quality of the data.  

• The Dataset-JSON as Alternative Transport Format for Regulatory Submissions project within the Optimizing the Use of Data Standards Working Group has published a new white paper. A pilot was executed to test the feasibility of using Dataset-JSON as a transport format for study data submitted with regulatory applications. The goal was to demonstrate that Dataset-JSON can serve as a transport file for study data without loss of data and no significant impact on business operations.    

CDISC Dataset-JSON is a modern dataset format designed for data exchange. Many software applications already import and export JSON, and in the longer term more significant benefits will emerge as the constraints imposed by XPT are removed.   

Overall, the findings were consistent across industry and the FDA in supporting the use of Dataset-JSON as an alternative transport format for XPT v5. There were several key findings from the pilot, and the solutions to these will be implemented in Dataset-JSON v1.1 (due end of 2024).   

PHUSE and CDISC will continue to collaborate with the FDA/PMDA to discuss next steps for regulatory support. FDA/CDER/CBER testing strongly suggests that analytical tools used for regulatory review are updated to take advantage of the metadata and flexibility provided by the Dataset-JSON format.  

• The SEND Industry Feedback Survey project within the Nonclinical Topics Working Group has published the 2024 SEND Survey Results. The survey, which was presented at the PHUSE/FDA Computational Science Symposium (CSS) 2024, covered topics such as Manual Edits, Implementing SEND, Harmonisation, and Next Steps.  

• The Quality and Reusability of Real World Data project within the Real World Evidence Working Group has published its third blog post, RWD: The FDA’s Guidance on Assessing Registry Data to Support Regulatory Decision-Making. This blog post will explore the regulatory guidance’s main messages, the challenges we foresee in applying it, and how registry data can be used in regulatory applications.  

• The Nonclinical Study Data Reviewer's Guide is in the final review status.  

• The AE Groupings in Safety (AEGiS) white paper has been out for public review, and the team are working through the feedback. 

• The Treatment Emergent Definitions Recommendations white paper is going through publication.  

• The Good Transparency Practices white paper was shared for public review, and the team is continuing to work through the feedback.  

• The Optimizing the Use of Data Standards white paper is going through publication. 

• Best Data Practices for Rare Disease Patient Foundations and Researchers 

• Adverse Event Collection Recommendations