Working Groups – Q3 2024 Summary
Welcome to the PHUSE Quarter 3 2024 Working Group Report Summary. Our Working Groups have had a productive summer, with more published deliverables and new projects. The Risk Based Quality Management Working Group is pleased to welcome Mireille Lovejoy as a new Lead. Mireille has extensive experience with RBQM, having previously led the development of processes, resources and technology underpinning the approach at two biopharmaceutical companies. You can learn more about Mireille via the Risk Based Quality Management page. Estimands for RWD/RWE is a new project within the RWE Working Group, led by Matt Baldwin. This project recently kicked off and aims to expand the principles set out in the FDA guidance Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products – February 2023 (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-design-and-conduct-externally-controlled-trials-drug-and-biological-products), but with more guidance and examples.

Working Groups – Q3 2024 Summary

Welcome to the PHUSE Quarter 3 2024 Working Group Report Summary. Our Working Groups have had a productive summer, with more published deliverables and new projects.

The Risk Based Quality Management Working Group is pleased to welcome Mireille Lovejoy as a new Lead. Mireille has extensive experience with RBQM, having previously led the development of processes, resources and technology underpinning the approach at two biopharmaceutical companies. You can learn more about Mireille via the Risk Based Quality Management page.

Estimands for RWD/RWE is a new project within the RWE Working Group, led by Matt Baldwin. This project recently kicked off and aims to expand the principles set out in the FDA guidance Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products – February 2023 (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-design-and-conduct-externally-controlled-trials-drug-and-biological-products), but with more guidance and examples.

New Projects This Quarter
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.

New Projects This Quarter

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

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

Closed Projects
Best Data Practices for Rare Disease Patient Foundations and Researchers Adverse Event Collection Recommendations

Published
We have four more published deliverables, with many more in progress. The published deliverables this quarter are: Adverse Event Collection Recommendations White Paper Adverse Event Collection Recommendations project, within the Safety Analytics Working Group have published a White Paper. 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. Dataset-JSON as Alternative Transport Format for Regulatory Submissions White Paper 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. SEND Industry Feedback Survey Results 2024. 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. Quality and Reusability of Real World Data Blog 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.

Published

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

Adverse Event Collection Recommendations White Paper

Adverse Event Collection Recommendations project, within the Safety Analytics Working Group have published a White Paper. 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.

Dataset-JSON as Alternative Transport Format for Regulatory Submissions White Paper

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.

SEND Industry Feedback Survey Results 2024.

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.

Quality and Reusability of Real World Data Blog

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.

Under Review
The Nonclinical Study Data Reviewer's Guide is in the final review stage. 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.

Under Review

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

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