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

• Real World Evidence has Estimands for RWD/RWE.
• Risk Based Quality Management has Enhanced Risk-Based 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.

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

We have four more published deliverables, with many more in the worksprogress. The published deliverables this quarter are: Data Standards

• Adverse Event Collection Recommendations White Paper

• The white paper was authored by members of the PHUSE data standards community, comprised of leaders and experts responsible for the governance and implementation of data standards across the biopharmaceutical industry.  

Ensuring Registry Data Relevance and Reliability for Regulatory Use  

• The Best Data Practices for Rare Disease Patient Foundations and Researcher within the Real World Evidence Working Group published a new deliverable. The poster – ‘Ensuring Registry Data Relevance and Reliability for Regulatory Use’ – focuses on data governance, integrity, fit-for-purpose data, and security/privacy. 

EU CTR Blog – Year 2 Update 

• The EU CTR Implementation project within the Data Transparency Working Group posted a blog post on EU CTR implementation. 
  The European Union (EU) Clinical Trials Regulation No 536/2014 (‘the Regulation’) has been operational since 31 January 2022. It covers interventional clinical trials of investigational medicinal products for human use and has important impacts on all aspects of trial set-up, operation and reporting in the EU and European Economic Area (EEA). 

QTL White Paper  

• The Quality Tolerance Limits project within the Risk Based Quality Management Working Group have published a new deliverable, a white paper on Assessing the Use of Quality Tolerance Limits in the Pharmaceutical Industry. This white paper aims to provide the reader with valuable insights into the depth and breadth of the use of QTLs and address how QTLs are used in a wider scope of implementing RBQM. Following all their hard work, the project is now closing out. 

Educate the General Population on Data Privacy and Data Sharing 

• The Educate the General Population on Data Privacy and Data Sharing project within the Data Transparency Working Group have published video 2: ‘What Will I Receive and When Will I Receive It?’  

SDTM ADaM Implementation FAQ  

• The team have published a new FAQ within Validation/Conformance Rules and they continue to receive and work through more FAQs. 

The Quality and Reusability of Real World Data project within the Real World Evidence Working Group has published two new blog posts.

• The Understanding the Data Quality Issues in Real-World Data Through Real-World Examples blog post takes a closer look at the experiences shared within the Working Group of the types of data quality hurdles encountered when using real-world data (RWD).
• The Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products blog post explores the use of real-world evidence (RWE) as a powerful tool for informing regulatory decisions and advancing medical innovation.

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.

• 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.