Working Group Report – Q2 2024

Working Groups – Q2 2024 Summary

Welcome to the PHUSE Quarter 2 Working Group Report. The second quarter of the year has been very busy for all Working Groups, with a lot of activity including new deliverables and events.

The Optimizing the Use of Data Standards Working Group are pleased to welcome two new Leads to the team: Sandra VanPelt Nguyen and Edwin Van Stein. Sandra has been involved with PHUSE since 2015, leading and supporting PHUSE projects under the Optimizing the Use of Data Standards and the Real World Evidence Working Groups. Edwin is an active PHUSE member, contributing to the PHUSE EU Connect as a presenter, Stream Chair and Connect Chair, and as a PHUSE Wiki Administrator. He is currently a permanent member of the EU and US Connect Planning Committees. Sandra and Edwin will be joining the current Leads, Jane Owens and Janet Low. You can learn more about the new Leads via the Optimizing the Use of Data Standards page.

The Safety Analytics Working Group also welcomes two new Leads: Mac Gordon and Ellis Unger. Mac Gordon has a master’s in statistics and graduate certificates in public health, pharmacovigilance and pharmacoepidemiology and has been with Johnson & Johnson for 15 years and in industry for 20 years. Mac has been involved with lupus research since joining the organisation, with focus areas in late-development immunology and clinical trial safety. Ellis Unger, MD, is a Principal Drug Regulatory Expert at Hyman, Phelps & McNamara, a leading consulting FDA law firm. Dr Unger is a board-certified cardiologist, who retired from the U.S. Food and Drug Administration following a 24-year career, where he served in senior leadership roles in the Office of New Drugs, Center for Drug Evaluation and Research (CDER). Mac and Dr Unger will be joining Mary Nilsson, Greg Ball and Scott Proestel. You can learn more about the new Leads on the Safety Analytics page.

Our data standards community, which is comprised of leaders and experts responsible for the governance and implementation of data standards across the biopharmaceutical industry, authored a new white paper, which highlights current challenges in data standardisation across the biopharma industry and identifies opportunities where we can work together to tackle them.

New Projects This Quarter

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

Published

It’s been another busy quarter across all eight Working Groups. There have been eight published deliverables, with many more in the works. The published deliverables are:

Data Standards 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.

Under Review

Implementation of Estimands (ICH E9 (R1)) using Data Standards had an incredible response to their white paper during their public review earlier this year. They are still working through all the comments and getting the final version ready for publication.
The Real World Evidence Working Group will soon be publishing a blog post titled ‘Understanding the Data Quality Issues in Real World Data through Real World Examples’.
The Adverse Event Collection Recommendations white paper is going through a final review and will soon be ready for publishing.
The Treatment Emergent Definitions Recommendations white paper was shared for public review and the team are now working through the feedback.
The Good Transparency Practices white paper was shared for public review and the team are now working through the feedback.