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We’ve just wrapped up another successful CDISC + TMF EU Interchange event! This year’s event, which was attended by 352 people, featured parallel sessions dedicated to the Trial Master File (TMF) Reference Model, which has been a part of CDISC standard development initiatives for over a year now. This addition is crucial, as documentation is a serious and important aspect of our industry. Each of us must contribute to this documentation effort and stick to the principle “if it is not documented, it did not happen.”<\/p>\n
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Though not particularly my area of expertise, I attended one of the TMF parallel streams, specifically the “TMF Essentials” stream. Here, I gained insights into the TMF standard and realized the urgent need for standardization in how TMFs are managed and structured. It appears that companies still employ various and different approaches. While we at Cytel, as a biometrics CRO, do not directly manage TMFs, we generate artifacts that eventually find their place in the sponsor TMF. Hence, it’s crucial to be aware of content requirements, particularly what constitutes “Essential Documents,” and to proactively engage with our sponsors in TMF planning at the trial’s outset.<\/p>\n
For biometrics colleagues in the US, there will be specific training on the “TMF Reference Model for Biometrics Professionals\u201a” at the next CDISC US Interchange<\/a> in October.<\/p>\n This year, Cytel once again played a significant role in organizing the event. My colleague, Silvia Faini, and I are part of the CDISC EU committee responsible for defining the agenda. In addition to moderating the \u201cADaM\u201d and \u201cPiloting JSON\u201d streams, we also delivered two presentations:<\/p>\n <\/p>\n Cytel was also among the exhibitors, with my colleagues Russel Brown and Cyril Sombrin also participating.<\/p>\n The event commenced with visionary presentations by the new CDISC president, Chris Decker, and a CDISC roadmap presentation by Peter Van Reusel, Chief Standards Officer at CDISC. From Chris\u2019s presentation I\u2019d like to highlight his possible \u201cback to the future\u201d comment:<\/p>\n \u201cProvide regulatory agencies a connected and traceable submission package allowing reviewers to click their way from the clinical study report to analysis to data to protocol increasing confidence in the data and results.\u201d<\/p>\n The keynote speech by Rob DiCicco from TransCelerate BioPharma Inc. also set the tone for the event \u201cOur Industry Journey towards Digital Transformation.\u201d<\/p>\n I then jumped into the \u201cNavigating Submission Pathways\u201d stream as I had scheduled my presentation. Worth mentioning from this stream was the presentation from Jintao Shi \u201cUnderstand PFDD Guidance Series and the Impact on Data Submission,\u201d which discussed two recent FDA guidance documents and their implications on SDTM and ADaM datasets packages:<\/p>\n <\/p>\n While I was engaged in the \u201cNavigating Submission Pathways\u201d stream, Silvia attended the CDISC CORE<\/a> Workshop, focusing on the CDISC Open Rules Engine project. She found the workshop highly impressive and is thinking of joining the team. Many companies are already integrating CORE into their processes and tools, as discussed in the \u201cCore Implementation\u201d stream. Novo Nordisk, SGS, Argenx, and OCS shared their experiences as the first implementers of CORE rules. Clear rules are urgently needed to provide precise outcomes and improve reporting.<\/p>\n Silvia moderated the \u201cPivoting JSON\u201d stream. There are high expectations from this project within our industry, as there is a pressing need for a better method of exchanging datasets beyond SAS XPT. CDISC, with Sam Hume, discussed the results of the Datasets JSON Pilot, an initiative that actively involved the US FDA. While a full \u201creadout\u201d will be disclosed at the PHUSE CSS<\/a> conference in June 2024, Sam shared the progress made so far. The pilot demonstrated that Dataset-JSON can transport information successfully (verified by FDA testing on their environment). However, improvements are needed in guidance and the standard itself, along with enhancing tools such as Python conversion software. The team is still seeking volunteers to join<\/a> version 1.1 of the pilot.<\/p>\n I also attended \u201cSubmission Experience\u201d stream where my Italian friends from Chiesi Farmaceutici, Rebecca Moretti and Rocco Calabro\u2019, presented their experience submitting data packages to the NMPA Chinese health authority. Their presentation was recognized as the best of the entire conference. Another notable presentation came from Marilina Castellano of the Uppsala Monitoring Centre, who provided details about WHODrug and its implications for SDTM mapping.<\/p>\n On the regulatory side, all three main health authorities were represented live. Though FDA and PMDA provided no major updates, it\u2019s worth noting that PMDA mentioned \u201cForm A\u201d is no longer required for submission. EMA provided updates on current initiatives towards data standardization and submission, including the \u201cSubmission of individual patient data from clinical trials.\u201d Eftychia Eirini Psarelli presented some key learnings from the ongoing pilot project and announced the interim report\u2019s release by Q2 2024. Final decisions regarding EMA requirements for dataset submission are expected by late 2024 or early 2025.<\/p>\n From the \u201cADaM\u201d stream that I moderated, I\u2019d like to highlight Cedric Davister\u2019s (Merck KGaA) and Alexandra Cochinaire\u2019s (Johnson and Johnson) presentation, both members of the PHUSE Estimands Working Group. They provided an overview of the draft white paper on the Implementation of ICH E9(R1) Estimands Framework using Data Standards<\/a>. Their focus was on the implications of the Estimands Framework on ADaM datasets and, in particular, how to handle \u201cIntercurrent Events.\u201d<\/p>\n Many interesting presentations were scheduled on the agenda that I was unable to attend. Notably, the entire \u201cAI in Standards\u201d stream explored the use of Large Language Models (LLM) in aiding standard implementation, presented by SAS in collaboration with Deda Group and similarly with the Novo Nordisk with a practical application of AI agents as Co-Scientists.<\/p>\n I also had the pleasure of coordinating the poster session, reviewing drafts, and suggesting improvements to the authors. We had a total of 16 posters covering topics ranging from the use of CDISC Library to SDTM and ADaM implementation showcases, as well as Open-Source topics. The winner was Siddhant Prabhu from Novo Nordisk with \u201cData Quality Considerations when Providing Large Volumes of Consolidated Clinical Trial Data for Secondary Use.\u201d His work provided an interesting application to assess quality and adherence to SDTM standards in current and historical Novo Nordisk studies.<\/p>\n\n
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