Sarah Dolan was diagnosed with young onset Parkinson’s Disease 7 years ago. After leaving a 30-year career in the biopharma industry she has participated in multiple clinical trials, is a current member of Critical Path to Parkinson’s Endpoints Team, a current Consumer Representative for the FDA PCNS Advisory Committee, and is an active Ambassador for the Davis Phinney Foundation. Sarah lives in a cabin on a river in Cody, Wyoming and loves cycling, her horses, and spending time with her husband and three children.

Join your TMF management industry colleagues in this session to explore the latest thinking behind the TMF Reference Model’s next version! This engaging, panel-style discussion led by members of the V4 TMF Project Management Committee alongside select industry thought leaders, to unveil and explain foundational concepts slated for the new release.

The CDISC Tobacco Implementation Guide (TIG) v1.0, developed with the FDA Center for Tobacco Products (CTP), provides a framework for collecting, analysing, and exchanging tobacco product data to ensure consistent standards and high data quality. The accompanying TIG Conformance Rules v1.0 facilitate the creation of accurate, compliant data packages aligned with TIG standards. These rules build on existing SENDIG, SDTMIG, and ADaMIG rules, while introducing new ones for tobacco-specific use cases.

This presentation offers a high-level overview of TIG v1.0 and the pilot submission project, along with insights into integrating TIG rules into CDISC Open Rules. The process revealed key differences between implementations and provided valuable feedback to strengthen existing conformance rules.  

By sharing insights and experiences, the importance of these conformance rules in achieving consistent and compliant data will be emphasized, encouraging both broader industry adoption of the TIG framework and continued enhancement of conformance rule standards across implementations. 

From the e-protocol to the analysis results and back, this presentation will illustrate a few interconnected process tasks in clinical research and summarize a few process challenges. The process tasks will be demonstrated using a Proof of Concept (POC) implementation based on open-source software, either freely available or through other supported agreements.

By leveraging the use of data based on CDISC modeling standards USDM, CDASH, SDTM, ADaM, ARS, underlined CT, plus data-exchange standards ODM, Define-XML and Dataset-JSON, along with the CDISC CORE initiative, the presenters will cover some potential gaps that would need to be addressed to maintain data integrity and consistency across the different standards and data-exchange process.
This presentation aims at encouraging users to start taking steps towards a consistent implementation of CDISC standards from start to end, using available open-source software and service resources. Sponsors could start gradually growing their 360i capabilities as more knowledge is acquired.

Faro’s AI-powered protocol digitization solution transforms static clinical study protocols into structured, reusable data that drives efficiency across the drug development lifecycle.

Zifo’s solution transforms protocols from static documents into reusable, machine-readable assets - accelerating study design, standardization, and reuse.

The Smart BC suite, submitted by Saama, is a sophisticated framework designed to extract, standardize, and link Biomedical Concepts from clinical documents using an advanced AI-driven approach.

Lindus’ BC Registry Framework registers new BCs, checks against local registries and CDISC Library for matches, then falls back to an advanced NCIT ontology search.

Merck’s solution is an open-source traceability engine that automatically pulls together study files like protocols, CRFs, SDTM, ADaM, and TLFs. The engine then builds one clear “lineage graph” that shows how results, variables, and endpoints connect back to where they came.

Zifo establishes end-to-end traceability across trial artifacts by applying AI-driven metadata extraction and CDISC Standards, enabling dependency queries and impact analysis from study design through statistical outputs.

In clinical trials, the TMF serves as the backbone of regulatory compliance. Far beyond technology and services, the success of a TMF program hinges on the underlying cultural dynamics. This presentation, a collaboration between Intellia and Cencora focuses on how the critical role TMF Culture plays in shaping effective sponsor/CRO/Vendor relationships and ensuring the operational success of Sponsor’s TMF program and CRO/Vendors.

Our session will discuss four key strategies for fostering an environment where all
stakeholders—Sponsors, CROs, and Vendors—are aligned and motivated towards
common goals.

These four key strategies are: 

• Ensuring Operational Partnership Success
• Cultural Missteps and Lessons Learned
• Culture and Accountability
• Engagement and Relationship Building

By weaving together these strategies, our presentation seeks to illuminate how to make an impact of TMF culture within organizations. We aim to provide attendees with actionable insights, drawn from our diverse experiences, to enhance their own TMF culture.

There was no active community of TMF’ers in Japan, and each company has been struggling to manage challenges in TMF operation. To address these aspects, CDISC Japan User Group (CJUG) launched a TMF team in January 2025, bringing together 20+ TMF’ers from pharma, CRO, tech service provider, consultant and academia. We focus on 4 key challenges including ICH-E6(R3) vs TMF, Sponsor-CRO collaboration, TMF in academia and TMF RM penetration in Japan.
One of the practical challenges for Japanese pharma/CRO is managing Japan-specific regulatory requirements while complying with global standards from EMA, MHRA, and FDA. This presentation highlights a case study on safety information documentation in Japan, which uniquely requires IRB review for study continuation rather than just PI’s review as in EU/US. This process creates additional documentation and potential duplication, so CJUG TMF team aims to streamline TMF management practices while fostering greater integration with the global TMF community.

• Matt Lowery, MGH
• Colleen Butler, Syneos
• Steph Viscomi, Apellis

The digital transformation of clinical research is redefining how protocols are developed, managed, and executed across trials. A digital protocol can empower research teams to design studies with an increased probability of success by integrating real-time analytics and streamlining collaboration among stakeholders.  This paradigm shift from documents to data enables new efficiencies in study startup, faster and more representative patient recruitment, automation, and superior data quality throughout the clinical trial lifecycle.

Join this session to explore how your organization can embrace this transformation. Learn about implementation options, common challenges, and tools to help you succeed.

A robust standards metadata repository lays a strong foundation upon which automation and other cycle time reductions can be built. AstraZeneca is amid transforming its delivery of Study Instance Metadata through the implementation of a standards metadata repository, moving away from Excel spreadsheets. It was important to have a system that provided the study instance metadata in a machine-readable format to easily be consumed by other systems to enable automation and data re-use. We undertook a phased approach of first migrating global clinical data standards into the metadata repository followed by introducing new practices for building study instance metadata. We have laid a strong foundation that will allow us consistent re-use of metadata and metadata driven automation across the organization. 

Comprehensive data collection from underrepresented populations is essential to advancing health equity in clinical research. Historically, these groups have been underrepresented, leading to gaps in regulatory submissions and scientific generalizability. This presentation evaluates the impact of broadening data acquisition to include diverse demographic, socioeconomic, and clinical variables, aligned with CDISC standards for standardized data representation. Incorporating these enriched datasets enhances trial validity and regulatory readiness. Additionally, by leveraging CDISC compliant frameworks, clinical trial teams can accelerate the integration of inclusive data elements and improve the timeliness and quality of reporting. This data-driven approach fosters more representative clinical evidence, ultimately driving better health outcomes and regulatory compliance. 

The FDA's Real-Time Oncology Review (RTOR) program accelerates the review of oncology clinical trials by allowing for the early submission of top-line results and datasets. However, RTOR requires meticulous preparation and adherence to the FDA's specifications, particularly concerning the submission of comprehensive Analysis Data Model (ADaM) datasets based on data specifications provided by the FDA Oncology Center of Excellence (OCE) and Office of Oncologic Diseases (OOD) Safety Team.

This presentation delves into some of the nuances and challenges related to the implementation of these specifications, focusing on the adaptation of ADaM dataset requirements for RTOR.A key area of focus will be the non-standard Adverse Events Analysis Dataset for Cytokine Release Syndrome (CRS) and Neurotoxicity (NT) – ADCRSNT. 

This paper aims to present an overview of data traceability flow, data standard requirements, and lessons learned during the preparation of the Study Data Submission Plan (SDSP), tabulations, analyses, and BIMO package submissions. It will address key differences between the agency and its divisions, focusing on the FDA (CBER and CDER), PMDA, and NMPA. Special emphasis will be placed on guidance requirements, including TCG, technical specifications, rejection criteria, and validation rules, as well as the importance of documenting these elements. The study start dates will influence the implementation of data standards and their implications for submissions. We will highlight the differences between the SDSP and the SDSP Appendix, as well as the size requirements for SAS XPT files. These documents are subject to ongoing changes, which impact submissions throughout the product development lifecycle.

In this session we will introduce a proposal developed under the TMF RM V4 initiative to define the core record types for computerized systems used in clinical trials. These records are those that sponsors and their service providers should consider essential for inclusion in the Trial Master File (TMF), supporting both oversight activities and regulatory submissions.

The proposal reflects inputs from a multidisciplinary consultation and is aligned with the guiding principles of the TMF RM V4 initiative and CDISC standards. It is designed to meet known regulatory expectations while offering a practical and adaptable approach for diverse sponsor organizations and trial needs.

Additionally, we will present a template for documenting the trial system inventory, aimed at enhancing inspection readiness and facilitating efficient retrieval of both trial-specific and enterprise-level records.

This presentation will share how Beacon Therapeutics, a mid-sized biotech company, ensures high-quality and compliant oversight of the Trial Master File (TMF). Although many TMF Quality Control (QC) tasks are often outsourced, Beacon remains accountable for inspection readiness and regulatory compliance. From the perspective of the Sr. Clinical Documentation Manager, the session will cover practical strategies, governance frameworks, defined roles, SOPs, vendor oversight, and escalation pathways. The focus will be on taking a proactive, data-driven QC oversight using key metrics, rework frequency, and common errors. The presentation will show how regular QC reviews, spot-checks, and vendor monitoring can help improve quality over time and identify areas needing support or training.  It will also touch on effective communication, audit-ready documentation, and governance routines. Attendees will leave with practical strategies and insights to implement a consistent, compliant, and inspection-ready TMF QC oversight model across studies.

The partnership between Sponsors and External Service Providers (ESP/CRO) is vital for effective TMF management. A proactive approach to defining TMF responsibilities, ESP deliverables, and associated costs early - during RFI, RFP or contracting phase- enhances operational efficiency and drives inspection readiness improvements. 

Misaligned expectations and poorly defined budget assumptions often lead to downstream inefficiencies. Clearly defining scope and budget helps prevent miscommunication, compliance risks, and costly change orders. 

Sponsors should focus on understanding how ESPs will manage the TMF and what services are included. In turn, ESPs should maintain transparency on their pricing models and TMF capabilities. 

Standardizing definitions and cost structures across partnerships minimizes redundancy, improves governance, and ensures TMF quality. Leveraging this unified framework grounded in mutual agreement, the TMF plan delineates this defined responsibility and accountability for all TMF related activities. 

Ultimately, early budget alignment strengthens partnerships and drives a more efficient, inspection-ready TMF management model.

The CDISC 360i initiative was launched to realize CDISC’s mission of creating connected standards across the study information lifecycle - enabling accessible, interoperable, and reusable data to support more meaningful and efficient clinical research. The program aims to deliver a complete, metadata-driven study package: from protocol design to submission, including test data, executable tools, and an automated pipeline for generating analysis results.  

In this presentation, we will showcase how OpenStudyBuilder is integrated into the CDISC 360i ecosystem. As one of the key components in this collaborative initiative, OpenStudyBuilder functions as a central clinical metadata and study definition repository. We will highlight its role in supporting end-to-end automation and standardization, and how it interfaces with other tools and solutions within the 360i framework. Join us to learn how OpenStudyBuilder contributes to making the CDISC 360i vision a reality - and how its integration helps pave the way for a more streamlined and interoperable future in clinical research. 

In January 2025, a CDISC working group was established to define and model Analysis Concepts, aiming to enhance automation in clinical research as part of the CDISC 360i initiative. While the Unified Study Definition Model (USDM) standardizes key elements like study objectives and endpoints, there is a gap in structured metadata for derived and analyzed data. Analysis Concepts aim to fill this gap by providing a standardized framework for translating clinical questions into analytical outputs, supporting the creation of a digital Statistical Analysis Plan (eSAP), an    d informing data collection and analysis programming. This work aligns with CDISC's Analysis Results Standards (ARS), which describe statistical results and their derivation. Analysis Concepts focus on clinical intent and analytical approach, serving as upstream metadata for the ARS framework. The presentation will cover the rationale, key components, early models, and future directions for standardizing analysis planning. 

• Jamie Toth, BeOne Medicines USA, Inc.
• Bryan Souder, Merck
• Deb Wells, Novartis

FDA guidance requires Real World Data (RWD) submissions to follow formats listed in the FDA’s Data Standards Catalog, currently CDISC SDTM. However, transforming RWD—collected from billing and clinical care sources like claims and electronic health records—into SDTM is challenging. SDTM is designed for clinical research, and this complex transformation often causes data loss and unquantifiable biases, making RWD unreliable for regulatory use. To address this, CDISC’s RWD Lineage project is developing a standardized metadata model that provides detailed lineage and traceability for each RWD data point. This approach enables auditing of source data and assessment of data transformation quality. By standardizing lineage across diverse data sources, the project aims to unify tools, workflows, and analytics, supporting validation and regulatory audits. This presentation will share the latest progress and findings from the RWD Lineage team, highlighting efforts to improve the reliability and regulatory acceptance of RWD through enhanced data transparency and traceability.  

As digital health technologies like wearables increasingly capture clinical trial data, sponsors face challenges in integrating and mapping this data to CDISC standards for regulatory submissions. The diversity of data sources, high granularity, large volumes, use of software algorithms, and inclusion of supportive contextual data complicate this process. Current CDISC guidance offers limited examples, making standardization difficult. Some data may not fit clearly into existing SDTM domains, while other data presents challenges related to how it’s collected or reported by devices or vendors. This presentation will examine these challenges in mapping digital health technology (DHT) data and outline Pfizer’s approach to determining target mappings. It will also share practical examples illustrating how Pfizer navigates the complexities of aligning diverse DHT data with CDISC standards to support regulatory compliance and data integrity.  

The growing use of Real-World Data (RWD)—from electronic health records and claims—is transforming regulatory submissions for drugs and biologics, especially as digital health technologies expand. However, Real-World Evidence (RWE) derived from RWD presents challenges for regulatory reviewers due to missing contextual variables not captured under study protocols. This paper outlines key issues and proposes solutions using existing standards for observational and healthcare delivery data. It focuses on enhancing CDISC SDTM, particularly the Subject Visits (SV) domain, to better support RWE submissions. By examining standards like HL7 FHIR, OMOP, and Sentinel, the authors suggest updates to represent patient encounters with added context—such as provider identity, care site, and encounter details. These enhancements aim to improve the interpretability and reliability of RWD, reduce bias, and support regulatory analysis. The recommendations also contribute to ongoing efforts by standards development organizations to harmonize real-world data with randomized clinical trial frameworks.

• Rob Jones, Cencora
• Guillaume Gerard, Agatha
• Eleanor Hewes, Syneos

Moderator: Aaron Grant, Just in Time GCP

Panelists:

• Paul Carter, Montrium
• Gui Gerard, Agatha
• Jim Horstman, Veeva
• Rob Jones, Cencora
• Ricky Lakhani, Phamaseal
• Chris Sigley, Arkivum
• Jay Smith, Transperfect
• Leah Weitz, Egnyte