Content Comparison

In its 14th year, the PHUSE Computational Science Symposium (CSS) will be in Utrecht, the Netherlands 20-21 May.

At the heart of the event is the PHUSE Working Groups’ mission to tackle unmet computational science needs in support of health product development and regulatory review, ultimately bringing safe and effective medical products to those who need them.

The CSS agenda will feature a variety of activities, including expert-led plenary sessions, interactive workshops and dedicated Working Group Breakout Sessions that drive forward existing initiatives and explore new ones. Attendees will also have the chance to attend the Poster Session, providing the opportunity to share knowledge and engage in discussions on various computational science topics.

PHUSE is excited to welcome attendees from across pharma, CROs, academia, health authorities, technology vendors and SDOs to come together for this collaborative and forward-thinking experience. 

Please visit the PHUSE website for more information.

Call for Speakers is officially open for the Data Transparency Autumn Event 2025

The Optimizing the Use of Data Standards Working Group has a new project FDA Requests Public Comment on CDISC Dataset-JSON Standard and is calling for volunteers!

The U.S. Food and Drug Administration (FDA) has recently published a Federal Register Notice exploring the potential adoption of CDISC Dataset-JSON v1.1 as a new exchange standard for electronic study data submissions potentially replace the SAS v5 XPORT Transport Format (XPT) in the long term.

The FDA is seeking comments on whether to accept Dataset-JSON for regulatory applications in the future, specifically regarding:

• The risks and benefits of industry adoption of Dataset-JSON as a new exchange standard.

• Any integration challenges with existing tools and systems.

PHUSE, given its expertise and commitment to advancing the implementation of data standards within the pharmaceutical industry, and its recent PHUSE/CDISC pilot, is launching a short-term project to collect and synthesize comments from its members on this Federal Register Notice.

Building on the PHUSE/CDISC pilot this is an opportunity to:

• Influence the future of regulatory data submissions.

• Share your expertise and perspectives on data standards.

• Collaborate with fellow PHUSE members on a timely and impactful initiative.

• Contribute to PHUSE's position on this important topic.

We are seeking volunteers to participate in this project.

The timeline for this project will be relatively short, aligning with the FDA's comment period. We anticipate the main activities to take place during May.

If you are interested in volunteering for this important project and contributing your expertise, please express your interest by completing this Working Group Project Membership Form.

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This is a fantastic opportunity for those passionate about data transparency to share their bold ideas, fresh perspectives and real-world experience.

The event will take place virtually 16–18 September, meaning you can participate from anywhere in the world! Plus, there’s no need to block out the whole day, as sessions are delivered in short bite-sized chunks from 10:00-12:30 (EDT) / 15:00-17:30 (BST) / 16:00-18:30 (CEST)

Submit your 150-word abstract by 12 June for the chance to take part in one of the most focused, forward-looking events in the transparency space.

Webinar 2: Estimands in Real-World Evidence Studies

This webinar will explore the pivotal role of estimands in real-world evidence (RWE) studies, bridging the gap between regulatory guidance and practical implementation. The session will address challenges unique to RWE settings, such as heterogeneous patient populations, complex treatment regimens, and the impact of intercurrent events on study outcomes with a focus on generating RWE that can inform regulatory decision-making. Through practical examples, case studies, and an engaging panel discussion featuring domain experts, this session will highlight best practices for defining estimands that enhance the interpretability and reliability of RWE findings. Participants will leave with a systematic approach to estimand definition, empowered to conduct RWE studies that are robust, actionable, and aligned with evolving regulatory standards.

This webinar will be held virtually on 5 June 2025 at 11:00 (EDT) / 16:00 (BST) / 17:00 (CEST)

Digital health technologies (DHTs), such as wearable accelerometers, are increasingly being adopted in clinical trials, as they enable objective and continuous evaluations of health parameters, functions and behaviours in the participant’s naturalistic environment. The continuous data stream provided by DHTs combined with artificial intelligence/machine learning (AI/ML) tools can lead to the identification of novel digital endpoints (NDEs) and measuring objective outcomes that are meaningful to patients. These NDEs can support clinicians with early diagnosis, prognosis, and personalised treatments of a wide range of diseases.

To address the need to develop and validate NDEs before their large-scale deployment in clinical trials and gather evidence supporting their regulatory adoption, at Pfizer we have collected in-clinic and at-home data from hundreds of healthy volunteers across age ranges, spanning paediatrics to geriatrics, and device locations. In the first part of this presentation, considerations about the selection of fit-for-purpose DHTs and the validation of AI-driven algorithms for NDEs of gait and physical activity will be discussed as compared to standard measurements. Since understanding individual perception towards using new technologies is crucial for clinical adoption of NDEs, the evaluation of self-reported measures of usability, acceptability, and level of continued usage will also be addressed. Results suggest high reliability and accuracy of DHT-derived gait and physical activity endpoints, for example by effectively tracking walking at different speeds, while ensuring participants’ comfort in wearing DHTs. Finally, the deployment of the validated AI-driven digital biomarkers in clinical studies will be presented, with particular focus on older individuals at risk of frailty and malnutrition.

This Community Forum will be held virtually on 18 June at 11:00 (EDT) / 16:00 (BST) / 17:00 (CEST)

End-to-End RBQM Webinar: Value-Driven Clinical Data Review, Together

Clinical data review is no longer just a technical checkbox – it’s a strategic, cross-functional effort that can make or break the success of a clinical trial. When teams collaborate across functions, align on risk, and focus on value-driven review, the result is more reliable, coherent and compliant trial data.

In this session, we’ll explore how to move beyond traditional data cleaning by embracing a collaborative, risk-based approach. Learn how functional interdependencies – when properly understood and leveraged – can drive smarter, more efficient reviews.

We’ll also host a dynamic roundtable featuring voices from big pharma, tech and CROs, who will share real-world insights, challenges, and best practices for building effective, cross-functional data review strategies. Whether you’re in data management, clinical operations, biostatistics, or a supporting function, this session will offer actionable strategies to foster collaboration and value-driven data review processes.

This webinar will be held virtually on 26 June at 10:00 (EDT)/ 15:00 (BST) / 16:00 (CEST)

If you would like to get involved in a PHUSE Working Group Project, please explore the projects via the Volunteer Board and contact the PHUSE Office on workinggroups@phuse.global to express your interest.