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PHUSE collaborations are organised into a number of specialist Working Groups, each with a broad topic area. The Working Groups have specific projects designed to achieve a set of particular objectives. This page will highlight the latest news and information from our projects. Participation is open to anyone who wants to contribute and if you would like to get involved, please email workinggroups@phuse.global. |
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RWD for Regulatory Decision-Making: Learnings from Use Cases and Demonstration Projects
FDA guidance is clear that real world data (RWD) may be acceptable for regulatory approval when a randomised controlled trial is not feasible and the validity and trustworthiness of the real world study results are clearly demonstrated. To provide more operationalised guidance for meeting these standards, the presenters will share learnings from FDA use cases and ongoing demonstration projects.
This Community Forum will be taking place virtually on 28 January at 14:00-15:00 (GMT) / 9:00-10:00 (EDT) / 15:00 (CET)
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Active since 2014, PHUSE’s Data Transparency Working Group has provided subject matter expertise for the review of draft deliverables and guidance documents from regulatory bodies (such as the EMA and Health Canada), as well as other industry organisations (such as TransCelerate) and academia. Since their inception in 2020, the free-to-attend Data Transparency Events have gone from strength to strength. These virtual events have created an unrestricted space where questions can be asked and challenges addressed. Individuals passionate about the area can come together to share vital knowledge, develop new ideas and spark innovation through presentations, panel discussions and Q&A sessions alongside experts in the data-sharing field.
The PHUSE Data Transparency Autumn Event will take place from 4–6 February 2025. Data Transparency Events offer you the chance to gain knowledge and experience from a wide data transparency community, allowing you to come together with experts from a variety of companies and backgrounds. During this virtual event, presentations will be delivered across the three days in bitesize chunks from 15:00-17:30 (GMT). There will also be a panel discussion and Q&A session focused on the day's themes.
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We're thrilled to announce a new Working Group event coming your way in 2025 – the PHUSE Real World Data Spring Event!
Taking place 9–10 April, this innovative event will bring the PHUSE Community together to explore the transformative potential of real world data (RWD).
Call for speakers is open, so wherever you are in the world, you can join us virtually – and for free – to share insights, learn and collaborate with like-minded individuals. Find out more about the event here. Look out for registration opening on 17 February!
Here’s your chance to take the virtual stage and share your expertise! We’re looking for speakers to inspire, inform and lead the conversation on RWD. To guide your submission, here are some focus areas that will be central to the event:
- Data sources, lineage and provenance
- Vendor-sponsor governance and alignment
- Representing patient diversity
- Overcoming challenges with unstructured data
Do you have a unique perspective that doesn’t fit into these categories? We encourage you to think outside the box and propose ideas that reflect the cutting edge of RWD.
Submit your 150-word abstract by 22 January.
Status colour Blue title Submit Your Abstract
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We have just published the last Working Group Report for 2024! You can view the Quarter 4 summary here, including the project reports and some of the highlights across our Working Groups for this final quarter. Don’t forget to check out the Working Group Events Report too, which provides a breakdown of the data collected from the Working Group Events for 2024.
Well done to all our Working Groups who have had a fantastic 2024!
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The "Emerging Trends and Technology" (ET&T) Working Group is changing its name! This Working Group will now be called "Emerging Trends and Innovation" (ET&I). Hear from one of the Working Group's Leads, Mike Hamidi, on why they are making this change:
"The proposed name change from Emerging Trends & Technologies to Emerging Trends & Innovation reflects a broader focus beyond just technological enablement. This shift encompasses not only ongoing developments in areas such as AI/ML, digital health technologies, and healthcare standards but also aims to provide deeper insights into these and other evolving topics. We believe this adjustment will preserve the emphasis on emerging trends while enhancing our commitment to delivering valuable industry innovations."
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In its 14th year, the PHUSE/FDA Computational Science Symposium (CSS) will be expanding its global reach! In addition to Silver Spring, Maryland, we will be running a simultaneous event in Utrecht, the Netherlands!
Mark Your Calendars!
The US event will be in Silver Spring, Maryland 19-21 May, and the EU event will be in Utrecht, the Netherlands 20-21 May.
Please visit the PHUSE website for more information.
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The Analyses & Displays for Hepatotoxicity project within the the Safety Analytics Working Group has produced a white paper, ‘Recommendations for Analyses and Displays Associated with Hepatotoxicity with a Focus on Phase 2-4 Clinical Trials and Integrated Submission Documents Stage 1: Initial Assessment and Screening for Potential for Hepatotoxicity'. The purpose of this document is to provide recommendations for displaying, summarising, and analysing measures of hepatotoxicity in tables, figures, and listings (TFLs). The suggested TFLs will have implications for what and how liver-related data should be collected.
Please provide your comments by emailing workinggroups@phuse.global. Closing date for comments: 20 December
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The SEND Industry Feedback Survey project within the Nonclinical Topics Working Group have published their 2025 SEND survey! This is the 9th year the survey has taken place. The survey will be open from December to 15 January and the data collected will be anonymous. The results will be first available at the PHUSE Computational Science Symposium 2025 and will then be posted to the SEND Industry Feedback Survey site.
Status colour Blue title Submit Here
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The Submitting Real World Data project within the Real World Evidence Working Group, has published a Blog. This Blog post explores possible approaches for submission of real world data to regulatory bodies. Real world data, as defined by the Food and Drug Administration in their guidance, comprises data collected from non-interventional study designs. (1) From project group discussions, ‘claims’ and ‘electronic health records’ comprise most real world data used in providing clinical evidence. Fast Health Interoperability Resources, or FHIR, is a platform used by HL7 for health information exchange. (2) This platform is widely used to exchange healthcare information, as far as claims and EHR data is concerned. As a result, a significant portion of real world data used by the pharma/biotech industry follows HL7-FHIR.
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The SDTM ADaM Implementation FAQ project within the Optimizing the Use of Data Standards Working Group has published a new FAQ! They have answered a FAQ with SDTM/ADaM IG Nuances, which is about the Use of Different Versions of MedDRA SMQs and CMQs for a Single Study. You can view this question and see the FAQ's this project has answered so far via here.
Do you have a SDTM ADaM Implementation FAQ question? You can send your questions to the team by emailing workinggroups@phuse.global.
The Estimands in Safety Analytics, a new project within the Safety Analytics Working Group, is now calling for volunteers. The value of well-defined safety estimands is not generally appreciated and has not been communicated well to safety scientists.
- Safety analyses are often ambiguous and, in some cases, carry the risk of being misinterpreted.
- A better understanding and consequently a more consistent application of a robust estimand framework in the analysis of safety data would eliminate this ambiguity and drive clarity on both the specific question an analysis addresses, as well as the answer to that question.
If you would like to volunteer or learn more about this project, email workinggroups@phuse.global. Closing date: 14 February.
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Data Transparency Autumn Event 2025 – Call for Speakers is Open! | ||
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Call for Speakers is officially open for the Data Transparency Autumn Event 2025! 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.
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Real World Evidence Webinar Series | ||
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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)
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Community Forum: AI and SaMD in Healthcare: From Compliance to Patient-Centred Innovation | ||
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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)
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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)
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The Implementation of Estimands (ICH E9 (R1)) using Data Standards project within the Optimizing the Use of Data Standards Working Group, have published a new White Paper and Example Document! This white paper provides recommendations and examples to illustrate the implementation of the estimands framework introduced by the ICH E9(R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials [ICH E9(R1), 2019] using data standards (as part of the clinical data flow). |
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The Dataset-JSON as Alternative Transport Format for Regulatory Submissions project within the Optimizing the Use of Data Standards Working Group, has published its Business Case. Dataset-JSON is a widely accepted data format that will streamline data exchange between organisations. Moving to Dataset-JSON will prepare us for the current digital world and enable companies to more accurately, completely and efficiently represent their clinical research data, to better serve their patients/customers. Dataset-JSON supports current data standards but also allows for standards to evolve and removes current limitations imposed by xpt format. |
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If you would like to get involved in a PHUSE Working Group Project, please explore the projects via the |
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Volunteer Board and contact the PHUSE Office |
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on workinggroups@phuse.global to express your interest. |
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Group Report: Includes project updates, recent and upcoming deliverables and future plans for each Working Group. | |
Monthly Mailings: The monthly newsletter. Here you will find a full update from each month of the year, easily accessible and divided into key areas of PHUSE. | |
PHUSE Blogs: Fancy a quick read? A blog is a perfect way to catch up on all things Working Groups. Get the lowdown on the latest events from across the globe and stay updated on industry topics brought to you by industry professionals. |
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Initiate and lead a new project under the PHUSE Working Groups umbrella. The new project must address problems of significant relevance to computational science related to drug, biological and device development and must meet all of the guidelines for projects within the collaboration, including the following mandatory requirements:
New projects can be submitted anytime during the year, click here to submit. |