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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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Data Transparency Autumn Event |
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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 17–19 September 2024. 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.
The full event agenda can be found here.
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- What is the true cost of adopting open-source tooling for clinical trial data analysis?
- Can the industry fund open-source?
This Forum will be taking place over Zoom on 13 September at 15:00-16:00 (BST) / 10:00-11:00 (EDT).
Status colour Blue title Register Now
| Community Forum: Regulatory Landscape of AI/ML in DHTs (Current Landscape, Knowledge Gaps, Best Practices for Regulatory Submissions, Challenges of Regulating AI) |
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The Emerging Trends & Technologies Working Group is hosting its second Community Forum of the year, Regulatory Landscape of AI/ML in DHTs (Current Landscape, Knowledge Gaps, Best Practices for Regulatory Submissions, Challenges of Regulating AI). We will be hearing from both FDA and Pfizer speakers, who will be looking at how Digital Healthcare Technologies (DHTs) show significant potential for enhancing data acquisition in clinical trials by enabling objective and accurate data collection. Examples of DHTs include wearable devices – sensors, smartwatches, fitness trackers – that continuously monitor vital signs, physical activity, sleep patterns, and medication adherence. However, the emergence of DHTs also raises regulatory-related questions. In this PHUSE Community Forum, we will discuss the development of digitally derived measures (clinical outcome assessments and biomarkers) and germane regulatory policies from the perspectives of government and industry experts. This Community Forum will be taking place over Zoom on 25 September at 13:30-14:45 (BST) / 08:30-09:45 (EDT). |
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The Quality and Reusability of Real World Data project within the Real World Evidence Working Group has published its third Blog Post, RWD: The FDA’s Guidance on Assessing Registry Data to Support Regulatory Decision-Making. This Blog will explore the main messages in the regulatory guidance, what challenges we foresee in applying it, and how registry data can be used in regulatory applications.
You can learn more about this project and read their previous blogs here.
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The CDISC Standard for Exchange of Nonclinical Data (SEND) data standard has created new opportunities for collaborative development of open-source software solutions to facilitate cross-study analyses of toxicology study data.
A sub team of the BioCelerate SEND Implementation for Cross Study Analysis Initiative was formed and through collaborative efforts via a public–private partnership between BioCelerate and the FDA/Center for Drug Evaluation and Research (CDER), they worked to develop and publicise novel methods to facilitate cross-study analysis of SEND datasets.
As part of this work in collaboration with the Pharmaceutical Users Software Exchange (PHUSE), an R package called sendigR has been developed to enable users to construct a relational database from a collection of SEND datasets and then query that database to perform cross-study analyses. The sendigR package also includes an integrated Python package, xptcleaner, which can be used to harmonise the terminology used in SEND datasets by mapping to CDISC controlled terminologies. This package, sendigR, will provide data scientists and toxicologists with a free, open-source tool that can be utilised to query large repositories of electronic standardised toxicology study data. sendigR was published to the Comprehensive R Archive Network (CRAN) and GitHub - phuse-org/sendigR: Enable Cross-Study Analysis of 'CDISC' 'SEND' Datasets. An R Shiny web application was included in the R package to enable toxicologists with no coding experience to perform historical control analyses. Experienced R programmers will be able to integrate the package functions into their own custom scripts/packages and potentially contribute improvements to the functionality of sendigR.
To learn more, read the sendigR accompanying manuscript here: Frontiers | sendigR: an R package to leverage the value of CDSIC SEND datasets for cross-study analysis (frontiersin.org)
sendigR reference manual: Enable Cross-Study Analysis of CDISC SEND Datasets • sendigR (phuse-org.github.io)
sendigR R Shiny demo app: phuse-org.shinyapps.io/sendigR/
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The AE Groupings in Safety (AEGiS) project within the Safety Analytics Working Group has produced a White Paper, 'Utilization of Adverse Event Groupings in Clinical Trial Safety Assessment' This document outlines why using standard AE groupings for queries is key to understanding a drug's safety profile, discusses some assumptions underlying use of standard queries for estimation and outlines the benefits of standard queries when available. When standard queries are not available but detailed analysis of a specific medical condition is needed, this document recommends a process to develop and maintain a custom query that can be replicated by regulatory authorities.
Please provide your comments by emailing workinggroups@phuse.global. Closing date for comments: 6 September.
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2025 – Call for Speakers is Open! | ||
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Call for Speakers is officially open for the Data Transparency Autumn Event 2025! 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 15:00 to 17:30 (BST). 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 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 take place virtually on 16 June at 11:00 (EDT) / 16:00 (BST) / 17:00 (CEST)
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Data Transparency Autumn Event 2025 |
Active since 2014, 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 Data Transparency Autumn Event 2025 will take place on 16-18 September. 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. Registration for this event will open in July. |
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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. |
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Working 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. |