Content Comparison

The PHUSE Estimands for RWD/RWE project team, within the Real World Evidence Working Group, sponsors a webinar series – At the Intersection of Estimands and Target Trial Emulation (TTE) for RWE. The purpose of this webinar series is to cross-pollinate perspectives on the topic of frameworks, such as estimands and target trial emulation (TTE), as it relates to using RWD that results in RWE. The order of the webinars takes attendees on a journey from orientation to potential solutions. As you’ll see, the dates of the published papers range from 2021 to 2024, so the information is very recent, and likely not widely known or understood. The project team is also planning to publish its own white paper, and this webinar series will discuss ideas that will help drive the content.

This webinar will be taking place virtually on 22 April at 10:00–11:00 (EDT) / 15:00–16:00 (BST) / 16:00–17:00 (CEST)

The End to End RBQM Education project, within the Risk Based Quality Management Working Group are hosting their second webinar on the topic of Revolutionising Participant Safety Monitoring with Advanced Solutions!

This session will delve into Adverse Event (AE) monitoring and participant safety with a panel of industry experts who will be sharing their perspectives on the changing landscape of safety monitoring.

This webinar will be taking place virtually on 23 April at 10:00–11:00 (EDT) / 15:00–16:00 (BST) / 16:00–17:00 (CEST)

In its 14th year, the /wiki/spaces/WEL/pages/38993921 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.

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.

The Working Groups quarterly report is now live! These reports highlight the fantastic progress across our projects, featuring key achievements and the exciting work still underway. Explore the complete reports to see how each project is driving innovation and impact.

Revisit the events from this quarter and explore what’s coming up next.

The SDTM ADaM Implementation FAQ project within the Optimizing the Use of Data StandardsWorking Group is pleased to announce that all published FAQ’s are now available on the PHUSE Deliverable website. Click here to view the categories of answered FAQs. 

Real World Evidence

We are pleased to announce that Elena Valkanova will be taking over as the new Real World Evidence Working Group Lead.

Elena began her career in clinical research more than 20 years ago, specialising in statistical programming and data analysis across diverse therapeutic areas. Throughout her career, she has contributed to the design, validation and reporting of regulatory deliverables, ensuring accuracy, reproducibility and compliance with global standards such as CDISC. Her early academic research into algorithmic game theory focused on developing randomised and strategy-improvement algorithms for simple stochastic games – a rare class of combinatorial problems that lie in both NP and co-NP and have no known polynomial-time solutions – providing a strong foundation in probabilistic modelling, computational complexity, and optimisation.

Elena now leads collaborative initiatives to develop practical frameworks and implementation guidance for privacy-preserving methods, including differential privacy, federated learning and synthetic data generation. Her leadership centres on building cross-disciplinary resources that integrate statistical programming, machine learning (ML), artificial intelligence (AI) and large language models (LLMs) to enable compliant, scalable and scientifically robust use of real-world data (RWD).

The QC Workflow Optimisation project within the Emerging Trends & Innovation Working Group have published a survey exploring the current state of Quality Control across the industry.

This survey aims to capture an industry-wide view of current Quality Control practices, how they may need to evolve, and the potential role of emerging technologies such as AI. Your input will help shape future PHUSE discussions, highlight areas for improvement or standardisation, and guide next steps for the QC Workflow Optimisation project.

The survey will remain open from 3 December – 13 January. All responses will be collected anonymously.

The Cloud Adoption in the Life Sciences Industry project within the Emerging Trends & Innovation Working Group, has published a new White Paper, Cloud Services: A Framework for Adoption in the Regulated Life Sciences Industry.

This White Paper provides guidance for adopting cloud services in regulated life sciences. It explains how GxP requirements apply to cloud providers and offers a clear framework for planning and executing exit strategies to ensure data integrity, business continuity and compliance.

The Participant Data Return team has published a blog on their recent survey findings. Participant data return is the act of granting clinical trial participants access to the data that was collected about them during a clinical trial. In current clinical trial practice, we take measurements, conduct tests, and perform procedures. The results are reviewed by the investigator and stored by the sponsor, but participants rarely see their own results.

In spring 2025, PHUSE established the Participant Data Return project, with the aim of highlighting current industry practice and identifying gaps and challenges in implementation to speed industry uptake. As a first action, in summer 2025, we assessed the current state of data return throughout the industry by surveying PHUSE member companies. PHUSE received a total of 14 responses representing 14 companies worldwide.

The published blog summarises findings and next steps for this project team. If you are interested in getting involved please email workinggroups@phuse.global.

The /wiki/spaces/WEL/pages/220168194 team has completed their system review and documented their feedback.

The International Standard Randomised Controlled Trial Number registry (ISRCTN) recently released information for public review and comment regarding the new registry they are working on, and the results system prototype was open for review until the end of September. 

The PHUSE project team assembled to review the prototype was a diverse group of experts from the PHUSE Data Transparency Working Group. For the purposes of providing feedback, fields were tested but no results submitted. The summary of comments, included in the ISRCTN results system prototype feedback collated from our review at PHUSE can be found in this document.

The /wiki/spaces/WEL/pages/220168194 within the Data Transparency Working Group has published a new response to a question regarding MHRA Clinical Trials Regulation Guidance.

On 28 April 2025, the UK Parliament and Northern Ireland Assembly approved the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025 (available via this link). These new regulations introduce changes to the Medicines for Human Use (Clinical Trials) Regulations 2004, which currently govern the regulation of clinical trials of investigational medicinal products (CTIMPs) in the UK.

New guidance has been published by the UK’s Health Research Authority (HRA) to accompany the Medicines and Healthcare products Regulatory Agency (MHRA)’s updated clinical trials regulations, which come into force on 28 April 2026. The guidance explains important points on what will change in terms of processes, legal requirements, and expectations for anyone involved in setting up or delivering clinical trials. External stakeholders who wished to share feedback on the newly published guidance were asked to provide their submissions to the HRA by 10 September 2025.

View the team's response to the updated guidance here and the associated MHRA algorithm document here.

Community Forum: The Causal Roadmap,Targeted Learning and TMLE: What Is That All About?

29 January 2026 at 10:00-11:00 (EST) / 15:00-16:00 (GMT) / 16:00-17:00 (CET)

Information and Registration

The Nonclinical Topics Working Group is excited to announce a multi-day virtual event coming 10–12 February 2026.

The Nonclinical Advance Event will explore key themes including New Approach Methodologies For Nonclinical Safety Assessment, Nonclinical Predictive Modelling And Machine Learning & PHUSE Project Contributions And Developments

Full agenda and registration details coming soon!

We’re pleased to announce that the Real World Evidence Working Group will be holding its second multi-day virtual event from 30 September–1 October 2026.

 Stay tuned for more details and registration information coming soon!

The Value of Plain Language Summaries of Trial Results (PLSs) project team within the Data Transparency Working Group, is calling for volunteers!

This project has been initiated because whilst the industry has put significant efforts into developing and disseminating plain language summaries of trial results (PLSs) to promote transparency and engagement, there remains a gap in quantitative evidence of how these documents are used and what value they bring to the trial participants and the general public. Gathering feedback from trial participants – the primary audience for PLSs – and other readers will provide meaningful insights into their value and inform improvements.

The team will be creating a survey to evaluate the value of PLSs and the survey feedback will be shared as a white paper authored by the group.

If you’d like to become a valued project member, please get in touch with workinggroups@phuse.global by 19 January 2026 ahead of the kick off meeting scheduled for Tuesday 20 January 08:00 EST / 13:00 GMT / 14:00 CET.

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. 

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. 

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: 

• The projects must address significant research issues relevant to Computational Science

• The project must not attempt to address FDA policy issues

• There must be at least one Project Lead personally involved in planning and carrying out the project

New projects can be submitted anytime during the year, click here to submit.