Held on 3–5 June in Silver Spring, Maryland, the PHUSE/FDA Computational Science Symposium (CSS) 2024 brought together a community of 212 passionate individuals.

The event showcased a dynamic agenda packed with 30+ Working Group Breakout Sessions, two Plenary Sessions, two Hands-on Workshops and a Poster Session. Attendees gained access to direct insights into regulatory topics from health authorities and explored a range of ideas crucial to the future of computational science.

The CSS stands as a testament to the industry’s commitment to advancing drug development and regulatory science through both collective effort and data-driven decision-making.

Watch the event vlog here.

Monday – Day 1

Day 1 kicked off with two interactive Hands-on Workshops. During ‘Dataset-JSON for Regulatory Submissions’, participants gained insights into leveraging this new CDISC data exchange standard to streamline regulatory processes and enhance data exchange capabilities. And in ‘ISO/IEC 27559:2022 – An International Framework for Standard Anonymisation’, participants were equipped with the knowledge and tools for ensuring data privacy and compliance within their organisations.

The workshops were followed by an FDA-led Plenary Session, ‘The Future of Enabling Health Authority Access to Information’. Highlights included a celebration of 10 years of the Data Transparency Working Group, the presentation of the finding from the Data-JSON project, and a chance to explore innovative FDA projects precisionFDA and PRISM.

Before the session closed, we took a moment to celebrate some members of our community through our Plane Builder awards (find details of the Awards Winners below). Special recognition went to Jane Owens, Mary Nilsson and Stephen Wilson, who are retiring. The PHUSE Board took this opportunity to award both Mary and Stephen (Jane has previously received this recognition) the John Hirst Award for Lifetime Contribution for their significant contribution to PHUSE.

Attendees concluded the day with the Welcome Event, supported by Regeneron. Light refreshments and informal networking created the perfect atmosphere for forging new connections and set the tone for a collaborative event ahead.

Tuesday – Day 2

Day 2 was a full day of productive Breakout Sessions spanning seven of the PHUSE Working Groups and featuring three collaborative sessions. Participants exchanged ideas and worked together to drive exciting projects forward. A particular highlight was the sessions led by the EMA and Health Canada in the Data Transparency Working Group discussing the topics of EMA Policy 0070, Health Canada PRCI and the Anonymisation Report Template.

Throughout the regular breaks, attendees continued to make new connections and meet the contributors at their exhibitor booths.

We concluded on a high note with the Poster Session, supported by Entimo. The posters were a source of inspiration and sparked discussion on important industry topics. Attendees were impressed by the breadth and depth of the research presented and the collective dedication of the PHUSE Community. Details of the Poster Awards Winners can be found below.

Wednesday – Day 3

The final day of the event commenced with a Plenary Session, where we heard from the EMA on data submission in Europe and from the FDA on AI use in drug development. The session concluded with the Poster Awards, where we recognised our best FDA, Industry and Collaboration posters.

Thirteen Breakout Sessions filled the rest of the day’s agenda. Particular highlights were follow-up discussions in the Nonclinical Working Group on EMA submission of nonclinical data in SEND format, proof of concept study, and the continued discussions in the Safety Analytics Working Group on the topic of education.

Thank You

We would like to thank all those who attended the CSS for their enthusiasm, open-mindedness and spirit. In-person collaboration is a vital component in the success of the Working Groups – the number of constructive conversations taking place during both the Breakout Sessions and the coffee and lunch breaks is a testament to the value of the CSS. Particular thanks go to Jean-Marc Ferran and the Poster Judges for their contributions in encouraging and supporting the development of content for the event, and to the Working Group Leads and Project Leads, who drove the conversations in the Breakout Sessions.

While the CSS takes place just once a year, we would like to recognise the many knowledgeable and enthusiastic volunteers who make significant contributions in the lead-up to the event. We are very grateful for the efforts of all those involved in advancing the PHUSE Working Groups’ mission to address 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.

We would also like to thank our contributors for their time and dedication in making this event such a success.

A full summary of the CSS will be presented in July’s Webinar Wednesday. The Plenary Session slides and posters are available to access via the PHUSE Archive.

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Plane Builder Award Winners

Every year at the CSS, PHUSE recognise individuals from each Working Group who have been a key contributor over the previous 12 months. PHUSE would like to acknowledge this year’s Plane Builder award winners and thank them for their continued contribution.

Data Transparency – Dan Bilodeau, ICON

Dan Bilodeau has shown great enthusiasm and dedication since joining PHUSE around two years ago. He presented at the Data Transparency Winter Event 2023 and is a member of the Educate the General Population on Data Privacy and Data Sharing project. He has abundant knowledge in his field of plain language writing and shares great ideas with the team. He has recently taken a brief hiatus due to juggling a very busy period in his day-to-day role and hopes to be back soon when things have quietened down. We look forward to seeing Dan when he returns!

Data Visualisation & Open Source Technology – Melvin Munsaka, AbbVie

Melvin Munsaka has been a key contributor to the Clinical Visual Analytics for Review and Submission (CVARS) project over the last few years. He has made great strides in collaborating with the agency and continuing to evolve the CVARS project to expand its scope and value. Year after year, CVARS has added new capabilities to their product, bringing updates to present at the PHUSE/FDA CSS and displaying the value PHUSE provides as a forum of collaboration between industry and the agency.

Emerging Trends & Technologies – Radha Railkar, Merck and Ying Su, Pfizer

Radha Railkar and Ying Su have been honoured with this year’s Plane Builder award in recognition of their exceptional contributions and leadership as key members of the Emerging Trends & Technologies Working Group’s AI/ML in Digital Health Technologies (DHTs) project. Their dedication and passion for leading an innovative initiative that encompasses six sub-projects is truly commendable.

While AI/ML is revolutionising the healthcare industry, particularly within digital health technologies, their project has made significant strides. The initiative successfully launched its first public forum titled ‘Innovations in Healthcare in the Age of Generative AI’ on 17 April 2024, marking a notable beginning. The project continues to foster industry engagement and knowledge transfer, motivating the community to participate actively.

On behalf of PHUSE and the Emerging Trends & Technologies Working Group, we express our deepest appreciation for Radha’s and Ying’s remarkable contributions, dedication and exceptional work. This award is testament to the significant impact they have made and continue to make. We extend our warmest congratulations on this well-deserved recognition.

Nonclinical Topics – SEND Survey Project Team

We want to recognise the year-on-year hard work of the SEND Survey Project Team: Janice Fiori, Lindsy Eickhoff, Vanessa Chavez and Tania Smith, plus alumni members Mike Harwood, George Kahlbaugh and Lou Ann Kramer.

This year will be the 8th annual survey! The survey results presentations are a foundational part of the Nonclinical Topics Working Group Breakout Sessions. The survey team works with PHUSE to publish, distribute and interpret customised surveys each year which focus on current hot topics and anticipate upcoming challenges with SEND implementation. Through their diligent and transparent summarisation of annual survey results, the team provide benchmark insights from industry and foster many new project ideas for the Working Group. It is no small task! This year – with shortened timelines since the CSS 2023 – the team has succeeded in executing a new survey for 2024, the results of which they will be presenting this week with their usual insightful interpretation. All surveys are published in the PHUSE Deliverables library and remain excellent references for describing the maturation in industry of CDISC standards implementation for nonclinical data.

Optimizing the Use of Data Standards – Christine Rossin, Pfizer

Christine Rossin is not only very active in the Optimizing the Use of Data Standards Working Group, but she also participates in projects within the Real World Evidence Working Group. Christine is involved in the Clinical Integrated Study Data & Analysis Reviewer’s Guide project and other projects, such as Bioresearch Monitoring (BIMO). The Reviewer’s Guide project entails creating multiple documents, including a template, completion guidelines, and example documents for both the Integrated Analysis Data Reviewer’s Guide (iADRG) and the Integrated Clinical Data Reviewer’s Guide (icSDRG).

Christine also participates at the US Connect and other PHUSE events. She led the CSS 2023 Breakout Session on the Integrated Clinical Study Data Reviewer’s Guide (icSDRG) and has done the same at the CSS 2024. Her leadership and involvement with PHUSE is greatly appreciated.

Real World Evidence – Dhruba Sikdar, Johnson & Johnson

Dhruba Sikdar has successfully led the RWD Guideline for Programming and Analysis Processes project.

In the last year, he has been able to get the project team working on a consistent paper which aligns the experiences and internal company guides to an industry-wide guideline that can be used to aid the effective usage of real-world data in clinical research. We are looking forward to the final guideline being published later this year and thank him for his efforts in making this happen.

Risk Based Quality Management – Chris Wells, Roche and Mireille Lovejoy, GE Healthcare

We thank Chris Wells and Mireille Lovejoy, the Quality Tolerance Limits (QTL) Implementation Project Co-leads, for their efforts in leading the team to develop and publish an important white paper that focuses on the state of the pharmaceutical industry’s use and application of QTLs, and their wider implication within a risk-based quality management (RBQM) framework. The results suggest that, while the industry is aligning on the use of QTLs, opportunities remain to enhance their use and how to incorporate them in the wider RBQM methodology. Chris and Mireille kept the project team focused on the delivery of this important contribution to the industry’s understanding of QTL implementation and are to be commended.

Safety Analytics – Aimee Basile, Otsuka Pharmaceutical

The Safety Analytics Working Group chose to award the Plane Builder award to Aimee Basile, for her leadership in the Adverse Event Collection Recommendations project and previous related projects. Aimee has been an active member of the PHUSE–FDA collaboration for many years. Most recently, she led the development of an adverse event collection white paper, which is about to be published in the PHUSE Deliverables library. The white paper involved multiple debated topics in which achieving alignment is challenging. She also presented a poster about this deliverable at this year’s PHUSE/FDA CSS. Additionally, Aimee conducted a workshop at the CSS 2023 showcasing methods for adverse event collection using three of the most used electronic data capture (EDC) systems (Veeva Vault, Medidata Rave, Oracle InForm). She demonstrated how each system can be configured to capture changes to adverse events without re-entering common data fields. This demonstration was key to the recommendations provided in the white paper.

Poster Winners

Industry – Bhavin Busa, Clymb Clinical

PP18: Bridging the Gap – Enhancing Analysis Results Generation with the CDISC ARS Model and the Community TFL Designer

The construction of TFL mock-up shells and analysis results metadata in our industry faces challenges stemming from variability, lack of standards, and manual processes. Survey results from 253 participants highlight the pressing need for standardised, machine-readable TFL display shells and analysis results metadata to bolster efficiency and collaboration. This research advocates for industry-wide adoption of such standards to streamline processes.

This poster will present survey insights alongside updates on CDISC’s Analysis Results Standard, showcasing its pivotal role in addressing these challenges. We will spotlight the TFL Designer Community solution, a practical tool for leveraging community TFL display templates and crafting study-specific shells. By enabling the prospective generation of ARS-compliant metadata, this platform facilitates automated TFL and analysis results generation. With access to a diverse library of TFL templates, including recommendations from regulatory agencies such as the FDA and the PMDA, and industry best practices from PHUSE Working Groups, the TFL Designer Community offers a transformative approach to TFL design and generation.

Collaboration– Vanessa Chavez, Labcorp & Lindsy Eickhoff, Inotiv

PP03: SEND (Standard for Exchange of Nonclinical Data) Industry Feedback Survey: 2024 Results

As the scope of SEND broadens, sponsors must continuously evolve to keep pace with updated regulatory specifications. The 2024 Industry Feedback Survey will provide a snapshot of the SEND industry, focusing on three main topics: manual edits pre- and post- tabulation with a focus on confidence level in making edits, harmonisation of industry-wide project initiatives, and implementation efforts for future SEND standards. The collected responses provide commentary on these topics and were analysed to report any result trends seen across the trade. PHUSE Working Groups will use the summarised results of this survey to examine possible future initiatives and discussions for effective SEND standard operations. 

FDA– Shraddha Thakkar, FDA

PP23: A Framework Enabling LLMS into Regulatory Environment for Transparency and trustworthiness and its Application to Drug Labelling Document

Regulatory agencies consistently deal with extensive document reviews, ranging from product submissions to both internal and external communications. Large Language Models (LLMs), such as ChatGPT, can be invaluable tools for these tasks, though they present several challenges, most importantly the potential use of proprietary and private information. This, combined with the need for customised functions for specific review needs and transparency and transparency of the model’s output, makes the need for a local and customised solution imperative.

To tackle these challenges, we formulated a framework named AskFDALabel on FDA drug labeling documents, which is a crucial resource in the FDA drug review process. AskFDALabel operates within a secure IT environment and comprises two key modules: a semantic search (Module S) and a Q&A/text-generation module (Module T). Module S is built on word embeddings to enable comprehensive semantic queries within labeling documents. Module T uses a tuned LLM to generate responses based on references from Module S. As a result, our framework enabled small LLMs to perform comparably to ChatGPT, leveraging a computationally inexpensive solution for a regulatory application.

To conclude, through AskFDALabel, we have showcased a pathway that harnesses LLMs to support agency operations within a secure environment, offering tailored functions for the needs of regulatory research.

John Hirst Award for Lifetime Contribution

We would also like to give a very special mention to Mary Nilsson, Eli Lilly, and Stephen Wilson, FDA, who have been awarded The John Hirst Award for Lifetime Contribution.

Mary has been the Lead of the Safety Analytics Working Group since its inception, having previously led the Safety Analytics and Code Sharing Working Group since the first CSS in 2012. She has been instrumental in developing white papers describing the analysis of safety data and in training materials shared through webinars and PHUSE Education. Mary’s passion for safety analytics has been the driving force behind the success of the PHUSE Working Group.

As one of its originators, Stephen’s involvement in the PHUSE/FDA CSS has demonstrated his dedication to driving meaningful collaboration and engagement across all stakeholders. His passion is evident through both his regular presence as a plenary presenter at the CSS and his invaluable contributions to multiple PHUSE Working Group projects. Stephen’s annual encouragement to attendees to embrace the concept of ‘building the plane while we are flying it’ inspired the naming of the PHUSE Plane Builder awards. His remarkable dedication and contributions have made him an integral part of the ongoing collaboration between PHUSE and the FDA.