In today's rapidly evolving world of pharmaceuticals and healthcare, data-driven decision-making has become paramount. Regulatory agencies, such as the FDA, are increasingly relying on computational science and data analytics to expedite drug development, enhance safety assessments and ensure the efficacy of new treatments.
Year after year, the PHUSE/FDA Computational Science Symposium (CSS) continues to be a driving force behind shaping the future of drug development and regulatory science. The event stands as a testament to the pharmaceutical industry’s commitment to innovation and collaboration.
We are pleased to have welcomed 210 attendees to Silver Spring! 31 poster presentations, 30+ Working Group Breakout Sessions, three Hands-on Workshops and two Panel Discussions and filled the three event days with a wealth of inspiration.
Monday – Day 1
The event started with a bang with a Hands-on Workshop! Participants received valuable training on Dataset-JSON standardisation, an introduction to the CDISC Dataset-JSON standard, and got stuck in with practical demonstrations of working with Dataset-JSON. The workshop concluded with guidance on how to access useful Dataset-JSON resources.
Next was an FDA-led Plenary Session on ‘Collaboration Driving Technology Adoption’. This session, chaired by Lilliam Rosario (FDA), featured speakers from across the FDA, industry and CDISC addressing both the Dataset-JSON pilot and the growth of open source technologies.
This session concluded with the Plane Builder awards, which recognise all those who have made a significant contribution to the Working Groups over the previous 12 months – keep reading to view the winners!
Day 1 concluded with the Welcome Event supported by IBM, where attendees enjoyed drinks, canapés and refreshments – and plenty of networking!
Tuesday – Day 2
Tuesday was a busy day – 23 Breakout Sessions took place across seven of the Working Groups in order to drive existing initiatives forward and explore new ones.
Privacy Analytics delivered an excellent Hands-on Workshop on anonymised data, which covered generating synthetic health data, the privacy benefits and risks of synthetic data, and evaluating the utility of synthetic data. This was achieved through a mix of lectures and hands-on exercises using an online SDG tool.
Brought to you by the Nonclinical Topics Working Group, a mix of regulatory and industry individuals held an informative Panel Discussion on the role of SEND in the implementation of virtual controls. Attendees also enjoyed a joint session with the Safety Analytics and Data Visualisation & Open Source Technology Working Groups, which looked into CVARS.
It was then time for the ever-popular Poster Session, supported by Entimo, which proved a great success, as it allowed for Working Group and computational science topics to be addressed, and facilitated Q&A discussion between poster presenters and attendees.
Wednesday – Day 3
The final day opened with a Regulatory Plenary Session. Chaired by Jean-Marc Ferran (Qualiance), we heard from Marie Bradley (FDA) on the latest regarding real world evidence, from Anne-Sophie Henry-Eude (EMA) on the transparency aspects of the raw data pilot and the CTIS Guidance, and from Cheryl Grandinetti (FDA) on risk-based monitoring. The session finished with announcing the CSS Poster award winners.
15 Breakout Sessions took place on day 3. A highlight was the continuation from day 1, with two joint Breakout Sessions on the work of the new Dataset-JSON project. The first session was a collaboration between the Data Visualisation & Open Source Technology, Emerging Trends & Technologies, Nonclinical Topics and Optimizing the Use of Data Standards Working Groups, where they addressed the business requirements for Dataset-JSON. The second joint Breakout Session between the Data Visualisation & Open Source Technology and the Emerging Trends & Technologies Working Groups built on the content from the first session to consider how the business requirements could be implemented in technology.
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 within 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 Chairs for their contributions in developing content for the event, as well as 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 throughout the year. 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 contributing to this event. We are already busy planning for the CSS 2024, which will take place 3–5 June, with Call for Posters opening from 29 January to 1 March. We look forward to continuing the collaboration!
A full summary of the CSS will be presented in October’s Webinar Wednesday – click the link to register your place. Plenary Session slides and the posters are also now available to access via the PHUSE Archive.
CSS 2023 Plane Builder Award Winners
Every year at the CSS, PHUSE recognises individuals from each Working Group who have been a key contributor over the previous 12 months. PHUSE would like to acknowledge this year’s CSS 2023 Plane Builder award winners and thank them for their continued contribution.
Data Transparency – Abby McDonell, Privacy Analytics and Lauren Hepburn, ICON
We have selected Abby McDonell and Lauren Hepburn for their leadership of the Good Transparency Practices project. This project is filling a gap in the community by holistically addressing data sharing from a governance standpoint across all stakeholders involved. This project is also the first project where we had both the EMA and Health Canada involved as observers, and it was a successful experience. The project is now in its last round of reviews and will be a significant addition to the PHUSE Data Transparency eco-system of deliverables.
Data Visualisation & Open Source Technology – Lyn Taylor, PAREXEL International
Lyn Taylor has taken on leadership of the CAMIS project and has established new momentum. Their recent white paper was an introduction to the project, titled ‘Key Considerations When Understanding Differences in Statistical Methodology Implementations Across Programming Languages’, which has now been finalised and published. There has been an expansion of collaboration throughout, and the group is presenting on the CAMIS framework at the PHUSE/FDA quarterly meeting.
Emerging Trends & Technologies – Anders Vidstrup, NNIT
Anders Vidstrup has been awarded this year’s Plane Builder award in recognition of his exceptional contribution to and leadership as a key member of the PHUSE Emerging Trends & Technologies Working Group Cloud Adoption project. His dedication, expertise, and innovative approach have made a profound impact on the success of this critical initiative.
Throughout this project, Anders has demonstrated exemplary skills and a deep understanding of cloud technologies, and consistently driven the team forward with his valuable insights and ideas. His ability to navigate complex challenges and find effective solutions has been instrumental in advancing the group's objectives. His commitment to excellence and collaboration has been evident at every stage of the project, from the initial planning and strategy development to the implementation and evaluation phases. He has a fantastic ability to foster a positive and inclusive team environment and has greatly contributed to the overall success of this endeavour.
Anders’ work on the Cloud Adoption project not only showcases his technical prowess, but also highlights his exceptional leadership qualities. By inspiring and motivating other, he has maximised the team's potential and achieved outstanding results.
On behalf of PHUSE and the Emerging Trends & Technologies Working Group, we express our deepest appreciation for Anders’ remarkable contributions, dedication, and exceptional work. Please accept this award as a testament to the significant impact you have made. We extend our warmest congratulations on this well-deserved recognition.
Nonclinical Topics – Gitte Frausing, Data Standards Decisions
The Nonclinical Topics Working Group is very pleased to present this year’s Nonclinical Topics Plane Builder award to Gitte Frausing. Since the outset, Gitte Frausing has been a valued collaborator, motivated team member and enthusiastic project leader. Her deep knowledge has been evident throughout her project participation: from nonclinical protocol design, data collection and data standardisation, all the way through to electronic submission. She continues to provide strong experience across the wide subject range of our projects for nonclinical data challenges.
In 2012, Gitte co-led the Roadmap for Nonclinical Study Data Standardization and Elements to Improve Data Access project, which set the early stage in identifying priorities of the toxicology community for data standardisation. Since then, she has contributed again and again to projects such as how to make a custom SEND domain and development of the nSDRG. After last year’s CSS, Gitte partnered with Hepei Chen to develop a data challenge concept, then kicked off a new project addressing an unmet need. The Conformance with the tumor.xpt Specification project drew significant volunteers and enthusiasm to work towards future excellence of tumour data submissions. Complete mission alignment of our Working Group! Gitte brings tremendous positive energy to the nonclinical data standards development and implementation community, not only through her work with PHUSE but also with CDISC and her active consulting career.
Optimizing the Use of Data Standards – Lori VanMeter, Johnson & Johnson
Lori VanMeter joined the Working Group in 2021 as a Project Co-Lead for Implementation of Estimands (ICH E9 (R1)) using Data Standards along with Chris Price. She has leveraged her statistical background and understanding of the ICH E9 amendments to co-facilitate the project activities and organise the plans with her fellow PHUSE members and the FDA. Since joining, we have witnessed Lori raise awareness of the project, share knowledge at the CSS 2022 and facilitate the collection of best practice and considerations of estimands.
Lori is a collaborator and an appreciated member in our community. Congratulations to Lori for her contribution and dedication to co-leading this project.
Real World Evidence – Mary Anne Rutkowski, Merck & Co
Mary Anne Rutkowski has always been a very active project member within Real World Evidence, from when it was a project within Emerging Trends & Technologies to now being its own Working Group. She co-leads the Real World Evidence Community Forums, which have been a huge success. Mary ensures that these are planned quarterly and are well organised.
Risk Based Monitoring – Nathalie van Borrendam, Johnson & Johnson and Annett Keller, *Boehringer Ingelheim *
Nathalie van Borrendam and Annett Keller are leading the Quality Tolerance Limits project, which focuses on methodologies for threshold setting. Conceptually, the industry is starting to get aligned on QTLs but there is still much to learn about how to select the best parameters and get towards a threshold that has been set with a solid methodology and which truly adds value. The Working Group has written an excellent paper on this topic (which has not been done before) and the paper has been accepted by TIRS – Quality Tolerance Limits: A General Guidance for Parameter Selection and Threshold Setting.
Safety Analytics – Mac (Robert) Gordon, Johnson & Johnson
The Safety Analytics Working Group chose to award the Plan Builder award to Mac Gordon, for his leadership in consolidating comments from the Working Group on the FDA’s Standard Safety Tables and Figures document and FDA Medical Queries. Gathering comments was a lot of work within a short time period. Mac also co-leads the AE Groupings in Safety (AEGiS) project team, which is a very large and active group.
Poster Winners
Industry – Swathi Kotla and Madhusudhan Papasani, Merck
PP24 – Data Handling for Subjects Participating in Multiple Cohorts in Oncology Platform Trials: Considerations for Data Collection, SDTM, and ADaM Mappings
Platform trials have emerged as a transformative approach in oncology research, facilitating the investigation of multiple targeted therapies across diverse cohorts based on molecular subtypes. These trials offer the unique advantage of flexibility, allowing for the exclusion of ineffective interventions and the seamless addition of new treatments. Notably, platform trials may encounter scenarios where subjects exhibit multiple molecular subtypes, initially enrolling in one cohort, but the cohort being discontinued at an interim analysis due to lack of efficacy. The eligible subjects from the discontinued cohort are re-screened and re-enrolled in a newly added cohort. This poster will present practical examples of data collection methodologies for re-enrollers, emphasising the SDTM data mapping to essential domains such as DM (Demographics) and DC (Demographics as Collected). Additionally, it will highlight the handling of data for analysis purposes, presenting subject-level data in ADSL, ADTP (Trial Participation Analysis Dataset) and laboratory data in ADLB.
Collaboration – Abby McDonell, Privacy Analytics and Lauren Hepburn, ICON
PP12 – Good Transparency Practice: A Working Group Update
This poster will provide a summary on the Good Transparency Practices (GTP) Working Group deliverable. The group created a GTP guidance document that defines a set of best practices for data transparency. The GTP guidance aims to ensure anonymized data sufficiently protects patient privacy while retaining as much data utility as possible. Representatives from the European Medicines Agency (EMA) and Health Canada joined the group in an advisory capacity to ensure the GTP is compliant with regulatory policies. The group has published the GTP guidance on the PHUSE deliverables webpage.
FDA – Susan Butler and Kevin Snyder, FDA
PP17– SEND Sanitizer: Generation of Synthetic SEND Data from Existing Studies
SEND datasets are expensive and valuable outputs used to characterises toxicity profiles of new drug candidates. However, due to their proprietary nature, the availability of SEND data for toxicity modelling and large-scale cross-study analyses is limited. A possible solution to this problem is generating anonymized synthetic datasets based on the trends of real data using the application of Markov Chain Monte Carlo (MCMC) simulations. SEND Sanitizer is an R Shiny-based application designed for this purpose. A user can generate synthetic SEND format data with submitted trends such as example studies. If the example studies are of the same species and dose arm regiments, the application can generate a user-specified amount of .xpt files for the BW, DM, DS, EX, LB, MI, TA, TS and TX domains. This will allow for future modelling capabilities of SEND toxicological datasets to improve the performance of nonclinical toxicity studies.