The sun is shining, the flowers are budding, and the Working Groups have sprung into spring! We’re implementing some changes to the Working Groups in 2020. Projects will become more defined and the PHUSE CSS scope will be expanded to support Education and Technical activities. We’ll also receive support from senior leaders from pharma, CRO, technical and FDA stakeholders. With this, we’ll see new and exciting projects and deliverables come through the pipeline. For now, let’s take a look at what the projects have achieved so far this year.

Announcements:

Since the cancellation of the US Connect 2020 Orlando event, the PHUSE Board and the PHUSE Office have worked tirelessly to examine and evaluate all possible options to determine the best way forward.

In collaboration with the FDA, we will be postponing the CSS from June until 21st–23rd September. It is our current working assumption that travel restrictions will be lifted by the second half of the year, and the event will be able to go ahead at this later date. More information will be shared through the Working Groups and directly with the sponsors.

Data Transparency:

The Data Transparency team conducted a review of the Modernization of ClinicalTrials.gov via the National Library of Medicine. The documented review can be found on the PHUSE website.

Nonclinical Topics:

The Modeling Endpoints project achieved a white paper through extensive collaboration with the CDISC workstream evolving the Immunogenicity Specimens Domain. By working together with the CDISC team and ADA experts from nonclinical laboratories, the project developed an interim recommendation for modeling nonclinical ADA data in SEND 3.0 and 3.1 that supports e-data submission of this important data in anticipation and consideration of forthcoming CDISC SEND team efforts.

Across industry, there are a wide variety of quality control (QC) checks for SEND datasets. This is currently done without commonly acknowledged scope and intensity, based on companies’ own approaches. The SEND Dataset QC Best Practices project recently conducted a survey to explore the variety of QC procedures and tools among different stakeholders. The team are looking to develop recommendations for stakeholders for efficient and effective SEND QC practices.

Standard Analyses & Code Sharing:

The Standard Analyses & Code Sharing Working Group has a new project in collaboration with the American Statistical Association (ASA). The R-shiny Interactive Forest Plot project looks to develop an R-shiny application(s) to enable the generation of identified plots for direct inclusion in submission packages for regulatory agencies. The initial scope is to develop tools to generate forest plots for inclusion in submissions to the FDA.

Emerging Trends & Technologies:

The Data Visualisation project conducted an updated analysis of the current tools used to review subject level data, including the use of interactive visualisations for generating patient profiles and patient narratives. This paper focusses on three types of subject level data representations: Tabular Patient Profiles, Graphical Patient Profiles and Patient Narratives.

The Study Data Validation and Submission Conformance project is replacing the Going Translational with Linked Data project, extending existing work in both clinical and preclinical areas by further developing the data models and instance data conversion. The project will include modeling FDA Technical Rejection Criteria to facilitate submission of data to the FDA. The project will provide a gateway for Knowledge Graph technology to support the FDA’s Technology and Modernisation Action Plan, as well as produce a proof of concept which will demonstrate the creation of highly compliant, high-quality preclinical and clinical study data for submissions, using a much more automated process than is currently available. Do you have experience with Ontology Development and Linked Data? Interested in joining? Contact workinggroups@phuse.eu to find out how.

Optimizing the Use of Data Standards:

The Data Reviewer's Guide in XML project has released v2.0 of the Prototype Package. This prototype release looks to provide a more stable version for industry early adoption and testing for a future final publication that encompasses all DRGs.

The Best Practices for Data Collection Instructions project has conducted a survey on Adverse Events & Treatment Emergent, covering both data collection and analysis of adverse events. The results are in and the team are currently putting together a white paper documenting challenges and gaps with the CDASH CRF completion, as well as recommendations for a future version of CDASH.

What a busy few months! We have many things to look forward to this year, such as collaborations with ASA, BioCelerate and the FDA, not forgetting the Computational Science Symposium in September. Although we may face some uncertainty in these unprecedented times due to the outbreak of COVID-19, we’d like to thank you for your support over the last few weeks.

PHUSE is built upon connecting and sharing. This is something we will continue to do; although that may not be face to face, we look forward to seeing you all when the world is back to normal. Stay safe and continue to connect, share and advance.