An interactive R-Shiny app for quickly visualizing a tidy, long dataset with multiple dimensions with an application in clinical trial simulations for platform trials

Visualizing tidy, long data with multiple dimensions is a task often encountered when performing scientific computer simulations. In general, in such simulations the impact of many input variables on many outcome metrics is investigated. One area where such complex data structures containing the simulated data are encountered is clinical trial simulation. When visualizing the complex data, we are usually interested in the marginal effect of (one or several) input variables, which mostly renders existing visualization software useless or tedious to use. We have developed an R-Shiny app designed specifically for this task, which significantly speeds up the workflow. In this article, the app is described alongside two illustrative examples, one of which illustrates the use of the app for visualizing actual platform trial simulation data

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Summary: Platform trials bring the promise of making clinical research more efficient and more patient centric. While their use has become more widespread, including their prominent role during the COVID-19 pandemic response, broader adoption of platform trials has been limited by the lack of experience and tools to navigate the critical upfront planning required to launch such collaborative studies. The European Union-Patient-cEntric clinicAl tRial pLatform (EU-PEARL) initiative has produced new methodologies to expand the use of platform trials with an overarching infrastructure and services embedded into Integrated Research Platforms (IRPs), in collaboration with patient representatives and through consultation with U.S. Food and Drug Administration and European Medicines Agency stakeholders. In this narrative review, we discuss the outlook for platform trials in Europe, including challenges related to infrastructure, design, adaptations, data sharing and regulation. Documents derived from the EU-PEARL project, alongside a literature search including PubMed and relevant grey literature (e.g., guidance from regulatory agencies and health technology agencies) were used as sources for a multi-stage collaborative process through which the 10 more important points based on lessons drawn from the EU-PEARL project were developed and summarised as guidance for the setup of platform trials. We conclude that early involvement of critical stakeholder such as regulatory agencies or patients are critical steps in the implementation and later acceptance of platform trials. Addressing these gaps will be critical for attaining the full potential of platform trials for patients. Funding: Innovative Medicines Initiative 2 Joint Undertaking with support from the European Union’s Horizon 2020 research and innovation programme and EFPIA