Bringing platform trials closer to reality by enabling with digital research environment (DRE) connectivity.

Platform trials are generally regarded as an innovative approach to address clinical valuation of early stage candidates, regardless of modality as the evidence evolves. As a type of randomized clinical trial (RCT) design construct in which multiple interventions are evaluated concurrently against a common control group allowing new interventions to be added and the control group to be updated throughout the trial, they provide a dynamic and efficient mechanism to compare and potentially discriminate new treatment candidates. Their recent use in the evaluation of new therapies for COVID-19 has spurred new interest in the approach. The paucity of platform trials is less influenced by the novelty and operational requirements as opposed to concerns regarding the sharing of intellectual property (IP) and the lack of infrastructure to operationalize the conduct in the context of IP and data sharing. We provide a mechanism how this can be accomplished through the use of a digital research environment (DRE) providing a safe and secure platform for clinical researchers, quantitative and physician scientists to analyze and develop tools (e.g., models) on sensitive data with the confidence that the data and models developed are protected. A DRE, in this context, expands on the concept of a trusted research environment (TRE) by providing remote access to data alongside tools for analysis in a securely controlled workspace, while allowing data and tools to be findable, accessible, interoperable, and reusable (FAIR), version-controlled, and dynamically grow in size or quality as a result of each treatment evaluated in the trial.

A Modern Curriculum for Training Scientists in Model-Informed Drug Development: Progress Report on FDA Grant to Train Regulatory Scientists.

Under US Food and Drug Administration (FDA) grant (2U18FD005320-06), the Critical Path Institute (C-Path) and experienced private sector partners collaborated with global health organizations to create didactic video materials in an e-learning format on model-informed drug development (MIDD) topics relevant to a non-modeling audience. Several multinational pharmaceutical companies contributed case studies illustrating the application of the MIDD approach in practice. Training videos were created and divided into several modules: introducing the MIDD landscape for drug development and regulatory science, a review of various model types used for MIDD, discussions of how models inform drug development and regulatory decisions, future goals of MIDD, and discussions on the interconnectedness of models used for MIDD. Examples and vignettes from stakeholders and thought leaders were included. These educational materials fill a gap between academic and "on the job training" for regulators, academic, and industry scientists, delivering insights and value for those performing modeling and non-modelers reviewing the output of modeling and simulation work. A total of 13 hours of video content is currently available. A small panel of FDA reviewers is currently beta-testing the learning management system (LMS). Future efforts for this MIDD training initiative will include expansion of the content via an expanded and diverse faculty, 1:1 online mentorship sessions, and eventually broader access to this resource consistent with an open science approach and curriculum. The MIDD training LMS can accommodate a diverse learning ecosystem; further development may also accommodate different audiences in the future.

Advancing the utilization of real-world data and real-world evidence in clinical pharmacology and translational research-Proceedings from the ASCPT 2023 preconference workshop.

Real-world data (RWD) and real-world evidence (RWE) are now being routinely used in epidemiology, clinical practice, and post-approval regulatory decisions. Despite the increasing utility of the methodology and new regulatory guidelines in recent years, there remains a lack of awareness of how this approach can be applied in clinical pharmacology and translational research settings. Therefore, the American Society of Clinical Pharmacology & Therapeutics (ASCPT) held a workshop on March 21st, 2023 entitled "Advancing the Utilization of Real-World Data (RWD) and Real-World Evidence (RWE) in Clinical Pharmacology and Translational Research." The work described herein is a summary of the workshop proceedings.