TEAMSS – Transforming Expanded Access to Maximize Support & Study

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Project Summary The Expanded Access (EA) program of the Food and Drug Administration (FDA) provides an opportunity for patients who either lack therapeutic options or who are ineligible for clinical trials to potentially benefit from the clinical use of experimental drugs, biologics, and medical devices. This process represents one of the most immediate forms of translation because an experimental therapy is administered directly to an individual patient in need. However, the individual nature of EA interventions, the silos of care environments utilizing them, and the lack of standardized reporting have led to the inability to make broader inferences about safety or therapeutic potential. A more coordinated approach to EA that can achieve greater integration and yield more robust information could be transformative for the care of patients with rare or refractory disease. The NIH Clinical and Translational Science Awards (CTSA) consortium is an ideal network to develop innovative methods for regulatory navigation and support, oversight, and delivery of investigational products through EA. We therefore propose a CTSA-based program focused on Transforming Expanded Access to Maximize Support and Study (TEAMSS). Our long-term goal is to advance clinical care and translational research by improving patient access to experimental therapies through a federated, national consortium for EA interventions. The goal of this application is to develop a CTSA-based network that can demonstrate successful multisite integration of EA programs, leading to dissemination of best practices. We will pursue this goal by accomplishing the following specific aims: Develop, demonstrate, and disseminate best practices of network-based Expanded Access programs across the CTSA consortium; Develop a network for cohort-based Expanded Access programs; Create a database to standardize Expanded Access data reporting and develop a body of real-world data. The expected outcome of these aims is to demonstrate that a network-based approach to EA interventions and outcomes is both feasible and beneficial. We also expect to have achieved a thorough landscape assessment of EA infrastructure at each TEAMSS hub, as well as the successful dissemination of best practices that are applicable across the CTSA consortium. This will create a positive impact by, for the first time, creating a foundation for an integrated, nationwide approach to EA that can improve care for the most vulnerable patients.