NeuroREACH™ Platform for Expanded Access Program (EAP) Trials as the Foundation for Clinical Research of the Future (5463)

Objective: Develop a platform for rapid deployment of neurological EAP clinical trials to test investigational therapies and aggregate safety data in a compliant regulatory setting Background: Expanded Access Programs provide investigational therapies to patients who are not eligible or able to participate in clinical trials but have serious or life-threatening diseases, for which their physician has determined there are no appropriate treatment options. Many pharma/biotech offer investigational products and many investigators agreeing or insisting upon running multiple EAP trials for the benefit of their patient population who fail eligibility criteria of “regular” clinical trials. Introduction of a regulatory-compliant platform to execute EAPs became a necessity. The Center for Innovation and Bioinformatics (CIB) introduced the NeuroREACH™ platform to facilitate: - Rapid deployment of EAP trials - Capture of clinical and safety data from patients - Data Management - Real-time secure and controlled access to data visualization and reports Design/Methods: Any investigator with an IRB-approved EAP protocol and open enrollment of patients may capture and store phenotypical, safety, and biomarker data utilizing the NeuroREACH EDC platform. The CIB acts as the Data Coordination Center and its program management team will oversee the addition of new studies, data curation, visualization, and reporting. Results: The NeuroREACH platform has been deployed at CIB and available to investigators and sponsors around the world. Currently FOUR EAP trials utilize NeuroREACH. As patients may participate in multiple EAPs, NeuroGUIDs/NeuroSTAmPs are used to uniquely identify EAP participants across studies. Study sponsors and drug manufacturers have access to reports and dashboards to track enrollment data in real time. Conclusions: As EAPs become more and more prevalent method to test investigational products for safety and even efficacy, the investigators and study sponsors may use NeuroREACH to rapidly deploy trials. The captured EAP information utilized by pharma/biotech for new indications, safety analyses, and in negotiations with the FDA and insurance companies Disclosure: Yes - My institution received research funding on my behalf from Amylyx Pharmaceuticals, Origent Data Sciences, Mitsubishi Tanabe Pharma AmericaDr. Podesta has nothing to disclose. Dr. Walker has nothing to disclose. Dr. Yu has nothing to disclose. Dr. D’Agostino has nothing to disclose. Dr. Tarasenko has nothing to disclose. Dr. Sinani has nothing to disclose. Dr. Luppino has nothing to disclose. Dr. Scalia has nothing to disclose. Dr. Paganoni has received research support from Amylyx.Dr. Cudkowicz has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with takeda, biogen, cytokinetics, sunovian, immunitypharm, avexis.