Food and Drug Administration, Center for Drug Evaluation and Research Rare Diseases Public Workshop: Strategies, Tools, and Best Practices for Effective Advocacy in Rare Diseases Drug Development

摘要

The Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), is sponsoring a public workshop entitled "CDER Rare Diseases Public Workshop: Strategies, Tools, and Best Practices for Effective Advocacy in Rare Diseases Drug Development." This public workshop builds upon previous CDER patient advocacy public workshops and is primarily for the rare disease community to help them effectively understand what FDA needs to enhance drug development. This effort is consistent with FDA's efforts to support the integration of patient experience in drug development programs, including through implementation of the "Patient-Focused Drug Development" provisions of the 21st Century Cures Act (Cures Act). This public workshop will include case studies demonstrating the beneficial overlap of effective advocacy techniques and FDA regulations in rare disease drug development.