The RARE Foundation, joined by other leading patient advocacy organizations, submitted a proposal to the FDA requesting a Rare Disease Innovation, Science, and Exploration (RISE) Workshop. The goal: to collectively optimize risk assessment and mitigation strategies, including rapid exchange of shared learning across the gene therapy community.
In recent days, the urgency to ensure that the life-altering momentum of gene therapy delivery continues has been felt by our rare disease community more intensely than ever before.
We believe a RISE Workshop convened by FDA provides a forum for developers, regulators, and patient organizations to discuss and take actions to address cross-cutting challenges and opportunities. This would advance scientific and regulatory benefit risk approaches that can help ensure we realize the full potential of the gene therapy pipeline, while mitigating current and future safety concerns.
Once again, the RARE Foundation is proud to be part of a community that is galvanized and ready to work with regulators to ensure patients have access to potentially life-saving therapies.
