Comment from United Porphyrias Association

Summary: The United Porphyrias Association, a patient advocacy organization, supports the Haystack Project's petition for rulemaking to create a disease-contextual framework for rare disease drug development. They argue that the current "one-size-fits-all" FDA model fails to account for the unique clinical realities of rare diseases like porphyria, and that a new framework would better incorporate disease-specific biomarkers, patient experience, and expert consultation.