The EveryLife Foundation for Rare Diseases has submitted formal comments to the Food and Drug Administration to inform their efforts to optimize the use of and processes of its advisory committees.
EveryLife is dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy. The comments reflect the insights and experiences of a diverse coalition of patient advocacy organizations, industry leaders, and other stakeholders. With over 30 million Americans affected by rare diseases, and fewer than 5% of the estimated 10,000 rare diseases having FDA-approved therapies, the urgency is clear. Despite significant progress in rare disease research and over 1120 orphan drug approvals, many unmet needs remain. The Foundation commends the FDA’s efforts to review and reform its advisory committees to better serve patients and ensure timely access to safe and effective therapies.
