Testimony to FDA on Rare Diseases

Testimony of the Endocrine Society Submitted to the Food and Drug Administration Endocrinologic and Metabolic Drugs Advisory Committee

"Many endocrine conditions qualify as rare diseases as they affect fewer than 200,000 people each year, but patients lack effective treatments for many of these diseases. Although the FDA has approved more than 500 treatments since the passage of the Orphan Drug Act, only five percent of rare diseases have a treatment...Ongoing research to identify new treatments for many of these diseases could have a significant benefit to patients. The Society advocates for increased research funding for rare diseases and supports the FDA’s flexibility regarding clinical endpoints, patients sample sizes, and inclusion/exclusion criteria for clinical trials to increase our knowledge of rare diseases and to expand therapeutic options for treating these conditions..."

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