Monday, September 25, 2023

Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics

May 23, 2010 by  
Filed under Featured Stories

For the past week, I have been obsessing over rare disease statistics ever since it was published that 80% of all rare diseases are ‘ultra orphans’ – affecting 6,000 people per disease state or less – and only 15% of all rare disease drug applications filed with the FDA are for ‘ultra orphans.’ Yesterday, I […]

New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations

May 22, 2010 by  
Filed under Featured Stories

The Kakkis Every Life Foundation is leading the effort to request $10 million in the fiscal year 2011 Ag-Rural Development-FDA Appropriations bill directing the U.S. Food and Drug Administration to establish a new review division for Biochemical and Genetic Diseases within the Center for Drug Evaluation Research (CDER), Office of New Drugs. A letter from […]