Orphan Drug Exclusivity and Pediatric Exclusivity: Prevalence
• Click on the following link to go to orpha.net, the portal for rare diseases and orphan drugs.
• Search for beta thalassemia in the US and European Union
• Identify a novel or potential iron chelator therapy under orphan drug designation due blood transfusion for beta thalassemia (i.e., treatment for iron overload due to blood transfusion to treat beta thalassemia)
• Identify the prevalence of the population affected in both regions (US and EU) and explain how that meets one of the Orphan drug designation requirements in both regions (i.e., prevalence)
• Discuss some application challenges regarding orphan drug applications and pediatric development applications in both regions.