Evolution of orphan drug – Healthcare Economist

A examine by Fermaglich and Miller (2023) evaluated tendencies in orphan drug designations and approvals after the Orphan Drug Act of 1983 was handed within the US. The authors use FDA information to guage between 1983 and 2022 and discover that:

Over the 40 years of the ODA, 6,340 orphan drug designations had been granted, representing drug growth for 1,079 uncommon illnesses. Moreover, 882 of these designations resulted in at the least one FDA approval to be used in 392 uncommon illnesses. A lot of this growth has been concentrated in oncology as seven of the highest ten most designated and accredited illnesses had been uncommon cancers.

Of the orphan drug designations (approvals), the highest 5 prime illnesses areas had been:

• Oncology: 38% of designations (38% of approvals)
• Neurology: 14% (10%)
• Infectious Illness: 7% (10%)
• Metabolism 6% (7%)
• Hematology: 5% (8%)

You will discover extra element on orphan drug designations and approvals within the full paper here.