Orphan Drug Designations

We grasp the distinctions between Orphan Drug Designation (ODD) criteria in the United States and the European Union. In the U.S., the designation often pertains to the disease, offering a broader scope than the eventually approved label. In contrast, the EU necessitates the broadest possible indication.

For the U.S., the disease or condition's prevalence must be under 200,000 (affecting fewer than 200,000 people). In the EU, the threshold is even stricter, with the disease or condition affecting less than five in 10,000 people.

Notably, in the EU, ODD applicability must be reaffirmed upon approval (marketing authorization), a requirement absent in the U.S. Furthermore, the EU offers longer market exclusivity, granting a 10-year period, extendable to 12 years with an agreed Pediatric Investigation Plan (PIP) in place. Conversely, ODD in the U.S. provides the sponsor with seven years of market exclusivity.

Halloran typically files around seven ODD applications per year. While most are granted, some are not, providing us with unique insights into current FDA expectations.