Table 1 Comparison of FDA and EMA criteria for orphan drug designation
FDA Criteria [10] | EMA Criteria [7] |
|---|---|
Drugs (includes biologics) for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US OR | The drug must be intended for a disease that is life-threatening or chronically debilitating |
| Â | The prevalence of the condition must not be more than 5 in 10,000; AND |
Drugs that will not be profitable within 7Â years following approval by the FDA | The medicine must be of significant benefit to those affected by the condition |