Table 1 Orphan and non-orphan medicine (adapted from Spilker B)
From: A methodological framework for drug development in rare diseases
Orphan medicine | Non-orphan medicine |
|---|---|
Used in a limited patient population | Used in a large patient population |
Often used by only a few specialists | Generally used by a wide variety or number of physicians |
The manufacturer often loses money | The manufacturer is more likely to make money |
May require less patient exposure to obtain marketing authorization | Usually requires a standard quantity of data before marketing authorization |