| A (2) | B (4) | C (3) | D (2) | E (1) | total |
---|
Amifampridine | 6 | 12 | 9 | 6 | 3 | 36 |
Sodium phenylbutyratea | 6 | 12 | 9 | 2 | 3 | 32 |
Pyridoxal phosphatea | 6 | 8 | 9 | 6 | 3 | 32 |
Chenodesoxycholic acida | 2 | 12 | 9 | 6 | 3 | 30 |
Primaquine phosphatea | 2 | 12 | 9 | 6 | 3 | 30 |
Fenfluramine | 2 | 12 | 6 | 6 | 3 | 29 |
Sodium hydroxybutyrate | 2 | 12 | 3 | 6 | 3 | 26 |
Difencyprone | 6 | 4 | 6 | 6 | 3 | 25 |
Bimyconase | 2 | 8 | 6 | 2 | 1 | 19 |
- Priority list of the OAIs based on five criteria with (A) the prevalence of the corresponding rare disease, (B) severity of the disease, (C) degree of available evidence, (D) price of the orphan active ingredient and (E) the ease of compounding. The weighing factor (which was multiplied with the score of the individual criterium) indicating the criteriums importance is mentioned between brackets. aOAIs included in current study