Table 1: SMA drug: year 1: € 240,000 and NOT € 249,000.
| Disease | Drugs | Price* |
| Annual | ||
| Paroxysmal nocturnal hemoglobinuria (PNH) | Eculizumab | €358,000 |
| Hunter | Idursulfase | €600,000 |
| Cystic fibrosis (CF) | Lumacaftor/Ivacaftor | €169,386 |
| Cystic fibrosis (CF) | Ataluren | €270,000 |
| Primary biliary cholangitis (PBC) | Obeticholic acid | €38,021 |
| Spinal Muscular Atrophy (SMA) | Nusinersen | Year 1: €240,000 > year 1: €249,000 |
| Neuronal ceroid lipofuscinosis, late infantile type 2 (CLN2) | Cerliponase alpha | €595,971 |
| Metabolic disease - alpha-mannosidosis lysosomal disease | Velmanase alpha | € 500.000 |
| Congenital or acquired lipodystrophy | Metreleptin | €480,000 |
*Prices were based on horizon scan by health authorities.