Table 3: The results from the base case analysis.
Disease | Drugs | Price | Break-even price | Mark-up* | |
Annual | Annual | Absolute | % | ||
Paroxysmal nocturnal hemoglobinuria (PNH) | Eculizumab | €358,000 | €458,870 | -€100,870 | -22% |
Hunter syndrome | Idursulfase | €600,000 | -€476,579 | €476,579 | -44% |
Cystic fibrosis (CF) | Lumacaftor/ivacaftor | €169,386 | €65,861 | €103,525 | 157% |
Cystic fibrosis (CF) | Ataluren | €270,000 | €254,464 | €15,536 | 6% |
Primary biliary cholangitis (PBC) | Obeticholic acid | €38,021 | €46,652 | -€8,631 | -19% |
Spinal Muscular Atrophy (SMA) | Nusinersen | €240,000 | €95,860 | €144,140 | 150% |
Neuronal ceroid lipofuscinosis, late infantile type 2 (CLN2) | Cerliponase alpha | €595,971 | €799,744 | -€203,773 | -25% |
Metabolic disease - alpha-mannosidosis lysosomal disease | Velmanase alpha | €800,000 | €799,744 | €256 | 0.1% |
Congenital or acquired lipodystrophy | Metreleptin | €480,000 | €509,623 | -€29,623 | -6% |
*A positive difference indicates that actual prices are higher than break-even prices and a negative difference indicates that actual prices are lower than break-even prices.