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.