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Table 2 The attributes for selection of 10 Orphan Medicinal Products

From: The European challenges of funding orphan medicinal products

  Field of the indicationa Other therapeutic alternative(s)?a Clinical/Relative Effectiveness (potentially curative/non-curative treatment)a Rarity (orphan/ ultra-orphan status)a Cost commitmentb
Alglucosidase alfa Glycogen storage disease type II No Incremental (non-curative) UO Medium
Eculizumab Paroxysmal nocturnal haemoglobinuria or for atypical haemolytic uremic syndrome No Major (non-curative) O High
Icatibant Hereditary angioedema Yes Major (non-curative) O Medium
Idursulfase Mucopolysaccharido-sis type II No Incremental (non-curative) UO High
Nelarabine Special types of leukaemia or lymphoma No Curative O Medium
Rufinamide Lennox–Gastaut syndrome Yes Major (non-curative) O Low
Romiplostim Idiopathic thrombocytopenic purpura No Curative O Medium
Sapropterin Phenylketonuria Yes Major (non-curative) O Low
Sildenafil Pulmonary hypertension No Major (non-curative) O Low
Trabectedin Sarcomas and ovarian neoplasms No Incremental (non-curative) O High
  1. Abbreviations: O Orphan Medicinal Product, UO Ultra-orphan medicinal product
  2. Sources: a The European public assessment reports (EPAR) for human medicines published by the European Medicines Agency (EMA)
  3. bNational Health Insurance Fund Administration of Hungary (Országos Egészségbiztosítási Pénztár, OEP)