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 |