Table 2.
The attributes for selection of 10 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 |
Abbreviations: O Orphan Medicinal Product, UO Ultra-orphan medicinal product
Sources: a The European public assessment reports (EPAR) for human medicines published by the European Medicines Agency (EMA) http://www.ema.europa.eu/ema/
bNational Health Insurance Fund Administration of Hungary (Országos Egészségbiztosítási Pénztár, OEP)