Table 2.
Orphan drugs with highest number of users, highest prices and highest budget impact (2012)
| Rank | INN | Trade name | Setting | Patients | |
| 1 | Imatinib | Glivec® | Outpatient | 1,485 | |
| 2 | Lenalidomide | Revlimid® | Outpatient | 1,089 | |
| 3 | Sildenafil | Revatio® | Outpatient | 1,052 | |
| 4 | Thalidomide | Thalidomide Cellgene® | Outpatient | 1,030 | |
| 5 | Bosentan | Tracleer® | Outpatient | 901 | |
| Rank | INN | Trade name | Setting | Cost/Patient | |
| 1 | Galsulfase | Naglazyme® | Inpatient | €600,000 | |
| 1 | Idursulfase | Elaprase® | Inpatient | €600,000 | |
| 3 | Alglucosidase alfa | Myzoyme® | Inpatient | €474,857* | |
| 4 | Eculizumab | Soliris® | Inpatient | €358,000 | |
| 5 | Aldurazyme | Laronidase® | Inpatient | €300,000 | |
| Rank | INN | Trade name | Setting | Budget impact (millions) | Cumulative budget impact 2006–2012 (millions) |
| 1 | Alglucosidase alfa | Myozyme® | Inpatient | €40.3 | €203.4 |
| 2 | Imatinib | Glivec® | Outpatient | €36.4 | €251.2 |
| 3 | Lenalidomide | Revlimid® | Outpatient | €36.2 | €127.4 |
| 4 | Bosentan | Tracleer® | Outpatient | €23.0 | €132.4 |
| 5 | Pegvisomant | Somavert® | Outpatient | €14.3 | €72.5 |
INN = International Nonproprietary Names; *weighted average of infantile patients (€706,666/patient) and adult patients (€422,314/patient).