Table 1.
HTA recommendations issued between 2007 and 2011 for orphan drug technologies by AHTAPol.
| No. | Brand name (active substance) | Indication | HTA guidance | Reason for HTA recommendation |
|---|---|---|---|---|
| 1 | Fabrazyme (Agalsidase beta) | Fabry disease (alpha-galactosidase A deficiency) – long-term replace therapy | Negative | Insufficient clinical effectiveness, safety concerns, not cost-effective vs standard of care |
| 2 | Elaprase (Idursulfase) | Mucopolysaccharidosis type II, MPS II (Hunter syndrome) – long-term treatment | Negative | Insufficient clinical effectiveness |
| 3 | Kuvan (Tetrahydrobiopterin) | Hyperphenylalaninemia (HPA) in patients with tetrahydrobiopterin (BH4) deficiency | Positive | Restricted with time limits |
| 4 | Increlex (Mecasermin) | Insulin-like growth factor deficiency -IGFD (Laron Syndrome) – long-term treatment | Positive | Restricted with time limits |
| 5 | Somavert (Pegvisomant) | Acromegaly | Negative | Insufficient clinical effectiveness, not cost-effective vs standard of care |
| 6 | Ventavis (Iloprost) | Pulmonary arterial hypertension (PAH) | Positive | Restricted to subpopulations |
| 7 | Tracleer (Bosentan) | Pulmonary arterial hypertension (PAH) | Positive | Restricted to subpopulations |
| 8 | Cystadane (Betaine anhydrous) | Homocystinuria | Positive | Unrestricted |
| 9 | Zavesca (Miglustat) | Niemann-Pick type C syndrome (disease) | Positive | Restricted with time limits, conditional on the reduction of cost of therapy |
| 10 | Volibris (Ambrisentan) | Pulmonary arterial hypertension (PAH) | Positive | Unrestricted |
| 11 | Nexavar (Sorafenib) | Renal cell carcinoma (RCC) | Negative (2008) | Insufficient clinical effectiveness, not cost-effective vs standard of care, unacceptable budget impact |
| 12 | Nexavar (Sorafenib) | Renal cell carcinoma (RCC) | Negative (2009) | Not cost-effective vs standard of care, unacceptable budget impact |
| 13 | Nexavar (Sorafenib) | Hepatocellular carcinoma (HCC) | Positive | Restricted to subpopulations |
| 14 | Nplate (Romiplostim) | Chronic immune (idiopathic) thrombocytopenic purpura (ITP) | Positive | Restricted to subpopulations |
| 15 | Torisel (Temsirolimus) | Renal cell carcinoma (RCC) | Negative | Insufficient clinical effectiveness, not cost-effective vs standard of care |
| 16 | Tasigna (Nilotinib) | Chronic myeloid leukemia (CML) | Positive | Restricted to subpopulations, conditional on the reduction of cost of therapy |
| 17 | Vidaza (Azacitidine) | Acute myelogenous leukemia (AML) | Positive | Unrestricted |
| 18 | Glivec (imatinib) | Philadelphia chromosome positive chronic myeloid leukemia (ALL Ph+) | Positive | Unrestricted |
| 19 | Glivec (imatinib) | Myelodysplastic/myeloproliferative diseases (MDS/MPD) | Positive | Unrestricted |
| 20 | Glivec (imatinib) | Dermatofibrosarcoma protuberans (DFSP) | Positive | Unrestricted |
| 21 | Glivec (imatinib) | Malignant gastrointestinal stromal tumors (GIST) | Positive | Unrestricted |
| 22 | Revlimid (Lenalidomide) | Multiple myeloma (MM) | Positive | Restricted to subpopulations |
| 23 | Revlimid (Lenalidomide) | Myelodysplastic/Myeloproliferative syndrome (MM/S) (off-label indication) | Positive | Off-label indication, restricted to subpopulations, conditional on the reduction of cost of therapy |
| 24 | Yondelis (Trabectedin) | Soft tissue sarcoma | Positive | Restricted to subpopulations, conditional on the reduction of cost of therapy |
| 25 | Sprycel (Dasatinib) | Chronic myeloid leukemia (CML) | Positive | Restricted to subpopulations |
| 26 | Revatio (Sildenafil) | Pulmonary arterial hypertension (PAH) | Positive | Unrestricted |
| 27 | Atriance (Nelarabine) | T-cell acute lymphoblastic leukemia | Positive | Restricted to subpopulations, conditional on the reduction of cost of therapy |