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Table 1 HTA recommendations issued between 2007 and 2011 for orphan drug technologies by AHTAPol.

From: Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs

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