|
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
|
