Table 1 HTA outcomes for OMPs issued by the Polish HTA agency between
Entry | Brand name (Active substance) | Indication | HTA outcome | Data source | Reason for HTA recommendation |
|---|---|---|---|---|---|
1 | Adcetris (brentuximab vedotin) | Lymphoma CD30+: Hodgkin Disease (C81), Lymphoma, Non-Hodgkin (C84.5) | Negative | Rekomendacja prezesa AOTM nr 96/2013 | Clinical reasons: insufficient evidence for use (poor quality data). Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
2 | Adempas (riociguat) | Chronic Thromboembolic Pulmonary Hypertension (CTEPH) (ICD-10 I27, I27.O and/or I26) | Positive | Rekomendacja prezesa AOTM nr 261/2014 | Minor restrictions: use at lower price |
3 | Arzerra (ofatumumab) | Chronic Lymphocytic Leukemia in patients who are refractory to fludarabine and alemtuzumab | Negative | Rekomendacja prezesa AOTM nr 5/2012 | Clinical reasons: insufficient evidence for use (poor quality data). Economic reasons: unacceptable budget impact, insufficient justification of the treatments cost in relation to its benefit |
4 | Atriance (Nelarabine) | treatment of patients with T-cell acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens, eligible for a bone marrow transplant | Positive | Stanowisko Rady Konsultacyjnej nr 13/04/2009 | Minor restrictions: use at lower price |
5 | Bramitob (tobramycin) | treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis (ICD-10 E84) | Negative | Rekomendacja prezesa AOTM nr 83/2013 | Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
6 | Cometriq (cabozantinib) | Thyroid Neoplasms (ICD-10 C73) | Negative | Rekomendacja prezesa AOTM nr 51/2015 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data), poor safety. Economic reasons: poor economic data, insufficient justification of the treatments cost in relation to its benefit |
7 | Cystadane (Betaine anhydrous) | Homocystinuria | Positive | Rekomendacja prezesa AOTM nr 6/2010, Stanowisko Rady Konsultacyjnej nr 9/3/2010 | Minor restriction: monitoring required |
8 | Elaprase (Idursulfase) | Mucopolysaccharidosis type II, MPS II (Hunter syndrome) – long-term treatment | Negative | Komunikat na stronie www AOTM | Clinical reasons: insufficient evidence for use (poor quality data) |
9 | Esbriet (pirfenidone) | Idiopathic Pulmonary Fibrosis (ICD-10 J 84.1) | Negative | Rekomendacja prezesa AOTM nr 79/2013 | Clinical reasons: insufficient evidence for use (poor quality data), poor safety |
10 | Evoltra (clofarabine) | Treatment of acute lymphoblastic leukaemia (ALL) in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens and where there is no other treatment option anticipated to result in a durable response, in patients eligible for a hemapoietic stem cell transplant | Positive | Rekomendacja prezesa AOTM nr 127/2012 | Major restriction: used restricted to specific subpopulation |
11 | Exjade (deferasirox) | Treatment of chronic iron overload | Positive | Rekomendacja prezesa AOTM nr 68/2012 | Minor restriction: monitoring required |
12 | Fabrazyme (Agalsidase beta) | Fabry disease (alpha-galactosidase A deficiency) – long-term replace therapy | Negative | Stanowisko Rady Konsultacyjnej nr 20/06/2009 | Clinical reasons insufficient evidence for use (inappropriate comparator or poor quality data), poor safety. Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
13 | Firazyr (icatibant) | Treatment of acute attacks of hereditary angioedema (HAE) in adults (with C1-esterase-inhibitor deficiency) | Negative | Rekomendacja prezesa AOTM nr 22/2015 | Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
14 | Gazyvaro (obinutuzumab) | Chronic lymphocytic leukaemia (CLL) (ICD-10: C.91.1) | Negative | Rekomendacja prezesa AOTM nr 60/2015 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data), poor safety. Economic reasons: poor economic data, insufficient justification of the treatments cost in relation to its benefit |
15 | Glivec (Imatinib) | Myelodysplastic/myeloproliferative diseases (MDS/MPD) | Positive | Rekomendacja prezesa AOTM nr 7/2011 | Unrestricted |
16 | Glivec (Imatinib) | Dermatofibrosarcoma protuberans (DFSP) | Positive | Rekomendacja prezesa AOTM nr 5/2011 | Unrestricted |
17 | Glivec (Imatinib) | Malignant gastrointestinal stromal tumors (GIST) | Positive | Komunikat wraz z uzasadnieniem na stronie AHTAPol | Unrestricted |
18 | Glivec (Imatinib) | Philadelphia chromosome positive chronic myeloid leukemia (ALL Ph+) | Positive | Rekomendacja prezesa AOTM nr 6/2011 | Unrestricted |
19 | Increlex (Mecasermin) | Insulin-like growth factor deficiency –IGF-1 (Laron Syndrome) – long-term treatment | Positive | Stanowisko Rady Konsultacyjnej nr 43/12/2009 | Major restriction: resubmission required after certain time |
20 | Jakavi (ruxolitinib) | primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythaemia-vera myelofibrosis or post-essential-thrombocythaemia myelofibrosis | Positive | Rekomendacja prezesa AOTM nr 120/2014 | Unrestricted |
21 | Kalydeco (ivacaftor) | Cystic fibrosis (CF) (ICD-10 E84) | Negative | Rekomendacja prezesa AOTM nr 54/2015 | Clinical reasons: insufficient evidence for use (poor quality data). Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
22 | Kuvan (sapropterin) | Hyperphenylalaninemia (HPA) in patients with tetrahydrobiopterin (BH4) deficiency | Positive | Rekomendacja prezesa AOTM nr 55/2011 | Major restriction: resubmission required after certain time |
23 | Mabthera (rituximab) | Non-Hodgkin’s lymphoma (NHL) | Positive | Rekomendacja prezesa AOTM nr 7/2012 | Major restriction: used restricted to specific subpopulation |
24 | Mabthera (rituximab) | Non-Hodgkin’s lymphoma classified to code ICD-10 C84 | Negative | Rekomendacja prezesa AOTM nr 24/2012 | Clinical reason: insufficient evidence for use (inappropriate comparator or poor quality data) |
25 | Mabthera (rituximab) | Code ICD-10 C85 (Other and unspecified types of non-Hodgkin lymphoma) | Positive | Rekomendacja prezesa AOTM nr 25/2012 | Unrestricted |
26 | Mabthera (rituximab) | Hodgkin Lymphoma (Hodgkin disease-HD) | Positive | Rekomendacja prezesa AOTM nr 19/2012 | Major restriction: used restricted to specific subpopulation |
27 | Mepact (mifamurtide) | Osteosarcoma (ICD-10 C40–41) | Negative | Rekomendacja prezesa AOTM nr 78/2013 | Clinical reasons: insufficient evidence for use (poor quality data). Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
28 | Mozobil (plerixafor) | In combination with granulocyte-colony-stimulating factor to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma whose cells mobilise poorly (ICD-10: C81–85, C90) | Negative | Rekomendacja prezesa AOTM nr 182/2013 | Clinical reason: poor safety. Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
29 | Myozyme (alglucosidase alfa) | Pompe disease (acid-α-glucosidase deficiency) (ICD-10 E74.0) | Negative | Rekomendacja prezesa AOTM nr 8/2013 | Clinical reason: insufficient evidence for use (poor quality data). Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
30/31 | Nexavar (Sorafenib) | Renal cell carcinoma (RCC) | Negative | Rekomendacja prezesa AOTM nr 48/2009, Stanowisko Rady Konsultacyjnej nr 27/10/26/2009, and Uchwała Rady Konsultacyjnej nr 22/07/2008 | Clinical reasons: insufficient evidence for use (poor quality data), poor efficacy. Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
32 | Nexavar (Sorafenib) | Hepatocellular carcinoma (HCC) | Positive | Rekomendacja prezesa AOTM nr 26/2010 | Major restriction: used restricted to specific subpopulation |
33 | Nplate (Romiplostim) | Chronic immune (idiopathic) thrombocytopenic purpura (ITP) | Positive | Rekomendacja prezesa AOTM nr 13/2010 | Major restriction: used restricted to specific subpopulation |
34 | Opsumit (macitentan) | long-term treatment of pulmonary arterial hypertension (PAH) in combination (ICD-10 I27, I27.0) | Negative | Rekomendacja prezesa AOTM nr 23/2015 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data). Economic reasons: unacceptable budget impact |
35 | Revatio (Sildenafil) | Pulmonary arterial hypertension (PAH) | Positive | Uchwała Rady Konsultacyjnej nr 1/01/2008 | Unrestricted |
36 | Revlimid (Lenalidomide) | Myelodysplastic/Myeloproliferative syndrome (MM/S) (off-label indication) | Positive | Rekomendacja prezesa AOTM nr 83/2011 | Off-label indication. Major restriction: used restricted to specific subpopulation. Minor restriction: use at lower price |
37 | Revlimid (Lenalidomide) | Myelodysplastic/Myeloproliferative syndrome (MM/S) | Positive | Rekomendacja prezesa AOTM nr 11/2012 | Major restriction: used restricted to specific subpopulation. Minor restriction: use at lower price |
38 | Signifor (pasireotide) | Cushing’s disease for whom surgery is not an option or for whom surgery has failed (ICD-10 E 24.0) | Positive | Rekomendacja prezesa AOTM nr 99/2013 | Minor restriction: use at lower price |
39 | Somavert (Pegvisomant) | Acromegaly | Negative | Rekomendacja prezesa AOTM nr 4/2011 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data), poor efficacy. Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
40 | Sprycel (Dasatinib) | Chronic myeloid leukemia (CML) | Positive | Uchwała Rady Konsultacyjnej nr 23/07/2008 | Major restriction: use only as second or subsequent line treatment |
41 | Sprycel (dasatinib) | Indication clacisified to codes: ICD10:C96.2, within non-standard chemiotherapy programme | Negative | Rekomendacja prezesa AOTM nr 66/2014 | Clinical reasons: poor efficacy |
42 | Sutent (sunitinib) | unresectable or metastatic malignant gastrointestinal stromal tumors (GIST) in adults with disease progression | Positive | Rekomendacja prezesa AOTM nr 20/2012 | Unrestricted |
43 | Tasigna (Nilotinib) | Chronic myeloid leukemia (CML) with resistance or intolerance to prior therapy | Positive | Uchwała Rady Konsultacyjnej nr 53/15/2008 | Major restriction: use only if intolerant to other treatment. Minor restriction: use at lower price |
44 | Thalidomide Celgene (thalidomide) | In combination with melphalan and prednisone as first-line treatment of patients with untreated multiple myeloma aged ≥65 years or ineligible for high-dose chemotherapy | Positive | Rekomendacja prezesa AOTM nr 106/2012 | Minor restriction: use at lower price |
45 | Torisel (Temsirolimus) | Renal cell carcinoma (RCC) | Negative | Rekomendacja prezesa AOTM nr 47/2009. Stanowisko Rady Konsultacyjnej nr 26/10/26/2009 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data). Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
46 | Torisel (temsirolimus) | treatment of adult patients with advanced renal-cell carcinoma (RCC) with unfavorable prognostic (ICD-10: C64) (RCC-up) | Negative | Rekomendacja prezesa AOTM nr 58/2013 | Clinical reasons: insufficient evidence for use (inappropriate comparator or poor quality data), poor safety. Economic reasons: insufficient justification of the treatments cost in relation to its benefit |
47 | Tracleer (Bosentan) | Pulmonary arterial hypertension (PAH) | Positive | Uchwała Rady Konsultacyjnej nr 1/01/2008 | Major restrictions: used restricted to specific subpopulation, use only as second or subsequent line treatment. Minor restriction: monitoring required. |
48 | Trisenox (arsenic trioxide) | for induction of remission and consolidation in adult patients with pro-Myelotic Leucaemia (APL)/ Retinoic-Acid receptor-alpha PML/RAR alpha | Negative | Rekomendacja prezesa AOTM nr 6/2012 | Clinical reason: insufficient evidence for use (poor quality data). Economic reason: insufficient justification of the treatments cost in relation to its benefit |
49 | Ventavis (Iloprost) | Pulmonary arterial hypertension (PAH) | Positive | Uchwała Rady Konsultacyjnej nr 1/01/2008 | Major restrictions: used restricted to specific subpopulation, use only as second or subsequent line treatment. Minor restriction: monitoring required. |
50 | Vidaza (Azacitidine) | Acute myelogenous leukemia (AML) | Positive | Rekomendacja prezesa AOTM nr 18/2011 | Unrestricted |
51 | Volibris (Ambrisentan) | Pulmonary arterial hypertension (PAH) (ICD-10 I27, I27.0) | Positive | Rekomendacja prezesa AOTM nr 29/2010 | Unrestricted |
52 | Votubia (everolimus) | Treatment of patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) who require therapeutic intervention but are not amenable to surgery (ICD-10 Q85.1) | Positive | Rekomendacja prezesa AOTM nr 81/2014 | Minor restriction: use at lower price |
53 | Vpriv (velaglucerase alfa) | Gaucher disease (ICD-10: E-75) | Positive | Rekomendacja prezesa AOTM nr 120/2013 | Minor restriction: use at lower price |
54 | Yondelis (Trabectedin) | Soft tissue sarcoma | Positive | Rekomendacja prezesa AOTM nr 19/2011 | Major restriction: used restricted to specific subpopulation. Minor restriction: use at lower price |
55 | Zavesca (Miglustat) | Niemann-Pick type C syndrome (disease) | Positive | Rekomendacja prezesa AOTM nr 20/2011 | Major restriction: resubmission required after certain time Minor restrictions: use at lower price, monitoring required |
56 | Xagrid (anagrelide) | Indication classified to codes: ICD-10: D.45 with extensions and D.47 with extensions | Positive | Rekomendacja prezesa AOTM nr 142/2013 | Unrestricted |
57 | Xagrid (anagrelide) | Chronic myeloid leukemia (CML) (ICD-10 C92.1) | Negative | Rekomendacja prezesa AOTM nr 161/2013 | Clinical reasons: insufficient evidence for use (poor quality data) |