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Table 1 HTA outcomes for OMPs issued by the Polish HTA agency between

From: Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis

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)