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Tytuł:
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Estimating the annual economic burden for the management of patients with transthyretin amyloid polyneuropathy in Spain.
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Autorzy:
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Galan L; Neurology, Hospital Clínico San Carlos, Madrid, Spain.
Gonzalez-Moreno J; Internal Medicine Services, Hospital Universitario Son Llàtzer, Palma De Mallorca, Spain.
Martínez-Sesmero JM; Pharmacy Services, Hospital Clínico San Carlos, Madrid, Spain.
Muñoz-Beamud F; Internal Medicine Services, Hospital Universitario Juan Ramón Jiménez, Huelva, Spain.
Santos-Rubio MD; Pharmacy Services, Hospital Juan Ramón Jimenez, Huelva, Spain.
Tran D; Value & Evidence, EVERSANA Life Science Services, Burlington, Canada.
Lebeau P; Value & Evidence, EVERSANA Life Science Services, Burlington, Canada.
Stewart M; Patient & Health Impact, Pfizer Inc, Groton, CT, USA.
Mallaina P; ATTR Amyloidosis/Rare Cardiology, Pfizer S.L.U, Madrid, Spain.
Tarilonte P; Rare Disease Medical Department, Pfizer S.L.U, Alcobendas, Madrid, Spain.
Peral C; Health Economics and Outcomes Research Department, Pfizer S.L.U, Alcobendas, Madrid, Spain.
Rozenbaum MH; Patient & Health Impact, Pfizer Inc, Capelle Aan Den IJssel, The Netherlands.
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Źródło:
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Expert review of pharmacoeconomics & outcomes research [Expert Rev Pharmacoecon Outcomes Res] 2021 Oct; Vol. 21 (5), pp. 967-973. Date of Electronic Publication: 2021 Mar 24.
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Typ publikacji:
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Comparative Study; Journal Article
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Język:
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English
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Imprint Name(s):
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Publication: 2015- : Abingdon, Oxford : Taylor & Francis
Original Publication: London : Future Drugs Ltd, [2001-
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MeSH Terms:
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Cost of Illness*
Amyloid Neuropathies, Familial/*therapy
Benzoxazoles/*administration & dosage
Oligonucleotides/*administration & dosage
RNA, Small Interfering/*administration & dosage
Amyloid Neuropathies, Familial/economics ; Benzoxazoles/economics ; Drug Costs/statistics & numerical data ; Health Care Costs/statistics & numerical data ; Hospitalization/economics ; Humans ; Oligonucleotides/economics ; RNA, Small Interfering/economics ; Spain
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Contributed Indexing:
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Keywords: Hereditary amyloidosis; onpattro; rare disease; tegsedi; treatment cost analysis; vyndaqel
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Substance Nomenclature:
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0 (Benzoxazoles)
0 (Oligonucleotides)
0 (RNA, Small Interfering)
0IEO0F56LV (Inotersen)
50FKX8CB2Y (patisiran)
8FG9H9D31J (tafamidis)
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SCR Disease Name:
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Amyloidosis, Hereditary, Transthyretin-Related
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Entry Date(s):
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Date Created: 20210316 Date Completed: 20211006 Latest Revision: 20211006
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Update Code:
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20240104
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DOI:
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10.1080/14737167.2021.1900738
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PMID:
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33724140
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Background : Transthyretin amyloid polyneuropathy (ATTR-PN) is a fatal disease associated with substantial burden of illness. Three therapies are approved by the European Medicines Agency for the management of this rare disease. The aim of this study was to compare the total annual treatment specific cost per-patient associated with ATTR-PN in Spain. Methods : An Excel-based patient burden and cost estimator tool was developed to itemize direct and indirect costs related to treatment with inotersen, patisiran, and tafamidis in the context of ATTR-PN. The product labels and feedback from five Spanish ATTR-PN experts were used to inform resource use and cost inputs. Results : Marked differences in costs were observed between the three therapies. The need for patisiran- and inotersen-treated patients to visit hospitals for pre-treatment, administration, and monitoring was associated with increased patient burden and costs compared to those treated with tafamidis. Drug acquisition costs per-patient per-year were 291,076€ (inotersen), 427,250€ (patisiran) and 129,737€ (tafamidis) and accounted for the majority of total costs. Overall, the total annual per-patient costs were lowest for patients treated with tafamidis (137,954€), followed by inotersen (308,358€), and patisiran (458,771€). Conclusions : Treating patients with tafamidis leads to substantially lower costs and patient burden than with inotersen or patisiran.