Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa

Orphan drugs definition should be related to prevalence criteria. In Europe the prevalence criterion is 5/10.000. These drugs are called “orphans” because the pharmaceutical industry has little interest under normal market conditions in developing and marketing products intended for only a small num...

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Main Authors: Orietta Zaniolo, Mario Eandi, Pier Luigi Zinzani
Format: Article
Language:English
Published: SEEd Medical Publishers 2004-09-01
Series:Farmeconomia: Health Economics and Therapeutic Pathways
Online Access:https://journals.seedmedicalpublishers.com/index.php/FE/article/view/793
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author Orietta Zaniolo
Mario Eandi
Pier Luigi Zinzani
author_facet Orietta Zaniolo
Mario Eandi
Pier Luigi Zinzani
author_sort Orietta Zaniolo
collection DOAJ
description Orphan drugs definition should be related to prevalence criteria. In Europe the prevalence criterion is 5/10.000. These drugs are called “orphans” because the pharmaceutical industry has little interest under normal market conditions in developing and marketing products intended for only a small number of patients. For this reason governments have emphasized the need for economic incentives to encourage drug companies to develop and market orphan drugs. Aim of this study is the analysis of the contributing factors involved in the price definition of orphan drugs in Italy, focusing on the case of DepoCyte®, a new orphan drug recently approved by the European Medicines Agency. DepoCyte® is a slow-release formulation of cytarabine designed for intrathecal administration in the treatment of neoplastic meningitis due to metastatic cancers. It maintains cytotoxic concentrations of free cytarabine in the cerebrospinal fluid for more than 14 days following a single injection. In two randomized clinical trials DepoCyte® was compared to standard formulation of cytarabine, showing a better time to neurologic progression and survival trend in favor of DepoCyte®, associated with an improved mean change about quality of life in Karnofsky performance score. The innovative technology and the efficacy of DepoCyte® allow to frame some interesting pharmacoeconomical consequences: the results of the present work showed that DepoCyte® is more expensive but also more effective than standard formulation, and the new formulation-correlated improvement in the patients’ quality of life seems to justify the difference between the costs of the two alternatives.
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spelling doaj.art-272e2e63eaa745d3b77216d7b3cd41ce2022-12-21T18:22:03ZengSEEd Medical PublishersFarmeconomia: Health Economics and Therapeutic Pathways2240-256X2004-09-015314115010.7175/fe.v5i3.793738Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosaOrietta Zaniolo0Mario Eandi1Pier Luigi Zinzani2Farmacologia clinica, Università di Torino
Farmacologia clinica, Università di Torino
Istituto di Ematologia e Oncologia Medica “L. e A. Seragnoli”, Università di Bologna

Orphan drugs definition should be related to prevalence criteria. In Europe the prevalence criterion is 5/10.000. These drugs are called “orphans” because the pharmaceutical industry has little interest under normal market conditions in developing and marketing products intended for only a small number of patients. For this reason governments have emphasized the need for economic incentives to encourage drug companies to develop and market orphan drugs. Aim of this study is the analysis of the contributing factors involved in the price definition of orphan drugs in Italy, focusing on the case of DepoCyte®, a new orphan drug recently approved by the European Medicines Agency. DepoCyte® is a slow-release formulation of cytarabine designed for intrathecal administration in the treatment of neoplastic meningitis due to metastatic cancers. It maintains cytotoxic concentrations of free cytarabine in the cerebrospinal fluid for more than 14 days following a single injection. In two randomized clinical trials DepoCyte® was compared to standard formulation of cytarabine, showing a better time to neurologic progression and survival trend in favor of DepoCyte®, associated with an improved mean change about quality of life in Karnofsky performance score. The innovative technology and the efficacy of DepoCyte® allow to frame some interesting pharmacoeconomical consequences: the results of the present work showed that DepoCyte® is more expensive but also more effective than standard formulation, and the new formulation-correlated improvement in the patients’ quality of life seems to justify the difference between the costs of the two alternatives.https://journals.seedmedicalpublishers.com/index.php/FE/article/view/793
spellingShingle Orietta Zaniolo
Mario Eandi
Pier Luigi Zinzani
Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
Farmeconomia: Health Economics and Therapeutic Pathways
title Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
title_full Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
title_fullStr Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
title_full_unstemmed Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
title_short Il prezzo dei farmaci orfani in Italia: il caso di Citarabina Depot (DepoCyte®) nella meningite linfomatosa
title_sort il prezzo dei farmaci orfani in italia il caso di citarabina depot depocyte r nella meningite linfomatosa
url https://journals.seedmedicalpublishers.com/index.php/FE/article/view/793
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AT marioeandi ilprezzodeifarmaciorfaniinitaliailcasodicitarabinadepotdepocytenellameningitelinfomatosa
AT pierluigizinzani ilprezzodeifarmaciorfaniinitaliailcasodicitarabinadepotdepocytenellameningitelinfomatosa