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ISTITUTO SUPERIORE DI SANITÀ Italian Blood System 2017: demand for plasma-derived medicinal products. Volume 2 Fabio Candura (a), Maria Lavinia Salvatori (a), Gabriele Calizzani (a), Samantha Profili (a), Cristiana Chelucci (a), Chiara Brutti (b), Claudia Biffoli (b), Giancarlo Maria Liumbruno (a) (a) Centro Nazionale Sangue, Istituto Superiore di Sanità, Roma (b) Direzione Generale del Sistema Informativo, Ministero della Salute, Roma ISSN: 1123-3117 (cartaceo) • 2384-8936 (online) Rapporti ISTISAN 20/13 IstitutoSuperiore di Sanità Italian Blood System 2017: demand for plasma-derived medicinal products. Volume 2. Fabio Candura, Maria Lavinia Salvatori, Gabriele Calizzani, Samantha Profili, Cristiana Chelucci, Chiara Brutti, Claudia Biffoli, Giancarlo Maria Liumbruno 2020, vii, 121 p. Rapporti ISTISAN 20/13 With the aim of fulfilling the task assigned it pursuant to national regulations regarding coordinating and providing technical support to the planning of self-sufficiency in blood components and plasma-derived medicinal products at regional and national level, the Italian National Blood Centre has conducted an analysis in collaboration with the Information and Statistics Department of the Italian Health Ministry. The analysis of the demand for plasma-derived medicinal products and recombinant therapies included the assessment of the self-sufficiency levels achieved and the costs sustained by the Italian National Health Service for the provision of these products. The content of this document, an update of the data for the year 2016 published in the Rapporto ISTISAN 19/12, was obtained by conducting a comparative analysis of the available data sources. The document is also an invaluable tool for planning self-sufficiency at national level. Key words: Plasma-derived medicinal products; Demand; Self-sufficiency; Expenditure Istituto Superiore di Sanità Sistema trasfusionale italiano 2017: analisi della domanda dei medicinali plasmaderivati. Volume 2. Fabio Candura, Maria Lavinia Salvatori, Gabriele Calizzani, Samantha Profili, Cristiana Chelucci, Chiara Brutti, Claudia Biffoli, Giancarlo Maria Liumbruno 2020, vii, 121 p. Rapporti ISTISAN 20/13 (in inglese) Al fine di adempiere ai compiti ad esso assegnati dalla normativa vigente in materia di coordinamento e supporto tecnico alla programmazione dell’autosufficienza regionale e nazionale di emocomponenti e medicinali plasmaderivati, il Centro Nazionale Sangue ha effettuato, in collaborazione con l’Ufficio IV della Direzione Generale del Sistema Informativo e Statistico Sanitario del Ministero della Salute, l’analisi della domanda dei prodotti medicinali plasmaderivati e delle alternative terapeutiche di natura ricombinante, le valutazioni dei livelli di autosufficienza regionale e nazionale e la stima della spesa farmaceutica a carico del Servizio Sanitario Nazionale. Il confronto delle diverse fonti dati disponibili ha consentito l’elaborazione del presente documento che riporta l’aggiornamento relativo all’anno 2016 dei dati sull’argomento pubblicati nel Rapporto ISTISAN 19/12 e che si configura come uno strumento fondamentale per la programmazione dell’autosufficienza nazionale. Parole chiave: Medicinali plasmaderivati; Domanda; Autosufficienza; Spesa Si ringraziano per il contributo al presente documento: Giovanna Antonini, Annalisa Di Vito, Pierluigi Russo (Area Strategia e Politiche del Farmaco, Agenzia Italiana del Farmaco), Odile Tchangmena Befeuka, Concettina Oliva, Domenico Di Giorgio (Ufficio Qualità dei Prodotti e Contrasto al Crimine Farmaceutico, Agenzia Italiana del Farmaco); Lorella Lombardozzi, Alessandra Mecozzi (Direzione Regionale Salute e Integrazione Sociosanitaria - Area Politica del Farmaco, Regione Lazio); Ilaria Mazzetti (Servizio Assistenza Territoriale - Area Farmaco e Dispositivi Medici, Direzione Generale Sanità e Politiche Sociali e per l’Integrazione, Regione Emilia-Romagna); Livia Cannata, Liviana Catalano, Anna Ceccarelli (Centro Nazionale Sangue, Istituto Superiore di Sanità); le Aziende: Kedrion SpA, Baxalta SpA, Pfizer Italia, Grifols Italia SpA, CSL Behring SpA, Takeda Italia SpA, Biotest Italia Srl, Bayer SpA, Octapharma Italy SpA, Novo Nordisk A/S. Per informazioni su questo documento scrivere a: [email protected] Il rapporto è accessibile online dal sito di questo Istituto: www.iss.it Citare questo documento come segue: Candura F, Salvatori ML, Calizzani G, Profili S, Chelucci C, Brutti C, Biffoli C, Liumbruno GM. Italian Blood System 2017: demand for plasma-derived medicinal products. Volume 2. Roma: Istituto Superiore di Sanità; 2020. (Rapporti ISTISAN 20/13). Legale rappresentante dell’Istituto Superiore di Sanità: Silvio Brusaferro Registro della Stampa - Tribunale di Roma n. 114 (cartaceo) e n. 115 (online) del 16 maggio 2014 Direttore responsabile della serie: Paola De Castro Redazione: Sandra Salinetti e Manuela Zazzara La responsabilità dei dati scientifici e tecnici è dei singoli autori, che dichiarano di non avere conflitti di interesse. © Istituto Superiore di Sanità 2020 viale Regina Elena, 299 – 00161 Roma Rapporti ISTISAN 20/13 TABLE OF CONTENTS Acronyms and abbreviations ......................................................................................................... .v Presentation ......................................................................................................................................... vii Introduction ............................................................................................................................................ 1 Sources and methodology ............................................................................................................... 3 Data sources ......................................................................................................................................... 3 Drug traceability flow .................................................................................................................... 3 Information flow of accredited pharmacies .................................................................................... 4 Information flow of the direct provision of medicinal products ..................................................... 4 Information flow of medicines consumed in hospitals ................................................................... 6 Data on plasma-derived medicinal products produced from Italian plasma ................................... 6 Data on plasma-derived medicinal products subject to import procedures .................................... 7 Data processing and the ATC drug classification system .................................................................... 7 Active ingredients and measurement units ........................................................................................... 8 Self-sufficiency and pharmaceutical expenditure ................................................................................ 9 PART A Plasma-derived medicinal products from toll fractionation Albumin (ATC B05AA01) ............................................................................................................. 13 Quantification and characterisation of the demand ...................................................................... 14 Normal human immunoglobulins for subcutaneous use (ATC J06BA01) and for intravenous use (ATC J06BA02) ................................................................................. 17 Quantification and characterisation of the demand ...................................................................... 19 Normal human immunoglobulins for subcutaneous use ......................................................... 20 Normal human immunoglobulins for intravenous use ............................................................ 22 Antithrombin (ATC B01AB02) .................................................................................................... 25 Quantification and characterisation of the demand ...................................................................... 25 Coagulation factor VIII (ATC B02BD02), coagulation factor VIII and von Willebrand factor in combination (ATC B02BD06), von Willebrand factor (ATC B02BD10) and Recombinant factor VIII (ATC B02BD02) .......................................... 28 Quantification and characterisation of demand ............................................................................ 31 Plasma-derived Factor VIII .................................................................................................... 33 Recombinant Factor VIII ........................................................................................................ 35 Coagulation factor IX (ATC B02BD04), Recombinant coagulation factor IX (ATC B02BD09) .............................................................................................................................. 38 Quantification and characterisation of the demand ...................................................................... 39 Plasma-derived Factor IX ....................................................................................................... 41 Recombinant Factor IX ........................................................................................................... 43 3-Factor Prothrombin Complex Concentrates (ATC B02BD) and 4-Factor Prothrombin Complex Concentrates (ATC B02BD01) ......................................................... 46 Quantification and characterisation of the demand .....................................................................
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