DOCSLIB.ORG

Coagulation Factor Viii

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Coagulation Factor Viii 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 .....................................................................
Load more

Recommended publications
  • Clinical Commissioning Policy: Susoctocog Alfa for Treating Bleeding Episodes in People with Acquired Haemophilia a (All Ages)
    Clinical Commissioning Policy: Susoctocog alfa for treating bleeding episodes in people with acquired haemophilia A (all ages) NHS England Reference: 170061P 1 NHS England INFORMATION READER BOX Directorate Medical Operations and Information Specialised Commissioning Nursing Trans. & Corp. Ops. Commissioning Strategy Finance Publications Gateway Reference: 07603 Document Purpose Policy Clinical commissioning policy: Susoctocog alfa for treating bleeding Document Name episodes in people with acquired haemophilia A (all ages) Author Specialised Commissioning Team Publication Date 29 June 2018 Target Audience CCG Clinical Leaders, Care Trust CEs, Foundation Trust CEs , Medical Directors, Directors of PH, Directors of Nursing, NHS England Regional Directors, NHS England Directors of Commissioning Operations, Directors of Finance, NHS Trust CEs Additional Circulation #VALUE! List Description Routinely Commissioned - NHS England will routinely commission this specialised treatment in accordance with the criteria described in this policy. Cross Reference 0 Superseded Docs 0 (if applicable) Action Required 0 Timing / Deadlines By 00 January 1900 (if applicable) Contact Details for [email protected] further information 0 0 0 0 0 0 Document Status This is a controlled document. Whilst this document may be printed, the electronic version posted on the intranet is the controlled copy. Any printed copies of this document are not controlled. As a controlled document, this document should not be saved onto local or network drives but should always be accessed from the intranet. 2 Standard Operating Procedure: Clinical Commissioning Policy: Susoctocog alfa for treating bleeding episodes in people with acquired haemophilia A (all ages) First published: June 2018 Prepared by the National Institute for Health and Care Excellence (NICE) Commissioning Support Programme Published by NHS England, in electronic format only.
    [Show full text]
  • Coagulation Factor IX (Recombinant) - Drugbank
    12/7/2017 Coagulation Factor IX (Recombinant) - DrugBank Coagulation Factor IX (Recombinant) Targets (7) Biointeractions (4) IDENTIFICATION Name Coagulation Factor IX (Recombinant) Accession Number DB00100 (BTD00038, BIOD00038) Type Biotech Groups Approved Biologic Classification Protein Based Therapies Blood factors Description Recombinant Coagulation Factor IX is a purified Factor IX glycoprotein produced by recombinant DNA technology. It has a primary amino acid sequence that is identical to the Ala148 allelic form of human factor IX, and has structural and functional characteristics similar to those of endogenous factor IX. It is not derived from human blood (unlike human Factor IX complex), and is instead produced by a genetically engineered Chinese hamster ovary (CHO) cell line that secretes recombinant Factor IX into cell medium that is then processed and purified for use as a pharmaceutical agent. Recombinant Factor IX is indicated for the control and prevention of bleeding episodes in adult and pediatric patients with congenital factor IX deficiency (Hemophilia B). Protein structure Protein chemical formula C2041H3136N558O641S25 Protein average weight 46548.2 Da https://www.drugbank.ca/drugs/DB00100 1/13 12/7/2017 Coagulation Factor IX (Recombinant) - DrugBank 46548.2 Da Sequences >DB00100 sequence YNSGKLEEFVQGNLERECMEEKCSFEEAREVFENTERTTEFWKQYVDGDQCESNPCLNGG SCKDDINSYECWCPFGFEGKNCELDVTCNIKNGRCEQFCKNSADNKVVCSCTEGYRLAEN QKSCEPAVPFPCGRVSVSQTSKLTRAEAVFPDVDYVNSTEAETILDNITQSTQSFNDFTR VVGGEDAKPGQFPWQVVLNGKVDAFCGGSIVNEKWIVTAAHCVETGVKITVVAGEHNIEE
    [Show full text]
  • Suomen Lääketilasto 2019
    SUOMEN LÄÄKETILASTO S LT FINNISH STATISTICS ON MEDICINES FSM 2019 Keskeisiä lukuja lääkkeiden myynnistä ja lääkekorvauksista vuonna 2019 Milj. € Muutos vuodesta 2018, % Lääkkeiden kokonaismyynti 3 460 5,2 avohoidon reseptilääkkeiden myynti (verollisin vähittäismyyntihinnoin) 2 284 4,4 avohoidon itsehoitolääkkeiden myynti (verollisin vähittäismyyntihinnoin) 357 0,8 sairaalamyynti (tukkuohjehinnoin) 818 9,9 Lääkkeistä maksetut korvaukset 1 551 6,2 peruskorvaukset 316 3,0 erityiskorvaukset 1 029 5,2 lisäkorvaukset 205 17,7 Key figures for medicine sales and their reimburssement in 2019 € million Change from 2018, % Total sales of pharmaceuticals 3,460 5.2 prescription medicines in outpatient care (at pharmacy prices with VAT) 2,284 4.4 OTC medicines in outpatient care (at pharmacy prices with VAT) 357 0.8 sales to hospitals (at wholesale prices) 818 9.9 Reimbursement of medicine costs 1,551 6.2 Basic Refunds 316 3.0 Special Refunds 1,029 5.2 Additional Refunds 205 17.7 Lähde: Fimean lääkemyyntirekisteri, Kelan sairausvakuutuskorvausten tilastointitiedosto. Source: Finnish Medicines Agency, Drug Sales Register; Register of Statistical Information on National Health Insurance General Benefit Payments. SUOMEN LÄÄKETILASTO FINNISH STATISTICS ON MEDICINES 2019 Lääkealan turvallisuus- ja kehittämiskeskus Fimea ja Kansaneläkelaitos Finnish Medicines Agency Fimea and Social Insurance Institution Helsinki 2020 LÄÄKEALAN TURVALLISUUS- KANSANELÄKELAITOS JA KEHITTÄMISKESKUS FIMEA FINNISH MEDICINES AGENCY FIMEA SOCIAL INSURANCE INSTITUTION Lääketurvallisuus Analytiikka- ja tilastoryhmä Pharmacovigilance Section for Analytics and Statistics Mannerheimintie 166 Nordenskiöldinkatu 12 P.O. Box 55 P.O. Box 450 FI-00034 Fimea FI-00056 Kela Finland Finland [email protected] [email protected] Puh. 029 522 3341 Puh. 020 634 11 Tel. +358 29 522 3341 Tel.
    [Show full text]
  • Kongressjournal Wmw Wiener Medizinische Wochenschrift
    W Band 8 / Heft 10 www.springer.at/wmw-skriptum ISSN Print 1613-3803 P. b. b. Verlagspostamt 1201 Wien Plus.Zeitung 07Z037513P SpringerMedizin.at/schwerpunkt/oegari-2011 10/11 skriptum Kongressjournal wmw Wiener Medizinische Wochenschrift 15. bis 17. September 2011, Wien Jahrestagung der Österreichischen Gesellschaft für Anästhesiologie, Reanimation und Intensivmedizin Austrian International Congress 2011 – AIC Wissenschaftliche Leitung: Prim. Univ.-Prof. Dr. Sibylle Kozek-Langenecker inhalt 10/11 Inhalt brief der herausgeberin Jahrestagung der 4 Willkommen in Wien! Österreichischen S. Kozek-Langenecker Gesellschaft für beiträge Anästhesiologie, 6 Sedierung durch Nicht-AnästhesistInnen Reanimation und Robert D. Fitzgerald, Wien Intensivmedizin 7 Gerinnungsmanagement bei traumatisch bedingter (ÖGARI) Massivblutung D. Fries, P. Innerhofer, P. Perger, M. Gütl, S. Heil, N. Hofmann, W. Kneifl, L. 15. bis 17. September Neuner, T. Pernerstorfer, G. Pfanner, H. Schöchl, B. Ziegler, C. Köblinger und 2011, Wien S. Kozek-Langenecker, ARGE für perioperative Gerinnung der ÖGARI 10 Erstversorgung von SHT W. Mauritz und A. Brazinova, Wien 11 Extrakorporaler Gasaustausch W. Oczenski und C. Hörmann, Wien–St. Pölten 16 „Coagulation Day 2010“: Thrombosemanagement beim kritisch Kranken E. Schaden, Wien 19 Koma: Differenzialdiagnose im Notfall Schloss Schönbrunn E. Schmutzhard, Innsbruck 20 Präklinische Intubation A. von Goedecke, Steyr 21 Perioperative Gerinnungsoptimierung P. Innerhofer, D. Fries und M. Mittermayr, Innsbruck 24 Ist Muskelrelaxation noch zeitgemäß? K. Khünl-Brady, Innsbruck 26 Impressum wmw skriptum © Springer-Verlag 10/2011 3 brief der herausgeberin Willkommen in Wien! Sehr geehrte Kolleginnen, sehr geehrte Kollegen! Kurzfristig wurde uns die Möglichkeit eröffnet, Beiträge vom diesjährigen Kongress der Österreichi- schen Gesellschaft für Anästhesiologie, Reanimation und Intensivmedizin (ÖGARI) mit diesem PRIM.
    [Show full text]
  • COMPARISON of the WHO ATC CLASSIFICATION & Ephmra/Intellus Worldwide ANATOMICAL CLASSIFICATION
    COMPARISON OF THE WHO ATC CLASSIFICATION & EphMRA/Intellus Worldwide ANATOMICAL CLASSIFICATION: VERSION June 2019 2 Comparison of the WHO ATC Classification and EphMRA / Intellus Worldwide Anatomical Classification The following booklet is designed to improve the understanding of the two classification systems. The development of the two systems had previously taken place separately. EphMRA and WHO are now working together to ensure that there is a convergence of the 2 systems rather than a divergence. In order to better understand the two classification systems, we should pay attention to the way in which substances/products are classified. WHO mainly classifies substances according to the therapeutic or pharmaceutical aspects and in one class only (particular formulations or strengths can be given separate codes, e.g. clonidine in C02A as antihypertensive agent, N02C as anti-migraine product and S01E as ophthalmic product). EphMRA classifies products, mainly according to their indications and use. Therefore, it is possible to find the same compound in several classes, depending on the product, e.g., NAPROXEN tablets can be classified in M1A (antirheumatic), N2B (analgesic) and G2C if indicated for gynaecological conditions only. The purposes of classification are also different: The main purpose of the WHO classification is for international drug utilisation research and for adverse drug reaction monitoring. This classification is recommended by the WHO for use in international drug utilisation research. The EphMRA/Intellus Worldwide classification has a primary objective to satisfy the marketing needs of the pharmaceutical companies. Therefore, a direct comparison is sometimes difficult due to the different nature and purpose of the two systems.
    [Show full text]
  • Innovationsreport 2019 Kurzfassung
    Innovationsreport 2019 Auswertungsergebnisse von Routinedaten der Techniker Krankenkasse aus den Jahren 2016 bis 2017 Herausgeber: Gerd Glaeske und Wolf‐Dieter Ludwig Erstellt mit freundlicher Unterstützung der Techniker Krankenkasse (TK) 2 Herausgeber Prof. Dr. Gerd Glaeske Prof. Dr. Wolf‐Dieter Ludwig Experten für ausgewählte Kapitel Prof. Dr. med. Manfred Anlauf, Bremerhaven Prof. Dr. med. Peter Berlit, Berlin Prof. Dr. med. Winfried V. Kern, Freiburg Prof. Dr. med. Joachim Labenz, Siegen Prof. Dr. med. Ulrich A. Müller, MSc, Jena Prof. Dr. med. Bruno Müller‐Oerlinghausen, Berlin Prof. Dr. med. Wolfgang Schramm, München Prof. Dr. med. Dieter Ukena, Bremen PD Dr. med. Tobias Weberschock, Frankfurt am Main Autoren der Wirkstoffkapitel Dr. Stanislava Dicheva‐Radev, Dörte Fuchs, Dr. Iris Hinneburg, Anja Lübs, André S. Morawetz, Lutz Muth, Saskia Ritter, Dr. Kristin Sauer unter Mitarbeit von Eleonora Durakovic, Friederike Höfel, Linda Jespersen, Linda Richter, Marle Wilhelm Anschrift: Universität Bremen, SOCIUM, Mary‐Somerville‐Str. 5, 28359 Bremen Aus Gründen der besseren Lesbarkeit wurde auf die Nennung beider geschlechtsspezifischer Formen verzichtet. Im Allgemeinen ist aber das jeweils andere Geschlecht ebenfalls gemeint. 3 4 Glossar ........................................................................................... 6 Vorwort zum Innovationsreport 2019 ........................................... 13 Zweites Vorwort zum Innovationsreport 2019 .............................. 15 1 Einleitung ...............................................................................
    [Show full text]
  • A Guide for People Living with Von Willebrand Disorder CONTENTS
    A guide for people living with von Willebrand disorder CONTENTS What is von Willebrand disorder (VWD)?................................... 3 Symptoms............................................................................................... 5 Types of VWD...................................................................................... 6 How do you get VWD?...................................................................... 7 VWD and blood clotting.................................................................... 11 Diagnosis................................................................................................. 13 Treatment............................................................................................... 15 Taking care of yourself or your child.............................................. 19 (Education, information, first aid/medical emergencies, medication to avoid) Living well with VWD......................................................................... 26 (Sport, travel, school, telling others, work) Special issues for women and girls.................................................. 33 Connecting with others..................................................................... 36 Can I live a normal life with von Willebrand disorder?............. 37 More information................................................................................. 38 2 WHAT IS VON WILLEBRAND DISORDER (VWD)? Von Willebrand disorder (VWD) is an inherited bleeding disorder. People with VWD have a problem with a protein
    [Show full text]
  • ER Guide to Bleeding Disorders
    Bleeding disorders ER guide to bleeding disorders 1 Table of contents 4 General Guidelines 4–5 national Hemophilia Foundation guidelines 5–10 Treatment options 10 HemopHilia a Name:__________________________________________________________________________________________________ 10–11 national Hemophilia Foundation guidelines Address:________________________________________________________________________________________________ 12 dosage chart Phone:__________________________________________________________________________________________________ 14–15 Treatment products 16 HemopHilia B In case of emergency, contact: ______________________________________________________________________________ 16 national Hemophilia Foundation guidelines Relation to patient:________________________________________________________________________________________ 17 dosage chart 18 Treatment products 19 HemopHilia a or B with inHiBiTors Diagnosis: Hemophilia A: Mild Moderate Severe 20 national Hemophilia Foundation guidelines Inhibitors Inhibitors Bethesda units (if known) ____________________________________ 21 Treatment products Hemophilia B: Mild Moderate Severe 22–23 Von willeBrand disease Inhibitors Inhibitors Bethesda units (if known) ____________________________________ 23–24 national Hemophilia Foundation guidelines von Willebrand disease: Type 1 Type 2 Type 3 Platelet type 25 Treatment products 27 Bibliography Preferred product:_________________________________________________________________________________________ Dose for life-threatening
    [Show full text]
  • BMJ Paediatrics Open Is Committed to Open Peer Review. As Part of This Commitment We Make the Peer Review History of Every Article We Publish Publicly Available
    bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from BMJ Paediatrics Open is committed to open peer review. As part of this commitment we make the peer review history of every article we publish publicly available. When an article is published we post the peer reviewers’ comments and the authors’ responses online. We also post the versions of the paper that were used during peer review. These are the versions that the peer review comments apply to. The versions of the paper that follow are the versions that were submitted during the peer review process. They are not the versions of record or the final published versions. They should not be cited or distributed as the published version of this manuscript. BMJ Paediatrics Open is an open access journal and the full, final, typeset and author-corrected version of record of the manuscript is available on our site with no access controls, subscription charges or pay- per-view fees (http://bmjpaedsopen.bmj.com). If you have any questions on BMJ Paediatrics Open’s open peer review process please email [email protected] http://bmjpaedsopen.bmj.com/ on September 25, 2021 by guest. Protected copyright. BMJ Paediatrics Open bmjpo: first published as 10.1136/bmjpo-2020-000880 on 30 December 2020. Downloaded from Confidential: For Review Only Does the EU’s Paediatric Regulation work for new medicines for children in Nordic countries? A retrospective database review. Journal: BMJ Paediatrics Open Manuscript ID bmjpo-2020-000880 Article Type: Original research Date Submitted by the 16-Sep-2020 Author: Complete List of Authors: Lepola , Pirkko ; Helsinki University Central Hospital, Children and Adolescents Wang, Siri; Norwegian Medicines Agency Tötterman, Ann Marie; Finnish Medicines Agency Fimea Gullberg, Ninna; Swedish Medicinal products Agency Moll Harboe, Kirstine ; Danish Medicines Agency Kimland, Elin; Swedish Medicinal Products Agency Keywords: Therapeutics, Pharmacology http://bmjpaedsopen.bmj.com/ on September 25, 2021 by guest.
    [Show full text]
  • Justification
    Justification to the Resolution of the Federal Joint Committee (G-BA) on an Amendment of the Pharmaceuticals Directive (AM ‑ RL): Annex XII – Resolutions on the Benefit Assessment of Medicinal Products with New Active Ingredients in Accordance with Section 35a SGB V Damoctocog alfa pegol From 20 June 2019 Contents 1. Legal basis ................................................................................................................ 2 2. Key points of the resolution ..................................................................................... 2 2.1 Additional benefit of the medicinal product in relation to the appropriate comparator therapy ..................................................................................................... 3 2.1.1 Approved therapeutic indication of damoctocog alfa pegol (Jivi®) in accordance with the summary of product characteristics ............................................ 3 2.1.2 Appropriate comparator therapy ................................................................... 3 2.1.3 Extent and probability of the additional benefit .............................................. 5 2.1.4 Summary of the assessment ........................................................................ 6 2.2 Number of patients or demarcation of patient groups eligible for treatment ...... 6 2.3 Requirements for a quality-assured application ................................................ 7 2.4 Treatment costs ..............................................................................................
    [Show full text]
  • Australian PI
    AUSTRALIAN PRODUCT INFORMATION OBIZUR® (susoctocog alfa) 1 NAME OF THE MEDICINE Susoctocog alfa (bhk). 2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each OBIZUR vial contains nominally 500 units (U) of susoctocog alfa, which is a B domain deleted recombinant derived antihaemophilic factor VIII (rpFVIII), porcine sequence. After reconstitution, OBIZUR contains nominally 500 U/mL susoctocog alfa. Each vial of OBIZUR is labelled with the actual rpFVIII activity expressed in units determined by a one-stage clotting assay, using a reference rpFVIII material calibrated against the World Health Organization (WHO) 8th International Standard for human factor VIII concentrates. The specific activity of OBIZUR is in the range of 11000 - 18000 Units per milligram of protein. The potency values of OBIZUR determined by the chromogenic assay vary and are approximately 20-50 % lower than those of the one-stage clotting assay. Susoctocog alfa is expressed in a genetically engineered baby hamster kidney (BHK) cell line and secreted into the cell culture medium, and the protein is purified using a series of chromatography and filtration steps. The production process includes two dedicated viral clearance steps - a solvent/detergent treatment step for viral inactivation and a nanofiltration step through a series of two 15-nm filters for removal of viruses. No additives of human or animal origin are used in the formulation of OBIZUR. Excipient(s) with known effect Each vial of OBIZUR contains maximally 4.4 mg (198 mM) sodium per mL of reconstituted solution (see Section 4.4 SPECIAL WARNINGS AND PRECAUTIONS FOR USE). For the full list of excipients, see Section 6.1 LIST OF EXCIPIENTS.
    [Show full text]
  • Novoeight HCP Brochure
    ® READY UpUp to to Novoeight is ready to go TO GO when your patients are forfor up upto to ADMINIS- A recombinant factor VIII for today’s generation TRATION months 1 3 months of people living with hemophilia A Please see Prescribing Information for complete storage conditions. EFFICACY AND SAFETY Dylan lives with hemophilia A. PRODUCT ATTRIBUTES SUPPORT Indications and Usage • Novoeight® (antihemophilic factor, recombinant) is indicated for use in adults and children with hemophilia A for on-demand treatment and control of bleeding episodes, perioperative management, and routine prophylaxis to reduce the frequency of bleeding episodes • Novoeight® is not indicated for the treatment of von Willebrand disease SUMMARY Important Safety Information Contraindications • Do not use in patients who have had life-threatening hypersensitivity reactions, including anaphylaxis, to Novoeight® or its components, including hamster proteins ISI PI Please tap the ISI tab for additional Important Safety Information and tap the PI tab for Prescribing Information. Blerim lives with hemophilia A. READY WITH NOVOEIGHT® TO GO YOUR PATIENTS ARE ADMINIS- ready to go TRATION EFFICACY AND SAFETY “I’m planning on working as a camp counselor this summer. PRODUCT ATTRIBUTES Having a factor I can store and take with me is a huge relief.” —Patient with hemophilia A SUPPORT Important Safety Information SUMMARY Warnings and Precautions • Anaphylaxis and severe hypersensitivity reactions are possible. Patients may develop hypersensitivity to hamster proteins, which are present in trace amounts in the product. Should symptoms occur, discontinue Novoeight® and administer appropriate treatment ISI PI Please tap the ISI tab for additional Important Safety Information and tap the PI tab for Prescribing Information.
    [Show full text]