Plasma zur Fraktionierung(PzF) in Europa

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1 Plasma zur Fraktionierung(PzF) in Europa Helmi Storch Haemo- Consult D Brotterode-Trusetal nach einem Vortrag ECA-Seminar April 2013, Vienna, Austria ARGE Plasmapherese, Magdeburg 23.November 2013 European Compliance Academy (ECA) 1

2 PzF in Europa Übersicht Konzept Europäische Selbst Versorgung (SS) Driving Faktoren für Plasma Proteine (PP) SS in Europe mit PzF und PP Plasmapherese(PPH) der Schlüssel zur SS Zusammenfassung Ausblick European Compliance Academy (ECA) 2

3 Konzept der Europäischen SS EU Council entscheidet sich für ein Konzept European Self Sufficiency Programme for blood products * Gründe: Plasma Defizit seit Jahrzehnten, Abhängigkeit von Importen von Source Plasma aus den USA. PzF in Europa Die epidemiologische Situation kann einen kurzzeitigen Wechsel der Orte der Plasmagewinnung bedingen (HIV in den USA in den 80ern) * Council Directive ,Official Journal of the European Communities (89/381/EEC) European Compliance Academy (ECA) 3

4 PzF in Europa Driving Faktoren. Albumin war der driver für PzF in den 70ern. Faktor VIII war der driver bis zum Ende der 90er. Seit Ende der 90er sind Immunoglobuline (IVIG/SCIG) die driver European Compliance Academy (ECA) 4

5 PzF in Europa Driving Faktoren Globaler Verbrauch von IVIG/SCIG ,41 Tonnen ,74 Tonnen ,82 Tonnen ,89 Tonnen seit 2010 = 7% globales Wachstum /Jahr European Compliance Academy (ECA) 5

6 PfF in Europe European Compliance Academy (ECA) 6

7 PzF in Europa Driving Faktoren Verbrauch von IVIG/SCIG in Deutschland* Kg Kg Kg seit % Wachstum *PEI- Berichte entsprechend 21 TFG European Compliance Academy (ECA) 7

8 PzF in Europa Selbstversorgung (SS). Unzureichende Daten zur Bereitstellung und Verbrauch von PP in Europa (einige Länder haben nationale Daten). Letzte Datenbasis: Council of Europe,2013 veröffentlicht EDQM Reports 2009/2010.The collection,testing and Use Blood and Blood Components in Europe.*. European Commission beauftragte Creativ Ceutical im Mai 2012 eine EU Übersicht des Markts von Blut, Blutkomponenten und Plasmaderivaten zur Verfügbarkeit für Patienten zu erstellen.(keine Daten derzeit verfügbar) *Not yet published. Acknowledgment: I am grateful to M.P.Janssen,C.L.van der Poel and M.-E.Behr-Gross,EDQM, who provided data for inclusion in my presentation. European Compliance Academy (ECA) 8

9 PzF in Europa SS (1)* nach 4 Jahren. There is still a significant deficit in the European plasma supply esp. in southern and eastern Europe. Health authorities believe that SS is an attainable goal within 5-10 years. Representatives of the plasma industry do not believe that SS can be attained without commercial plasmacenters and compensated donors * Piet J. Hagen, Blood transfusion in Europe: a white paper,council of Europe Press, 1993 European Compliance Academy (ECA) 9

10 PzF in Europa SS (2)* in 2000(nach 11Jahren) Belgium 200,000 L 19.63/1,000 inhabitants France 524,172 L 8.87/1,000 inhabitants Germany 1,555,000 L 18.94/1,000 inhabitants Italy 456,000 L 8.04/1,000 inhabitants Netherlands 233,000 L 14.67/1,000 inhabitants Spain 250,000 L 6.37/1,000 inhabitants Sweden 234,000 L 26.17/1,000 inhabitants UK and Portugal zero 59,247,400/9,902,100 population * Patrick Robert (2002).Personal communication. Blood Collection and Transfusion in 13 European Countries. The Marketing Research Bureau Inc.,284 Racebrook Road Orange,CT 06477,USA European Compliance Academy (ECA) 10

11 PzF in Europa SS (3)* in 2008(nach 19 Jahren) Belgium 163,089 L 15.53/1,000 inhabitants <as 2000 France 769,931 L 12.04/1,000 inhabitants >as 2000 Germany 2,526,637 L 30.81/1,000 inhabitants >as 2000 Italy 648,000 L 10.80/1,000 inhabitants >as 2000 Netherlands 225,557 L 13.67/1,000 inhabitants <as 2000 Poland 46,582 L 1.22/1,000 inhabitants n.d.before Sweden 113,419 L 12.25/1,000 inhabitants <as 2000 Greece 15,402 L 1.47/1,000 inhabitants n.d.before * EDQM,2008 Report The Collection,Testing an Use of Blood and Blood Components in Europe.Table 4. Issued 2011 European Compliance Academy (ECA) 11

12 PzF in Europa SS 2001, 2008* and 2010(4)** L/ /1,000 Inhabitants Belgium > 15.53* <16.16** 65% RP** France 7.78 < 12.04* <13.14** 74% RP** Finland > 12.87*<14.58** 91% RP** Italy 7.95 < 10.80*<12.04** 65% RP** Netherlands > 13.67*<20.34** 44% RP** Spain 5.56 < 7.29*< 8.29** n.d.available Sweden > 12.25*> 11.42** n.d.available Germany < 30.81*< 35.30** 37% RP** Czech Republic 6.17 < 24.01*< 52.38** 10% RP** * EDQM,2008 Report The Collection,Testing and Use of Blood and Blood Components in Europe Table4 issued 2011 ** EDQM,2010 Report. Table 4.1.Not yet published. See acknowledgment slide 8 European Compliance Academy (ECA) 12

13 PzF in Europa SS IVIG (5)* in 2008 Belgium 97.1g/1,000 inhabitants SS=47% France 79.3g/1,000 inhabitants SS=45% Finland 70.4g/1,000 inhabitants SS=54% Italy 52.5g/1,000 inhabitants SS=61% Netherlands 57.8g/1,000 inhabitants SS=71% Spain 52.7g/1,000 inhabitants no data available Sweden 93.8g/1,000 inhabitants SS=39% UK 46,1g/1,000 inhabitants SS= zero * Patrick Robert.The Marketing Research Bureau Inc.,284 Racebrook Road Orange,CT 06477,USA European Compliance Academy (ECA) 13

14 PzF in Europa SS mit IVIG/SCIG (6)in 2010*/2012** Population PfF IVIG/SCIG) PfF required Degree of SS Million Litres (000) kg Litres (000) Austria % Czech Rep % France ,020 4,323 1,441 71% Germany ,600 2, % Italy ,015 1,005 69% Switzerland % Spain , % Sweden % Germany ,075 5,321 1, %** * Patrick Robert. MRB. Dublin, January ** PEI, Bericht 21 TFG für 2012 vom European Compliance Academy (ECA) 14

15 PzF in Europa SS in Italien in 2011 * Albumin 601 kg/1 Mio Inhabitants SS=55% highest albumin consumption among the mostly industrialized countries IVIG 3,570,542 g= need of 892,635 L PfF SCIG 112,749 g= need of 28,187 L PfF SS=81% Need of PfF for Immunoglobulins= 920,822 L Kedrion with the monopoly as contract fractionator processed 747,982 L Deficit of 172,840 L *Report on the demand for the major blood-derived medicinal products in Italy , Instituto Superiore de Sanita European Compliance Academy (ECA) 15

16 PzF in Europa Rolle der Plasmapherese (Österreich) Plasmapherese startete Land in Europa April, 1975 Plasmapherese Gesetz 1.Land in Europa 1993 wurde die IG Plasma gegründet 2012 erzeugten IG Plasma Mitglieder L SP in18 PZ von Spenden = 786 ml/spende A distinguished national contribution of PfF in Europe European Compliance Academy (ECA) 16

17 PzF in Europa Rolle der Plasmapherese (Tschech. Republik)* Vor 2007:Plasmapherese BTS Spenden/Jahr 2007 erstes kommerzielles PZ in Zlín 2011 kommerzielle PZ produzieren L SP = 33,14 L/ EW 2011 BTS produzieren L SP 2011 Tschech. Republik produziert L PzF (18,8 % RP, 81,2 % SP) An engaged national contribution of PfF in Europe * UZIS. Statistical Bureau for Health Care of the Czech Republic. Activity of health care institutions in the section of transfusion service in the Czech Republic in 2011 European Compliance Academy (ECA) 17

18 PzF in Europa Rolle der Plasmapherese (Deutschland) Plasmapherese startete in den 70ern,Mannheim staatlich initiiertes Plasmapherese Programm 1997 Gründung ARGE Plasmapherese zu dieser Zeit wurden L SP produziert 2012 produzierten 21 Mitglieder L PzF in 93 PZ An engaged national contribution of PfF in Europe European Compliance Academy (ECA) 18

19 PzF in Europa PzF durch Plasmapherese in Europa 2010* Germany 54 % Czech Republic 14 % Austria 11 % Rest of Europe 21 % * Source: Patrick Robert, MRB European Compliance Academy (ECA) 19

20 Plasma zur Fraktionierung in Deutschland Gewinnung von Plasma zur Fraktionierung 3,0 2,5 2,0 1,691 1,833 1,820 1,861 Liter in Mio. 1,5 0,738 0,979 1,493 0,968 0,838 1,053 1,259 1,483 0,585 1,0 0,5 0,892 0,947 0,986 0,916 0,964 0,916 1,090 0,973 1,015 1,208 1,055 1,178 1,214 0, aus Vollblut aus Apherese European Compliance Academy (ECA) 20

21 Struktur u. Leistung der ARGE 21 Mitglieder erzeugten 2012 in 93 PZ mittels Plasmapherese Liter PzF und Liter z. direkten therapeutischen Anwendung (FFP) das sind L/PZ European Compliance Academy (ECA) 21

22 Plasmaaufkommen in Deutschland European Compliance Academy (ECA) 22

23 Plasmaaufkommen in Deutschland European Compliance Academy (ECA) 23

24 Wissenschaftliche Aktivität ARGE 1998 Stellungnahme(S3) AK-Blut pro SIPLA 2004 Publikation:45 L/Jahr, IgG- Messung, keine Immundefekte, kardiovaskuläres o.fe- Defizitrisiko* 2005 Publikation: Drop outs hpts. sozioökonomisch** 2006 Publikation :850 ml/spende KG> 70kg und 60 Spenden/Jahr bei sorgfältigem Monitoring*** 2013 SIPLA II: Sichere, intensive Plasmapherese auch bei Neuspendern**** Tagungsteile PP DGTI 2005 Erfurt,2012 Graz,2014 Dresden *P.Hellstern et al. Vox Sanguinis (2004)86, **P.Hellstern et al.vox Sanguinis (2005)88m ***P.Hellstern et al. Vox Sanguinis(2006)91, ****S.T.Kiessig et al. ISBT,Amsterdam European Compliance Academy (ECA) 24

25 Zusammenfassung In D wurden zwischen ,4-38,2 L PzF pro EW gespendet In EU A und CZ vergleichbar- Mehrzahl der Mitglieder defizitär zur SS ARGE PPH erzeugte 2012 mit L 98,5% des PPH- Plasma u. 59,6% des gesamten PzF in D PPH ist unverzichtbar bei weltweit steigendem Bedarf an PzF für Plasmaprodukte (Ig driving Faktor) Die Aufwandsentschädigung ist eine adäquate Kompensation f. Spender, die einen wertvollen Beitrag z. Gesundheit von Patienten mit seltenen Erkrankungen leisten Die EU sollte erhebliche Anstrengungen zur Steigerung des PzF durch PPH unternehmen und die Akzeptanz des in einem kontrolliertem, regulatorischen Umfeld in der EU erzeugten PzF als globalen Standard, gleichwertig mit US-Plasma erreichen European Compliance Academy (ECA) 25

26 Outlook PfF in Europe Problems with plasmapheresis in Europa Compensated donors Not enough hyperimmune plasma Efficiency compared to US-Plasma Need of scientific data* about yield, frequency and hemovigilance * T.Schulzki et al. A prospective multicentre study on the safety of long-term intensive plasmapheresis in donors(sipla) Vox Sanguinis(2006),91: ** St.Kießig et al. First results from the study on intensive plasmapheresis (SIPLA II) submitted and accepted 23.Regional Congress of ISBT,Amsterdam,June 2-5,2013 European Compliance Academy (ECA) 26

27 Outlook PfF in Europe The role of plasmapheresis in Europe Assumption: Plasmapheresis is the solution to get enough starting material (PfF)in order to meet the clinical need of PP for patients In European countries with a controlled, regulatory environment and EMA accepted epidemiological data national contributions are needed for SP obtained by plasmapheresis European Compliance Academy (ECA) 27

28 Plasma for Fractionation IVIG IS NOT APPROVED FOR ALZHEIMER'S DISEASE TREATMEN Volume of Plasma collected - Apheresis + Recovered Liters x 1,000 Region Asia & Oceania 4,585 4,736 4,993 5,371 Latin America North America 14,825 18,018 18,714 20,087 Middle East/Africa Europe 6,382 6,938 7,172 7,521 World 26,547 30,477 31,697 33,831 European Compliance Academy (ECA) 28

29 Outlook PfF in Europe Is European Self Sufficiency of PfF achievable?* If plasmapheresis will be extended in countries with a regulatory environment based on EU-directives and EMA accepted epidemiological conditions If UK will accept PfF sourced in Europe If the compensated donation will not be restricted If the effciency of plasmapheresis will be increased in order to be competitive with SP procured from US *excluded hyperimmune plasma European Compliance Academy (ECA) 29

30 Outlook PfF in Europe Is European Self Sufficiency of PfF achievable also for hyperimmune plasma? YES If diagnostic measures and immunization programmes will be established in EU countries with developed plasmapheresis NO If the compensated donation will be restricted If the problems of immunization e. g. Anti-D can not be solved European Compliance Academy (ECA) 30

31 Outlook PfF in Europe Is European Self Sufficiency of PP and PfF a reasonable goal in a gobalized world? Yes we can - if we take the necessary steps! European Compliance Academy (ECA) 31

32 Welcome to Trusetal in Thuringia Welcome in Trusetal European Compliance Academy (ECA) 32

33 European Compliance Academy (ECA) 33