Guide for the Assessment of Clotting Factor Concentrates

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Some measures which should be included in a program are:• Donors should be selected from low-risk populations. This can only be done using thekind of epidemiological data that can be obtained using tools such as the plasma masterfile model (see page 15).Conclusion• Once collected, plasma should be quarantined until the donor has been recalled and re-testedfor markers of infection. All such testing should be done with the most sensitive tests possible.If the donor does not return for re-testing the plasma should not be used. Re-testingshould be done using intervals which allow donors to seroconvert for the transfusiontransmittedviruses in case they were infected at the time of donation. The quarantine periodshould be related to the window period of infection for the relevant virus. This willdepend on whether NAT screening is used.• Testing for viruses using molecular technology should be introduced. Using NAT, theviral window period for the important viruses can be substantially decreased. This measureprobably has minimal effect on the safety of plasma derivatives in developed countriesbecause of the effect of viral inactivation; however, its use on products, such as cryo,which cannot undergo such inactivation would enhance safety considerably.• Producers and authorities may be able to further enhance the safety and quality of productthrough other means. For example, optimization of factor VIII yields may allow somelevel of viral inactivation to be achieved, since the loss of yield due to viral inactivationmay be tolerated if production yields are high. Pools of dedicated plasma donors, carefullyselected and repeatedly tested, can in time become a source of very safe raw material,compared to first-time donors. The pharmacological stimulation of donors to producemore factor VIII may be worth considering as a means of improving yields.The production of safe cryoprecipitate is a feasible option for countries without access to plasmaconcentrates. It requires a high level of commitment both in the generation of safe raw material and inthe technical production of the product. It should be pointed out that the safety features outlined inthis chapter require policies and investment which would benefit all aspects of blood safety. This levelof investment will increase the cost of cryoprecipitate. The technical expertise gained in optimizing cryoproduction should also be viewed as a long-term benefit for when the country’s economic developmentmakes plasma fractionation more feasibleSUMMARY• Small pool freeze-dried cryoprecipitate is a viable option for the treatment of hemophiliaA for countries lacking access to fractionation facilities.• Careful attention to technique can allow sufficient product to be generated to deal withmost everyday treatment requirements.• Safety of the product cannot be assured through viral inactivation; therefore, donor selectionand screening measures are all the more important.30 Guide for the Assessment of Clotting Factor Concentrates
CONCLUSIONChoosing appropriate products for the treatment of hemophilia is not an easy task. It depends on theresources and unique circumstances of each country. However, the principles and information givenhere can provide guidance to regulatory authorities when making decisions about the purchase ofhemophilia treatment products.The WFH updates this guide regularly, and welcomes comments for improving it.Please send any suggestions to:World Federation of Hemophilia1425 Rene-Levesque Boulevard West, Suite 1010Montreal, Quebec H3G 1T7CanadaTel.: (514) 875-7944Fax: (514) 875-8916E-mail: wfh@wfh.orgWeb: www.wfh.orgGuide for the Assessment of Clotting Factor Concentrates 31
Page 1: GUIDE FOR THE ASSESSMENTOF CLOTTING
Page 5 and 6: INTRODUCTIONSelecting therapeutic p
Page 7 and 8: SECTION 1FACTORS AFFECTING THE QUAL
Page 9 and 10: and authorities need to assess each
Page 11 and 12: There are a number of different vir
Page 13 and 14: Treatment Advantages Points to cons
Page 15: measures may have only limited bene
Page 18 and 19: • FDA draft guidelines• FDA ins
Page 20 and 21: Common strengths of U.S. & EU regul
Page 22 and 23: • Audit potential suppliers who m
Page 24 and 25: on HCV reactivity in product sample
Page 26 and 27: Basic requirementsThere are a numbe
Page 28 and 29: treatment, and therefore samples sh
Page 30 and 31: EvaluationSome of the consideration
Page 32 and 33: Figure 2: Production of cryoprecipi
Page 37 and 38: APPENDIX 1REGISTRY OF CLOTTING FACT
Page 39 and 40: TABLE 1A: SEROLOGIC TESTS ON INDIVI
Page 41 and 42: TABLE 2. FACTOR VIII CONCENTRATES M
Page 43 and 44: BRAND COMPANY SITE OF MANU-FACTUREP
Page 45 and 46: TABLE 7: OTHER CLOTTING FACTOR CONC
Page 47 and 48: APPENDIX 2MODEL PRODUCT ASSESSMENT
Page 49: (D) STABILITY AND SHELF LIFEInclude
Page 52 and 53: Nucleic acid testing (NAT): Testing
Page 56: World Federation of Hemophilia1425

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