Risk management for biological products

International Journal of Pharmaceutical Medicine

Volumn 18, Issue 3, 2004, Pages 149-157

  Nieminen, Outi ^a,c   Kurki, Pekka ^b  

^a AALTO UNIVERSITY   (Finland)

^b NATIONAL AGENCY FOR MEDICINES   (Finland)

^c Lab of Biochem and Microbiology   (Finland)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL PRODUCT; GENE PRODUCT; PLASMA DERIVED MEDICINAL PRODUCT; RECOMBINANT DNA; RECOMBINANT DNA PRODUCT; UNCLASSIFIED DRUG; VACCINE; VIRUS VECTOR;

BLOOD; CELL TRANSPLANTATION; CONSULTATION; DISEASE TRANSMISSION; DRUG SURVEILLANCE PROGRAM; EPIDEMIOLOGY; ETHICS; EUROPEAN UNION; EXPERT SYSTEM; GENE THERAPY; GENE TRANSFER; GOVERNMENT REGULATION; HEALTH HAZARD; HUMAN; HYBRIDOMA; IMMUNOGENICITY; INDUSTRY; INFORMATION DISSEMINATION; LAW; LEGAL ASPECT; LICENSING; LOCAL REACTION; NONHUMAN; PRACTICE GUIDELINE; PRIORITY JOURNAL; PUBLIC HEALTH; RECOMBINANT DNA TECHNOLOGY; REPLICATION COMPETENT VIRUS; REVIEW; RISK BENEFIT ANALYSIS; RISK COMMUNICATION; RISK MANAGEMENT; RISK REDUCTION; SAFETY; SIDE EFFECT; TISSUE ENGINEERING; VACCINATION; VIRUS RECOMBINANT; VIRUS REPLICATION; XENOGENEIC CELL; XENOTRANSPLANTATION;

EID: 9244265484     PISSN: 13649027     EISSN: None     Source Type: Journal    
DOI: 10.1097/00124363-200418030-00003     Document Type: Review

References (63)

1. Seligman PJ. Risk management: the FDA role [online]. Available from URL: http://www.fda.gov/cder/Offices/OPaSS/rmspeech/sld001.htm [Accessed 2003 Oct 7]
2. Draft consensus guideline - pharmacovigilance planning (PvP)-E2E, ICH steering committee, November 2003 [online]. Available from URL: http://www.ich.org [Accessed 2004 Sep 15]
3. Ball R. Post-marketing risk assessment and management in biologic products: 2003 DIA annual meeting [online]. Available from URL: http://www.fda.gov/ cber/summaries/dia061603rb.htm [Accessed 2003 Oct 7]
4. Sims L. Assessment of biotechnology products for therapeutic use. Toxicol Lett 2001; 120: 59-66
5. Jacobson RM, Adegbenro A, Pankratz VS, et al. Adverse events and vaccination: the lack of power and predictability of infrequent events in pre-licensure study. Vaccine 2001; 19: 2428-33
6. Keller-Stanislawski B. Feasibility of improving safety beyond certain limits in clinical trials. Vaccine 2002; 20 Suppl.: 45-6
7. Vaccine Adverse Event Reporting System (VAERS) [online]. Available from URL: http://www.fda.gov/cber/vaers/vaers.htm [Accessed 2004 Jan 12]
8. Surveillance for safety after immunization: Vaccine Adverse Event Reporting System (VAERS): United States, 1991-2001 [online]. Available from URL: http://www.cdc.gov/mmwr/preview/mmwrhtml/ss5201al.htm [Accessed 2004 Jan 12]
9. Heijbel H, Jefferson T. Vaccine safety: improving monitoring. Vaccine 2001; 19: 2457-60
10. Mullooly J, Drew L, DeStefano F, et al. Quality of HMO vaccination data bases used to monitor childhood vaccine safety: Vaccine Safety Data Link Team. Am J Epidemiol 1999; 149: 186-94
11. Poland GA, Jacobson RM. Understanding those who do not understand: a brief review of the anti-vaccine movement. Vaccine 2001; 19: 2440-5
12. Mäkelä A, Nuorti JP, Peltola H. Neurologic disorders after measles-mumps-rubella vaccination. Pediatrics 2002; 110 (5): 957-63
13. DeStefano F, Gu D, Kramarz P, et al. Childhood vaccinations and risk of asthma. Pediatric Infect Dis J 2002; 21: 498-504
14. Anderson RM, Donnelly CA, Gupta S. Vaccine design, evaluation, and community-based use for antigenically variable infectious agents. Lancet 1997; 350: 1466-70
15. Dittmann S. Vaccine safety: risk communication: a global perspective. Vaccine 2001; 19: 2446-56
16. Guertler LG. Virus safety of human blood, plasma, and derived products. Thromb Res 2002; 107: S39-45
17. Burckhardt JJ. Assessment of needs for plasma for fractionation in Europe. Biologicals 1999; 27: 337-41
18. Taylor DM. Inactivation of TSE agents: safety of blood and blood-derived products. Transfus Clin Biol 2003; 10: 23-5
19. Anderson JF, Andrealis TG, Vossbrinck CR, et al. Isolation of West Nile virus from mosquitoes, crows, and a cooper's hawk in Connecticut. Science 1999 Dec; 286: 2331-3
20. Hoots WK, Abrams C, Tankersleydagger D. The impact of Creutzfeldt-Jakob disease and variant Creutzfeldt-Jakob disease on plasma safety. Transfus Med Rev 2001 Apr; 15 (2 Suppl. 1): 45-59
21. EMEA/CPMP/BWP/2879/02, CPMP position statement on Creutzfeldt-Jakob disease and plasma-derived and urine-derived medicinal products [online]. Available from URL: http://www.emea.eu.int/pdfs/human/press/pos/ 287902en.pdf [Accessed 2004 Aug 18]
22. Pignatti F, Aronsson B, Gate N, et al. The review of drug applications submitted to the European Medicines Evaluation Agency: frequently raised objections, and outcome. Eur J Clin Pharmacol 2002; 58: 573-80
23. Feldmann M, Maini RN. Anti-TNFα therapy of rheumatoid arthritis: what have we learned? Annu Rev Immunol 2001; 19: 163-96
24. Green JD, Tsang L, Cavagnaro JA. 'Generic' or 'follow-on' biologics: scientific considerations and safety issues. Expert Opin Biol Ther 2003; 3 (7): 1019-22
25. Baert F, Noman M, Vermeire S, et al. Influence of immunogenicity on the long term efficacy of infliximab in Crohn's disease. N Engl J Med 2003; 348: 601-8
26. Casadevall N. Pure red cell aplasia and anti-erythropoietin antibodies in patients treated with epoetin. Nephrol Dial Transplant 2003 Nov; 18 Suppl. 8: viii37-41
27. Louët S. Lessons from Eprex for biogeneric firms. Nat Biotechnol 2003 Sep; 21 (9): 956-7
28. Guideline on comparability of medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues. EMEA/CPMP/3097/02/Final [online]. Available from URL: http:// www.emea.eu.int/ pdfs/human/ewp/309702en.pdf [Accessed 2004 Jan 12]
29. Mire-Sluis A. Specific criteria for the acceptability of immunogenicity assays. In: Building a framework for risk management in monitoring immunogenicity of biotechnology-derived therapeutic products, post-workshop conference; 2003 Oct 2; Paris
30. Crommelin DJA, Storm G, Verrijk R, et al. Shifting paradigms: biopharmaceuticals versus low molecular weight drugs. Int J Pharm 2003; 266: 3-16
31. Tsao A. Seeking a prescription for biogenerics. Business Week Online 2003 Oct 24 [online]. Available from URL: http://www.businessweek.com/technology/ content/oct2003/tc20031024_4053_tc074.htm [Accessed 2004 Aug 18]
32. Ragni MV. Safe passage: a plea for safety in hemophilia gene therapy. Mol Ther 2002 Oct; 6 (4): 436-40
33. EMEA/22880/03 report from the ad hoc meeting of CPMP Gene Therapy Expert Group 26-27 June 2003 [online]. Available from URL: http://www.emea.eu.int/ index/indexh1.htm [Accessed 2004 Aug 18]
34. Biological Response Modifiers Advisory Committee [online]. Available at URL: http://www.fda.gov/cber/advisory/brm/brmchart.htm [Accessed 2004 Feb 11]
35. Fox JL. Gene therapy safety issues come to fore. Nat Biotechnol 1999 Dec; 17: 1153
36. Gould DJ, Favorov P. Vectors for the treatment of autoimmune disease. Gene Ther 2003; 10: 912-27
37. Boissier M-C, Bessis N. Risks and benefits of articular gene therapy. Joint Bone Spine 2003; 70: 486-8
38. Connolly JB. Lentiviruses in gene therapy clinical research. Gene Ther 2002; 9: 1730-4
39. Brenner S, Malech HL. Current developments in the design of onco-retrovirus and lentivirus vector systems for hematopoietic cell gene therapy. Biochim Biophys Acta 2003; 1640: 1-24
40. Gene therapy patient tracking system [online]. Available from URL: http://www.fda.gov/cber/genetherapy/gttrack.htm [Accessed 2004 Jan 15]
41. Heads of Agencies Working Group. Establishing a European risk management strategy [online]. Available from URL: http://heads.medagencies.org/heads/ docs/summary.pdf [Accessed 2004 Jan 15]
42. Gee AP. The impact of regulatory policy on the development of somatic cell therapies in the United States. Transpl Immunol 2002; 9: 295-300
43. Tissue [online]. Available from URL: http://www.fda.gov/cber/tiss.htm. [Accessed 2004 Jun 15]
44. Heinonen M. Kudosteknologiatuotteiden tuotekehitys ja regulaatio [thesis]. Espoo: Helsinki University of Technology, 2003
45. Kleijwegt T. Regulation of tissue engineered medical products in member states of the European Union [thesis]. Utrecht: Utrecht University, 2003
46. Wassenaar C, Geertsman RE, Kallewaard M. Tissue engineered medical products (TEMPs): a preclude to risk management. Bilthoven: RIVM Report, 2001
47. Guidance on applications for products comprised of living autologous cells manipulated ex vivo and intended for structural repair or reconstruction, CBER, FDA, 1996 [online]. Available from URL: http://www.fda.gov/cber/gdlns/ GDEXV.TXT [Accessed 2004 Aug 18]
48. Guidance for the submission of chemistry, manufacturing and controls information and establishment description for autologous somatic cell therapy products, CBER, FDA, 1997 [online]. Available from URL: http://www.fda.gov/ cber/gdlns/xvcmc.txt [Accessed 2004 Aug 18]
49. Suitability determination for donors of human cellular and tissue-based products. Federal Register: 21 CFR parts 210, 211, 820, and 1271 [online]. Available from URL: http://www.fda.gov/cber/rules/suitdonor.pdf [Accessed 2004 Sep 15]
50. Guidance for industry: preventive measures to reduce the possible risk of transmission of Creutzfeldt-Jakob Disease (CJD) and variant Creutzfeldt-Jakob Disease (vCJD) by human cells, tissues, and cellular and tissue, and cellular and tissue-based products (HCT/Ps), Federal Register [online]. Available from URL: http://www.fda.gov/OHRMS/DOCKETS/98fr/062502d.pdf [Accessed 2004 Aug 18]
51. Barker JH, Polcrack L. Respect for persons, informed consent and the assessment of infectious disease risks in xenotransplantation. Med Health Care Philos 2001; 4: 53-70
52. Brown J, Matthews AL, Sandstrom, et al. Xenotransplantation and the risk of retroviral zoonosis. Trends Microbiol 1998 Oct; 6 (10): 411-5
53. Cooper DKC. Clinical xenotransplantation: how close are we? Lancet 2003; 362: 557-9
54. Gunzburg WH, Salmons B. Xenotransplantation: is the risk of viral infection as great as we thought? Mol Med Today 2000 May; 6: 199-208
55. Xenotransplantation action plan [online]. Available from URL: http://www.fda.gov/ cber/xap/comp.htm [Accessed 2004 Jan 14]
56. Guidance for industry: source animal, product, preclinical, and clinical issues concerning the use of xenotransplantation products in humans [online]. Available from URL: http://www.fda.gov/cber/gdlns/clinxeno.pdf [Accessed 2004 Aug 18]
57. Guidance for industry: public health issues posed by the use of nonhuman primate xenografts in humans [online]. Available from URL: http://www.fda.gov/ cber/gdlns/xenoprim.pdf [Accessed 2004 Aug 18]
58. PHS guideline on infectious diseases issues in xenotransplantation, 2001 [online]. Available from URL: http://www.fda.gov/cber/gdlns/xenophs0101.pdf [Accessed 2004 Aug 18]
59. Points to consider on xenogeneic cell therapy medicinal product, CPMP/1199/02, Dec 2003 [online]. Available from URL: http://www.emea.eu.int/ pdfs/human/regaffair/119902en.pdf [Accessed 2004 Aug 18]
60. Directive 2002/98/EC of the European Parliament and of the Council of 27 January 2003 setting standards of quality and safety for the collection, testing, processing, storage and distribution of human blood and blood components and amending Directive 2001/83/EC [online]. Available from URL: http://europa.eu.int/ eur-lex/pri/en/oj/dat/2003/1_033/1_03320030208en00300040. pdf. [Accessed 2004 Aug 18]
61. Common Position (EC) No 50/2003 of 22 July 2003 adopted by the Council, acting in accordance with the procedure referred to in Article 251 of the Treaty establishing the European Community, with a view to adopting a directive of the European Parliament and of the Council on setting standards of quality and safety for the donation, procurement, testing, processing, preservation, storage and distribution of human tissues and cells [online]. Available from URL: http://europa.eu.int/ smartapi/cgi/sga_doc?. smartapi!celexapi!prod!CELEXnumdoc&lg=en&num doc= 52003AG0050&model= guichett [Accessed 2004 Aug 18]
62. Biological product deviation reporting [online]. Available from URL: http:// www.fda.gov/cber/biodev/biodev.htm [Accessed 2004 Jan 15]
63. Commission of the European Communities. Communication from the Commission on the precautionary principle, February 2000 [online]. Available from URL: http://europa.eu.int/comm/dgs/health_consumer/library/pub/pub07_en.pdf [Accessed 2004 Sep 17]