Ethical considerations in clinical investigation: Exploring relevance in haemophilia research

Haemophilia

Volumn 14, Issue SUPPL. 3, 2008, Pages 122-129

  Dimichele, D M ^a  

^a Weill Cornell Cancer Center   (United States)

Author keywords

Developing world; Gene transfer; Haemophilia; Research ethics

Indexed keywords

ADULT; AGE; ARTICLE; CHILD; CLINICAL PRACTICE; CLINICAL RESEARCH; DEVELOPING COUNTRY; EVIDENCE BASED PRACTICE; GENE REPLACEMENT THERAPY; GENE TECHNOLOGY; GENE TRANSFER; HEMOPHILIA; HUMAN; INFORMED CONSENT; METHODOLOGY; PATIENT CARE; PRIORITY JOURNAL; RESEARCH ETHICS; RESEARCH SUBJECT; RISK REDUCTION;

BIOETHICAL ISSUES; BIOMEDICAL RESEARCH; CHILD; EVIDENCE-BASED MEDICINE; GENE THERAPY; GENE TRANSFER TECHNIQUES; HEMOPHILIA A; HUMAN EXPERIMENTATION; HUMANS; INFORMED CONSENT; PATIENT SELECTION; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 44249126461     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2008.01738.x     Document Type: Article

References (45)

1. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
2. Konkle BA, Ebbesen LS, Erhardtsen E et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 5: 1904-13.
3. Astermark J, Donfield SM, DiMichele DM et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: The FEIBA NovoSeven Comparative (FENOC) Study. Blood 2007; 109: 546-51.
4. DiMichele DM, Hay CR. The international immune tolerance study: A multicenter prospective randomized trial in progress. J Thromb Haemost 2006; 4: 2271-3.
5. Mannucci PM, Abshire T, DiMichele D, Santagostino E, Blanchette V. Inhibitor development, immune tolerance and prophylaxis in haemophilia A - the need for an evidence-based approach. Haemophilia 2006; 12: 429-34.
6. MASAC Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). Available at: http://www.hemophilia.org (last accessed on 31 March 2008). National Hemophilia Foundation MASAC document #179.
7. Miller FG, Brody H. A critique of clinical equipoise. Therapeutic misconception in the ethics of clinical trials. Hastings Cent Rep 2003; 33: 19-28.
8. Gifford F. Community equipoise and the ethics of randomized clinical trials. Bioethics 1995; 9: 127-48.
9. Appelbaum PS, Roth LH, Lidz CW, Benson P, Winslade W. False hopes and best data: Consent to research and the therapeutic misconception. Hastings Cent Rep 1987; 17: 20-4.
10. Appelbaum PS. Clarifying the ethics of clinical research: A path toward avoiding the therapeutic misconception. Am J Bioeth 2002; 2: 22-3.
11. Levine RJ. Ethics and Regulation of Clinical Research, 2nd edn. New Haven: Yale University Press, 1986; 8-10.
12. Emanuel EJ, Wendler D, Grady C. What makes clinical research ethical? JAMA 2000; 283: 2701-11.
13. Saenko EL, Ananyeva NM, Shima M, Hauser CA, Pipe SW. The future of recombinant coagulation factors. J Thromb Haemost 2003; 5: 922-30.
14. Pipe SW. Coagulation factors with improved properties for hemophilia gene therapy. Semin Thromb Hemost 2004; 2: 227-37.
15. Murphy SL, High KA. Gene therapy for haemophilia. Br J Haematol 2008; 140: 479-87.
16. Skinner MW, Lillicrap DP, McMillan J, Castro Ozelo M, Pierce GF. What is a cure and how do we get there? Haemophilia 2004; 10 (Suppl. 4): 115-8.
17. DiMichele D, Miller FG, Fins JJ. Gene therapy ethics and haemophilia: An inevitable therapeutic future? Haemophilia 2003; 9: 145-52.
18. DiMichele DM. Management of factor VIII inhibitors. Int J Hematol 2006; 83: 119-25.
19. Kodish E. Informed consent for pediatric research: Is it really possible? J Pediatr 2003; 142: 89-90.
20. McCullough LB, Coverdale JH, Chervenak FA. Preventative ethics for including women of childbearing potential in clinical trials. Am J Obstet Gynecol 2006; 194: 1221-7.
21. Kodish E, ed. Ethics and research with children an introduction. Ethics and Research with Children. Oxford University Press, 2005: 9-10.
22. Kodish E, ed. Ethics and research with children an introduction. Ethics and Research with Children. Oxford University Press, 2005: 12-6.
23. Bartholome WG. Informed consent, parental permission, and assent in pediatric practice. Pediatrics 1995; 96: 981-2.
24. Jaffe A, Prasad SA, Larcher V, Hart S. Gene therapy for children with cystic fibrosis - who has the right to choose? J Med Ethics 2006; 32: 361-4.
25. Kodish E, ed. Ethics and research with children an introduction. Ethics and Research with Children. Oxford University Press, 2005: 6.
26. Deichmann A, Hacein-Bey-Abina S, Schmidt M et al. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 2007; 117: 2225-32.
27. Li H, Murphy SL, Giles-Davis W et al. Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. Mol Ther 2007; 15: 792-800.
28. High KA. Update on progress and hurdles in novel genetic therapies for hemophilia. Am Soc Hematol Educ Program Book 2007: 466-72.
29. Lillicrap D, VandenDriessche T, High K. Cellular and genetic therapies for haemophilia. Haemophilia 2006; 12 (Suppl. 3): 36-41.
30. Ponder KP. Gene therapy for hemophilia. Curr Opin Hematol 2006; 13: 301-7.
31. Kimmelman J. Recent developments in gene transfer: Risk and ethics. BMJ 2005; 330: 79-82.
32. U.S. Department of Health and Human Services FDA Center for Drug Evaluation and Research; U.S. Department of Health and Human Services FDA Center for Biologics Evaluation and Research; U.S. Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industryU Patient-reported outcome measures: use in medical product development to support labeling claims: Draft guidance. Health Qual Life Outcomes 2006; 11: 4-79.
33. DiMichele D, Chuansumrit A, London AJ et al. Ethical issues in haemophilia. Haemophilia 2006; 12 (Suppl. 3): 30-5.
34. Darby SC, Keeling DM, Spooner RJ et al. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost 2004; 2: 1047-54.
35. Ashrani AA, Reding MR, Shet A et al. Successful liver transplantation in a patient with severe haemophilia A and a high-titre factor VIII inhibitor. Haemophilia 2004; 10: 735-7.
36. Nuffield Council on Bioethics. The Ethics of Research Related to Healthcare in Developing Countries, 2002. Available at: http://www.nuffieldbioethics.org (last accessed on 26 February 2008).
37. WHO. International Ethical Guidelines for Biomedical Research Involving Human Subjects The Council for International Organizations of Medical Sciences, WHO Meeting Geneva, 2002. Available at: http://www.cioms.ch (last accessed on 31 March 2008).
38. Emanuel EJ, Wendler D, Killen J, Grady C. What makes clinical research in developing countries ethical? The benchmarks of ethical research. J Infect Dis 2004; 189: 930-7.
39. Wendler D. The standard of care debate: Can research in developing countries be both ethical and responsive to those countries'health needs? Am J Public Health 2004; 94: 923.
40. Pace CA, Emanuel EJ. The ethics of research in developing countries: assessing voluntariness. Lancet 2005; 365: 11-2.
41. Diallo DA, Doumbo OK, Plowe CV et al. Community permission for medical research in developing countries. Clic Infect Dis 2005; 41: 255-9.
42. Fitzgerald DW. Comprehension during informed consent in a less-developed country. Lancet 2002; 360: 1301.
43. Srivastava A, You SK, Ayob Y et al. Hemophilia Treatment in Developing Countries: Products and Protocols. Semin Thromb Hemost 2005; 31: 495-500.
44. Mathews V, Viswabandya A, Baidya S et al. Surgery for hemophilia in developing countries. Semin Thromb Haemost 2005; 31: 538-43.
45. Chandy M. Management of hemophilia with minimal factor replacement in developing countries: Role of ancillary therapy. Semin Thromb Hemost 2005; 31: 501-6.