Overseeing innovative therapy without mistaking it for research: A function-based model based on old truths, new capacities, and lessons from stem cells

Journal of Law, Medicine and Ethics

Volumn 38, Issue 2, 2010, Pages 286-302

  Taylor, Patrick L ^a  

^a BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL TRIAL (TOPIC); COMPASSIONATE USE; CONFERENCE PAPER; CONFLICT OF INTEREST; ETHICS; FOOD AND DRUG ADMINISTRATION; GOVERNMENT REGULATION; HUMAN; HUMAN EXPERIMENT; LEGAL ASPECT; MASS COMMUNICATION; NONBIOLOGICAL MODEL; OFF LABEL DRUG USE; ORGANIZATION; ORGANIZATION AND MANAGEMENT; PRACTICE GUIDELINE; PROFESSIONAL STANDARD; SAFETY; STEM CELL TRANSPLANTATION; UNITED STATES;

CLINICAL TRIALS AS TOPIC; COMPASSIONATE USE TRIALS; CONFLICT OF INTEREST; DIFFUSION OF INNOVATION; ETHICS COMMITTEES, RESEARCH; GOVERNMENT REGULATION; GUIDELINES AS TOPIC; HUMAN EXPERIMENTATION; HUMANS; MODELS, ORGANIZATIONAL; OFF-LABEL USE; ORGANIZATIONAL OBJECTIVES; SAFETY; STEM CELL TRANSPLANTATION; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 77954918986     PISSN: 10731105     EISSN: 1748720X     Source Type: Journal    
DOI: 10.1111/j.1748-720X.2010.00489.x     Document Type: Conference Paper

References (37)

1. Fins J F. " Surgical Innovation and Ethical Dilemmas: Precautions and Proximity," Cleveland Clinic Journal of Medicine 2008, 75(Supp. 6):S7-S12.
2. Curley M A Q, Hickey P A. " The Nightingale Metrics," American Journal of Nursing 2006, 106(no. 10):1-5.
3. Ethical Principles and Guidelines for the Protection of Human Subjects of Research (Belmont Report) September 30, 1978, The National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, DHEW Publication OS78-0012, Washington, D.C.
4. Guidelines for the Clinical Translation of Stem Cells 2008, Task Force for the Clinical Translation of Stem Cells, International Society for Stem Cell Research, Chicago
5. Hyun I. " Allowing Innovative Stem Cell-Based Therapies Outside of Clinical Trials: Ethical and Policy Challenges," Journal of Law, Medicine & Ethics 2010, 38(no. 2).
6. " Gene Therapy for Cancer Clinical Trials: Questions and Answers," http://www.cancer.gov/cancertopics/factsheet/Therapy/Gene, National Cancer Institute, , last visited April 2, 2010
7. Bamford K B, Wood S, Shaw R J. " Standards for Gene Therapy Clinical Trials Based on Pro-active Risk Assessment in a London NHS Teaching Hospital Trust," QJM 2005, 98(no. 2):75-86.
8. Freedman B. " Equipoise and the Ethics of Clinical Research," New England Journal of Medicine 1987, 317(no. 3):141-145. T. L. Beauchamp and J. F. Childress, (New York: Oxford University Press, 2001): at 322-323.
9. http://www.emea.europa.eu/pdfs/human/ich/013595en.pdf,http://ohsr.od.nih.gov/guidelines/helsinki.html, 21 C.F.R. Parts 50, 56, 312, 314; 600, 812; 45 C.F.R. Part 46; International Conference on Harmonization (ICH) for Good Clinical Practice (GCP), July 2002,, last visited April 2, 2010); World Medical Association Declaration of Helsinki, , revised October 2008,, last visited April 8, 2010
10. " Accreditation Standards and Principles," http://www.aahrpp.org/www.aspx?PageID=21, Association for the Accreditation of Human Research Protection Programs, , last visited April 2, 2010); S. M. Wolf, F. P. Lawrenz, C. A. Nelson, J. P Kahn, M. K. Cho, E. W. Clayton, J. G. Fletcher, M. K. Georgieff, D. Hammerschmidt, K. Hudson, J. Illes, V. Kapur, M. A. Keane, B. A. Koenig, B. S. LeRoy, E. G. McFarland, J. Paradise, L. S. Parker, S. F. Terry, B. Van Ness, and B. S. Wilfond, " Managing Incidental Findings in Human Subject Research: Analysis and Recommendations," 36, no. 2 (2008): 219-248
11. " Accreditation Requirements," http://www.jointcommission.org/Standards/, Joint Commission on the Accreditation of Healthcare Organizations, revised 2009 as of March 26, 2009,, last visited April 2, 2010
12. Robertson J. " Legal Implications of the Boundaries between Biomedical Research involving Human Subjects and the Accepted or Routine Practice of Medicine," December 31, 1975, in the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, , at Appendix B, DHEW Publication OS78-0012, Washington, D.C., September 30, 1978
13. Levine R. " The Boundaries between Biomedical or Behavioral Research and the Accepted or Routine Practice of Medicine - Addendum," September 25, 1975, in the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research, , Appendix A, Section 1, DHEW Publication OS78-0012, Washington, D.C., September 30, 1978
14. at 1-43
15. Emanuel E J, Wendler D, Grady C. " What Makes Clinical Research Ethical?" JAMA 2000, 283(no. 2):2701-2711.
16. Capron A M. " Containing Health Care Costs: Ethical and Legal Implications of Changes in the Methods of Paying Physicians," Case Western Reserve Law Review 1986, 36(no. 4):708-77. N. Fost, " Ethical Dilemmas in Medical Innovation," 22, no. 3 (1998): 223-232.
17. Angell M. " Medicine: The Endangered Patient-Centered Ethic," Hastings Center Report 1987, 17(no. 1):S12-S13.
18. 530 U.S. 211 (2000)
19. Lo B, Wolf L E, Berkeley A. " Conflict-of-Interest Policies for Investigators in Clinical Trials," New England Journal of Medicine 2000, 343(no. 22):1616-1620. M. Angell, " Is Academic Medicine for Sale?" 342, no. 20 (2000): 1516-1518; C. D. DeAngelis and P. B. Fontanarose, " Impugning the Integrity of Medical Science: The Adverse Effects of Industry Influence," 299, no. 15 (2008): 1833-1835; Committee on Conflict of Interest in Medical Research, Education and Practice, Institute of Medicine, (Washington, D.C.: National Academies Press. 2009).
20. 35 U.S.C. 200-212, implementing regulations: 37 C.F.R. Part 401
21. Armstrong D. " Delicate Operation - How a Famed Hospital Invests In Device It Uses and Promotes. Cleveland Clinic Set Up Fund That Has Stock in Maker of Heart-Surgery System: Dr. Cosgrove's Multiple Roles,": Wall Street Journal. December 12, 2005, A1. at
22. AAHRPP Accredits 67 Organizations in 2008; Up 42.6 Percent over Last Year, News Release. December 2008, http://www.aahrpp.org/www.aspx?PageID=270, Association for the Accreditation of Human Research Protection Programs, available at, last visited April 2, 2010
23. Responsible Research: A Systems Approach to Protecting Research Participants 2002, Committee on Assessing the System for Protecting Human Research Participants of the Institute of Medicine of the National Academies of Medicine, Washington, D.C., National Academies Press
24. Emanuel E J, Wood A, Fleischman A, Bowen A, Getz K A, Grady C. " Oversight of Human Participants Research: Identifying Problems to Evaluate Reform Proposals," Annals of Internal Medicine 2004, 141(no. 4):282-291.
25. DeMets DL, Fost N, Powers M. " An Institutional Review Board Dilemma: Responsible for Safety Monitoring but Not in Control," Clinical Trials 2006, 3(no. 2):142-148.
26. Bankert E, Amdur R. " The IRB Is Not a Data Safety Monitoring Board," IRB: Ethics in Human Research 2000, 22(no. 6):9-11.
27. Ladd R E. " Roles and Responsibilities of Ethics Committees," American Journal of Bioethics 2009, 9(no. 4):41-42.
28. Pearson S D, Sabin J E, Emanuel E J. No Margin, No Mission - Health Care Organizations and the Quest for Ethical Excellence 2003, 25-44. New York, Oxford University Press, at, P. J. Boyle, E. R. DuBose, S. J. Ellington, D. E. Guinn, and D. B. McCurdy, (San Francisco: John Wiley & Sons, Inc., 2001): at 23-28, 56-57, 73-74
29. Guidelines for Human Embryonic Stem Cell Research 2005, Committee on Guidelines for Human Embryonic Stem Cell Research, National Research Council, Institute of Medicine, Washington, D.C., National Academies Press, Human Embryonic Stem Cell Research Task Force, International Society for Stem Cell Research, , Chicago, 2006
30. To Err Is Human 2000, Committee on Quality of Health Care in America, Institute of Medicine, Washington, D.C., National Academy Press, Committee on Quality of Health Care in America, Institute of Medicine, (Washington, D.C.: National Academy Press, 2001
31. Crossing the Quality Chasm 2001, 76. Committee on Quality of Health Care in America, Institute of Medicine, Washington, D.C., National Academy Press, at
32. Lee T H, Torchiana D F, Lock J E. " Is Zero the Ideal Death Rate?" New England Journal of Medicine 2007, 357(no. 2):111-113.
33. " Medicare Clinical Trial Policies - Overview," http://www.cms.hhs.gov/ClinicalTrialPolicies/,http://www.cancer.gov/clinicaltrials/developments/laws-about-clinical-trial-costs, last visited April 13, 2010); National Cancer Institute, " States that Require Health Plans to Cover Patient Care Costs in clinical Trials," , last visited April 13, 2010
34. McKinney's Consolidated Laws of New York, Insurance Law, Article 49, Title II; McKinney's Consolidated Laws of New York, Public Health Law, Article 49, Title II.
35. 45 C.F.R Part 46 (2009); 45 C.F.R 46.111 (" Criteria for IRB approval of research" ).
36. Porter ME, Teisberg EO. Redefining Health Care - Creating Value-Based Competition on Results 2006, See, e.g., Boston, Harvard Business School Press
37. Christensen C M, Grossman J H, Hwang J. The Innovator's Prescription - A Disruptive Solution for Health Care 2009, New York, McGraw Hill