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Overseeing Innovative Therapy without Mistaking it for Research: A Function-Based Model Based on Old Truths, New Capacities, and Lessons from Stem Cells

Published online by Cambridge University Press:  01 January 2021

Patrick L. Taylor
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Extract

Innovative therapy is the name we give to novel medical interventions, radically different from the standard of care, provided in order to benefit a patient, rather than to acquire new knowledge. They are paradigmshifting, not incremental, responses to serious patient problems that standard medical care inadequately addresses. Innovative therapies are often devised by clinicians, not basic science researchers; they do not follow the linear model of basic research, to translation, to clinical research, to application. Instead, they come from thinking backwards from the patient’s circumstances, and forward from deep knowledge of how the body functions, to challenge the limits of current mechanisms for effecting cures.

Type
Symposium
Information
Journal of Law, Medicine & Ethics , Volume 38 , Issue 2 , Summer 2010 , pp. 286 - 302
Copyright
Copyright © American Society of Law, Medicine and Ethics 2010

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References

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