The Structure of Clinical Translation: Efficiency, Information, and Ethics

Hastings Center Report

Volumn 45, Issue 2, 2015, Pages 27-39

  Kimmelman, Jonathan ^a   London, Alex John ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; INDOLE DERIVATIVE; NEW DRUG; PYRROLE DERIVATIVE; SUNITINIB;

BIOETHICS; CLINICAL TRIAL (TOPIC); DRUG APPROVAL; ETHICS; HUMAN; INFORMATION DISSEMINATION; METHODOLOGY; OFF LABEL DRUG USE; ORGANIZATION AND MANAGEMENT; PROCEDURES; STANDARDS; THEORETICAL MODEL; TRANSLATIONAL RESEARCH; TRENDS;

ANTINEOPLASTIC AGENTS; BIOETHICAL ISSUES; CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; DRUGS, INVESTIGATIONAL; HUMANS; INDOLES; INFORMATION DISSEMINATION; MODELS, THEORETICAL; OFF-LABEL USE; PYRROLES; RESEARCH DESIGN; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84923861103     PISSN: 00930334     EISSN: 1552146X     Source Type: Journal    
DOI: 10.1002/hast.433     Document Type: Article

References (51)

1. The Lancet, "What Is the Purpose of Medical Research?, " The Lancet 381 (2013): 347.
2. A. Von Eschenbach and R. Hall, "Von Eschenbach and Hall: FDA Approvals Are a Matter of Life and Death, " Wall Street Journal, June 17, 2012.
3. A. Von Eschenbach. "Medical Innovation: How the U.S. Can Retain Its Lead, " Wall Street Journal, February 14, 2012.
4. P. D. Jacobson and W. E. Parmet, "A New Era of Unapproved Drugs: The Case of Abigail Alliance v. Von Eschenbach, " Journal of the American Medical Association 297 (2007): 205-08.
5. A. Grove, " Rethinking Clinical Trials, " Science Magazine 333 (2011): 1679.
6. D. D. Rosa, J. Harris, and G. C. Jayson, " The Best Guess Approach to Phase I Trial Design, " Journal of Clinical Oncology 24, no. 1 (2006): 206-08.
7. I. Chalmers et al., "How to Increase Value and Reduce Waste When Research Priorities Are Set, " The Lancet 383 (2014): 156-65.
8. F. S. Collins, "Reengineering Translational Science: The Time Is Right, " Science Translational Medicine 3, no. 90 (2011): 90cm17.
9. National Center for Advancing Translational Sciences, About NCATS: Organization and Background, 2013, http://www.ncats.nih.gov/about/org/organization.html, accessed February 13, 2014.
10. J. Kimmelman, "A Theoretical Framework for Early Human Studies: Uncertainty, Intervention Ensembles, and Boundaries, " Trials 13, no. 1 (2012): 173;
11. S. P. Hey and J. Kimmelman, "Ethics, Error, and Initial Trials of Efficacy, " Science Translational Medicine 5, no. 184 (2013): 184fs16.
12. J. Kimmelman and A. J. London, "Predicting Harms and Benefits in Translational Trials: Ethics, Evidence, and Uncertainty, " PLoS Medicine 8, no. 3 (2011): e1001010.
13. M. Hordijk-Trion et al., "Patients Enrolled in Coronary Intervention Trials Are Not Representative of Patients in Clinical Practice: Results from the Euro Heart Survey on Coronary Revascularization, " European Heart Journal 27, no. 6 (2006): 671-78.
14. P. G. Casali, "The Off-Label Use of Drugs in Oncology: A Position Paper by the European Society for Medical Oncology (ESMO), " Annals of Oncology 18, no. 12 (2007): 1923-25;
15. M. Soares, "'Off-Label' Indications for Oncology Drug Use and Drug Compendia: History and Current Status, " Journal of Oncology Practice 1, no. 3 (2005): 102-05;
16. C. D. Mullins et al., 2012. "Recommendations for Clinical Trials of Off-Label Drugs Used to Treat Advanced-Stage Cancer, " Journal of Clinical Oncology 30, no. 6 (2012): 661-66.
17. T. Fong et al., "SU5416 Is a Potent and Selective Inhibitor of the Vascular Endothelial Growth Factor Receptor (Flk-1/KDR) That Inhibits Tyrosine Kinase Catalysis, Tumor Vascularization, and Growth of Multiple Tumor Types, " Cancer Research 59, no. 1 (1999): 99.
18. J. Schlessinger, "SU11248: Genesis of a New Cancer Drug, " The Scientist, April 11, 2005, 17;
19. L. Sun et al., "Discovery of 5-[5-fluoro-2-oxo-1, 2- dihydroindol-(3Z)-ylidenemethyl]-2, 4- dimethyl-1H-pyrrole-3-carboxylic acid (2-diethylaminoethyl) amide, A Novel Tyrosine Kinase Inhibitor Targeting Vascular Endothelial and Platelet-Derived Growth Factor Receptor Tyrosine Kinase, " Journal of Medicinal Chemistry 46, no. 7 (2003): 1116-19.
20. W. Fiedler et al., "A Phase 2 Clinical Study of SU5416 in Patients with Refractory Acute Myeloid Leukemia, " Blood 102, no. 8 (2003): 2763-67.
21. S. Faivre et al., "Safety, Pharmacokinetic, and Antitumor Activity of SU11248, A Novel Oral Multitarget Tyrosine Kinase Inhibitor, in Patients with Cancer, " Journal of Clinical Oncology 24, no. 1 (2006): 25-35.
22. R. J. Motzer et al., "Sunitinib versus Interferon alfa in Metastatic Renal-Cell Carcinoma, " New England Journal of Medicine 356 (2007): 115-24.
23. G. D. Demetri, "Efficacy and Safety of Imatinib Mesylate in Advanced Gastrointestinal Stromal Tumors, " New England Journal of Medicine 347 (2002): 472-80.
24. A. Carrato et al., "Fluorouracil, Leucovorin, and Irinotecan Plus Either Sunitinib or Placebo in Metastatic Colorectal Cancer: A Randomized, Phase III trial, " Journal of Clinical Oncology 31, no. 10 (2013): 1341-47;
25. L. B. Saltz et al., "Phase II Trial of Sunitinib in Patients with Metastatic Colorectal Cancer after Failure of Standard Therapy, " Journal of Clinical Oncology 25, no. 30 (2007): 4793-99.
26. R. K. Jain et al., "Lessons from Phase III Clinical Trials on Anti-VEGF Therapy for Cancer, " Nature Clinical Practice Oncology 3, no. 1 (2006): 24-40.
27. T. G. Roberts et al., "Trends in the Risks and Benefits to Patients with Cancer Participating in Phase 1 Clinical Trials, " Journal of the American Medical Association 292 (2004): 2130-40.
28. D. W. Kim et al., "An Orally Administered Multitarget Tyrosine Kinase Inhibitor, SU11248, Is a Novel Potent Inhibitor of Thyroid Oncogenic RET/Papillary Thyroid Cancer Kinases, " Journal of Clinical Endocrinology and Metabolism 91, no. 10 (2006): 4070-76.
29. J. Desai e al., "Hypothyroidism after Sunitinib Treatment for Patients with Gastrointestinal Stromal Tumors, " Annals of Internal Medicine 145, no. 9 (2006): 660-64.
30. F. C. Kelleher and R. McDermott, "Response to Sunitinib in Medullary Thyroid Cancer, " Annals of Internal Medicine 148, no. 7 (2008): 567.
31. M. J. Bugalho, R. Domingues, and A. Borges, "A Case of Advanced Medullary Thyroid Carcinoma Successfully Treated with Sunitinib, " Oncologist 14, no. 11 (2009): 1083-87.
32. L. L. Carr et al., "Phase II Study of Daily Sunitinib in FDG-PET-Positive, Iodine-Refractory Differentiated Thyroid Cancer and Metastatic Medullary Carcinoma of the Thyroid with Functional Imaging Correlation, " Clinical Cancer Research 16, no. 21 (2010): 5260-68;
33. U.S. National Institutes of Health, "Sunitinib Malate in Treating Patients with Iodine-Refractory Recurrent or Metastatic Thyroid Cancer, " Clinicaltrials.gov., December 6, 2011, http://www.clinicaltrials.gov/ct2/show/NCT00519896?term=NCT00519896&rank=1, accessed November 6, 2012.
34. American Thyroid Association Guidelines Taskforce on Thyroid, Nodules, Cancer Differentiated Thyroid, "Revised American Thyroid Association Management Guidelines for Patients with Thyroid Nodules and Differentiated Thyroid Cancer, " Thyroid 19, no. 11 (2009): 1167-214.
35. R. M. Tuttle et al. and Network National Comprehensive Cancer, "Thyroid Carcinoma, " Journal of the National Comprehensive Cancer Network 8, no. 11 (2010): 1228-74.
36. M. L. Telli et al., "Cardiotoxicity Associated with the Cancer Therapeutic Agent Sunitinib Malate, " Annals of Oncology 19, no. 9 (2008): 1613-18.
37. Rosa, Harris, and Jayson, "The Best Guess Approach to Phase I Trial Design."
38. G. Suntharalingam et al., "Cytokine Storm in a Phase 1 Trial of the Anti-CD28 Monoclonal Antibody TGN1412, " New England Journal of Medicine 355 (2006): 1018-28.
39. C. K. Daugherty et al., "Quantitative Analysis of Ethical Issues in Phase I Trials: A Survey Interview of 144 Advanced Cancer Patients, " IRB: Ethics & Human Research 22, no. 3 (2000): 6-14.
40. Chalmers et al., "How to Increase Value and Reduce Waste When Research Priorities Are Set."
41. A. J. London, J. Kimmelman, and B. Carlisle, "Rethinking Research Ethics: The Case of Postmarketing Trials, " Science 336 (6081): 544-45.
42. Food and Drug Administration, Food and Drug Administration Amendments Act (FDAAA) of 2007, Pub. L. 110-85, Title VIII, Section 801.
43. L. H. Camacho et al., "Presentation and Subsequent Publication Rates of Phase I Oncology Clinical Trials, " Cancer 104, no. 7 (2005): 1497-504;
44. R. T. Hoeg et al., "Publication Outcomes of Phase II Oncology Clinical Trials, " American Journal of Clinical Oncology 32, no. 3 (2009): 253-57;
45. R. W. Scherer, P. Langenberg, and E. von Elm, "Full Publication of Results Initially Presented in Abstracts, " Cochrane Database of Systematic Reviews 2 (2007): MR000005;
46. A. T. Turer et al., "Publication or Presentation of Results from Multicenter Clinical Trials: Evidence from an Academic Medical Center, " American Heart Journal 153, no. 4 2007): 674-80.
47. G. A. Freeman and J. Kimmelman, "Publication and Reporting Conduct for Pharmacodynamic Analyses of Tumor Tissue in Early Phase Oncology Trials, " Clinical Cancer Research 18, no. 23 (2012): 6478-84.
48. S. Mathieu et al., "Comparison of Registered and Published Primary Outcomes in Randomized Controlled Trials, " Journal of the American Medical Association 302 (2009): 977-84.
49. S. Niraula, "The Price We Pay for Progress: A Meta-analysis of Harms of Newly Approved Anticancer Drugs, " Journal of Clinical Oncology 30, no. 24 (2012): 3012-19.
50. S. A. Greenberg, "How Citation Distortions Create Unfounded Authority: Analysis of a Citation Network, " BMJ 339 (2009): b2680.
51. A. J. London, "A Non-Paternalistic Model of Research Ethics and Oversight: Assessing the Benefits of Prospective Review, " Journal of Law, Medicine, and Ethics 40, no. 4 (2012): 930-44.