1. Comis, R, Aldige, C, Stovall, E, et al. A quantitative survey of public attitudes towards cancer clinical trials. Proc Am Soc Clin Oncol 2000; 19: 10.
Google Scholar2. Daugherty, CK, Ratain, MJ, Emanuel, EJ, et al. Ethical, scientific, and regulatory perspectives regarding the use of placebos in cancer clinical trials. J Clin Oncol 2008; 26: 1371–1378.
Google Scholar | Crossref | Medline | ISI3. Ratain, MJ, Sargent, DJ. Optimising the design of phase II oncology trials: the importance of randomisation. Eur J Cancer 2009; 45(2): 275–280.
Google Scholar | Crossref | Medline4. Tang, H, Foster, NR, Grothey, A, et al. Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. J Clin Oncol 2010; 28: 1936–1941.
Google Scholar | Crossref | Medline | ISI5. Chvetzoff, G, Tannock, IF. Placebo effects in oncology. J Natl Cancer Inst 2003; 95: 19–29.
Google Scholar | Crossref | Medline6. Ghatalia, P, Morgan, CJ, Sonpavde, G. Meta-analysis of regression of advanced solid tumors in patients receiving placebo or no anti-cancer therapy in prospective trials. Crit Rev Oncol Hematol 2016; 98: 122–136.
Google Scholar | Crossref | Medline7. Le Tourneau, C, Paoletti, X, Coquan, E, et al. Critical evaluation of disease stabilization as a measure of activity of systemic therapy: lessons from trials with arms in which patients do not receive active treatment. J Clin Oncol 2014; 32: 260–263.
Google Scholar | Crossref | Medline8. Mills, EJ, Seely, D, Rachlis, B, et al. Barriers to participation in clinical trials of cancer: a meta-analysis and systematic review of patient-reported factors. Lancet Oncol 2006; 7(2): 141–148.
Google Scholar | Crossref | Medline | ISI9. ICH . Choice of control group and related issues in clinical trial E10. Geneva: International conference on harmonization of technical requirements for registration of pharmaceuticals for human use. 2000, https://database.ich.org/sites/default/files/E10_Guideline.pdf
Google Scholar10. Avins, AL. Can unequal be more fair? Ethics, subject allocation, and randomised clinical trials. J Med Ethics 1998; 24(6): 401–408.
Google Scholar | Crossref | Medline | ISI11. Dibao-Dina, C, Caille, A, Sautenet, B, et al. Rationale for unequal randomization in clinical trials is rarely reported: a systematic review. J Clin Epidemiol 2014; 67(10): 1070–1075.
Google Scholar | Crossref | Medline12. Dumville, JC, Hahn, S, Miles, JN, et al. The use of unequal randomisation ratios in clinical trials: a review. Contemp Clin Trials 2006; 27(1): 1–12.
Google Scholar | Crossref | Medline | ISI13. Hey, SP, Kimmelman, J. The questionable use of unequal allocation in confirmatory trials. Neurology 2014; 82: 77–79.
Google Scholar | Crossref | Medline | ISI14. Peckham, E, Brabyn, S, Cook, L, et al. The use of unequal randomisation in clinical trials: an update. Contemp Clin Trials 2015; 45(PtA): 113–122.
Google Scholar | Crossref | Medline15. Holubkov, R, Dean, JM, Berger, J, et al. Is “rescue” therapy ethical in randomized controlled trials. Pediatr Crit Care Med 2009; 10(4): 431–438.
Google Scholar | Crossref | Medline | ISI16. Korn, EL, Freidlin, B, Abrams, JS. Overall survival as the outcome for randomized clinical trials with effective subsequent therapies. J Clin Oncol 2011; 29: 2439–2442.
Google Scholar | Crossref | Medline | ISI17. Prasad, V, Grady, C. The misguided ethics of crossover trials. Contemp Clin Trials 2014; 37(2): 167–169.
Google Scholar | Crossref | Medline18. Dibao-Dina, C, Caille, A, Giraudeau, B. Unbalanced rather than balanced randomized controlled trials are more often positive in favor of the new treatment: an exposed and nonexposed study. J Clin Epidemiol 2015; 68(8): 944–949.
Google Scholar | Crossref | Medline19. Sekeres, MA, Lancet, JE, Wood, BL, et al. Randomized phase IIb study of low-dose cytarabine and lintuzumab versus low-dose cytarabine and placebo in older adults with untreated acute myeloid leukemia. Haematologica 2013; 98(1): 119–128.
Google Scholar | Crossref | Medline20. Solomon, MT, Selva, JC, Figueredo, J, et al. Radiotherapy plus nimotuzumab or placebo in the treatment of high grade glioma patients: results from a randomized, double blind trial. BMC Cancer 2013; 13: 299.
Google Scholar | Crossref | Medline21. Parker, C, Nilsson, S, Heinrich, D, et al. Alpha emitter radium-223 and survival in metastatic prostate cancer. N Engl J Med 2013; 369: 213–223.
Google Scholar | Crossref | Medline | ISI22. Sabbatini, P, Harter, P, Scambia, G, et al. Abagovomab as maintenance therapy in patients with epithelial ovarian cancer: a phase III trial of the AGO OVAR, COGI, GINECO, and GEICO—the MIMOSA study. J Clin Oncol 2013; 31: 1554–1561.
Google Scholar | Crossref | Medline23. Vansteenkiste, J, Zielinski, M, Linder, A, et al. Adjuvant MAGE-A3 immunotherapy in resected non-small-cell lung cancer: phase II randomized study results. J Clin Oncol 2013; 31: 2396–2403.
Google Scholar | Crossref | Medline | ISI24. Freedman, B. Equipoise and the ethics of clinical research. N Engl J Med 1987; 317: 141–145.
Google Scholar | Crossref | Medline | ISI25. Djulbegovic, B, Kumar, A, Miladinovic, B, et al. Treatment success in cancer: industry compared to publicly sponsored randomized controlled trials. PLoS ONE 2013; 8(3): e58711.
Google Scholar | Crossref | Medline26. Abola, MV, Prasad, V, Jena, AB. Association between treatment toxicity and outcomes in oncology clinical trials. Ann Oncol 2014; 25(11): 2284–2289.
Google Scholar | Crossref | Medline27. Michiels, S, Saad, ED, Buyse, M. Progression-free survival as a surrogate for overall survival in clinical trials of targeted therapy in advanced solid tumors. Drugs 2017; 77: 713–719.
Google Scholar | Crossref | Medline28. Paoletti, X, Lewsley, LA, Daniele, G, et al. Assessment of progression-free survival as a surrogate end point of overall survival in first-line treatment of ovarian cancer: a systematic review and meta-analysis. JAMA Netw Open 2020; 3: e1918939.
Google Scholar | Crossref | Medline29. Carlisle, BG, Doussau, A, Kimmelman, J. Patient burden and clinical advances associated with postapproval monotherapy cancer drug trials: a retrospective cohort study. BMJ Open 2020; 10: e034306.
Google Scholar | Crossref | Medline30. Carlisle, BG, Doussau, A, Kimmelman, J. Benefit, burden, and impact for a cohort of post-approval cancer combination trials. Clin Trials 2020; 17(1): 18–29.
Google Scholar | SAGE Journals | ISI31. Hutchinson, N, Carlisle, B, Doussau, A, et al. Patient participation in clinical trials of oncology drugs and biologics preceding approval by the US Food and Drug Administration. JAMA Netw Open 2021; 4: e2110456.
Google Scholar | Crossref | Medline32. Federico, CA, Wang, T, Doussau, A, et al. Assessment of pregabalin postapproval trials and the suggestion of efficacy for new indications: a systematic review. JAMA Intern Med 2019; 179: 90–97.
Google Scholar | Crossref | Medline33. Feustel, AC, MacPherson, A, Fergusson, DA, et al. Risks and benefits of unapproved disease-modifying treatments for neurodegenerative disease. Neurology 2020; 94: e1–e14.
Google Scholar | Crossref | Medline34. Kimmelman, J. Better to be in the placebo arm for trials of neurological therapies. Cell Transplant 2018; 27(4): 677–681.
Google Scholar | SAGE Journals | ISI35. Horng, S, Grady, C. Misunderstanding in clinical research: distinguishing therapeutic misconception, therapeutic misestimation, and therapeutic optimism. IRB 2003; 25(1): 11–16.
Google Scholar | Crossref | Medline36. Booth, CM, Del Paggio, JC. Approvals in 2016: questioning the clinical benefit of anticancer therapies. Nat Rev Clin Oncol 2017; 14: 135–136.
Google Scholar | Crossref | Medline37. Booth, CM, Eisenhauer, EA. Progression-free survival: meaningful or simply measurable? J Clin Oncol 2012; 30: 1030–1033.
Google Scholar | Crossref | Medline | ISI38. Chalmers, I, Glasziou, P. Avoidable waste in the production and reporting of research evidence. Lancet 2009; 374: 86–89.
Google Scholar | Crossref | Medline | ISI39. Hey, SP, Kimmelman, J. Do we know whether researchers and reviewers are estimating risk and benefit accurately. Bioethics 2016; 30(8): 609–617.
Google Scholar | Crossref | Medline40. Dibao-Dina, C, Caille, A, Giraudeau, B. Heterogeneous perception of the ethical legitimacy of unbalanced randomization by institutional review board members: a clinical vignette-based survey. Trials 2018; 19: 440.
Google Scholar | Crossref | Medline41. Benjamin, DM, Mandel, DR, Barnes, T, et al. Can oncologists predict the efficacy of treatments in randomized trials. Oncologist 2021; 26(1): 56–62.
Google Scholar | Crossref | Medline42. Del Paggio, JC, Azariah, B, Sullivan, R, et al. Do contemporary randomized controlled trials meet ESMO thresholds for meaningful clinical benefit? Ann Oncol 2017; 28: 157–162.
Google Scholar | Crossref | Medline