New treatments compared to established treatments in randomized trials

Cochrane Database of Systematic Reviews

Volumn 2012, Issue 10, 2012, Pages

  Djulbegovic, Benjamin ^a   Kumar, Ambuj ^b   Glasziou, Paul P ^c   Perera, Rafael ^d   Reljic, Tea ^e   Dent, Louise ^f   Raftery, James ^g   Johansen, Marit ^h   Di Tanna, Gian Luca ⁱ   Miladinovic, Branko ^b   Soares, Heloisa P ^j   Vist, Gunn E ^h   Chalmers, Iain ^k  

^a H LEE MOFFITT CANCER CENTER AND RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF SOUTH FLORIDA   (United States)

^c BOND UNIVERSITY   (Australia)

^d UNIVERSITY OF OXFORD   (United Kingdom)

^e UNIVERSITY OF SOUTH FLORIDA   (United States)

^f UNIVERSITY OF SOUTHAMPTON   (United Kingdom)

^g UNIVERSITY OF SOUTHAMPTON   (United Kingdom)

^h NORWEGIAN KNOWLEDGE CENTRE FOR THE HEALTH SERVICES   (Norway)

ⁱ SAPIENZA UNIVERSITY OF ROME   (Italy)

^j MOUNT SINAI MEDICAL CENTER   (United States)

^k James Lind Initiative   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; ETHICS; EXPERIMENTAL THERAPY; FINANCIAL MANAGEMENT; HUMAN; RANDOMIZED CONTROLLED TRIAL (TOPIC); STANDARD; TREATMENT OUTCOME;

FINANCING, GOVERNMENT; HUMANS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REFERENCE STANDARDS; THERAPIES, INVESTIGATIONAL; TREATMENT OUTCOME;

EID: 84871877801     PISSN: 14651858     EISSN: None     Source Type: Journal    
DOI: 10.1002/14651858.MR000024.pub3     Document Type: Review

References (57)

1. Dent L, Raftery J. Treatment success in pragmatic randomised controlled trials: a review of trials funded by the UK Health Technology Assessment programme. Trials 2011;12(109):1-10.
2. Djulbegovic B, Kumar A, Soares HP, Hozo I, Bepler G, Clarke M, et al. Treatment success in cancer: new cancer treatment successes identified in phase 3 randomized controlled trials conducted by the National Cancer Institute-sponsored cooperative oncology groups, 1955 to 2006. Archives of Internal Medicine 2008;168(6):632-42.
3. Johnston SC, Rootenberg JD, Katrak S, Smith WS, Elkins JS. Effect of a US National Institutes of Health programme of clinical trials on public health and costs. Lancet 2006;367:1319-27.
4. Machin D, Stenning S, Parmar M, Fayers P, Girling D, Stephens R, et al. Thirty years of medical research council randomized trials in solid tumours. Clinical Oncology 1997;9:100-14.
5. Bekelman JE, Li Y, Gross CP. Scope and impact of financial conflicts of interest in biomedical research. JAMA 2003;289(4):454-65.
6. Joffe S, Harrington DP, George SL, Emanuel EJ, Budzinski LA, Weeks JC. Satisfaction of the uncertainty principle in cancer clinical trials: retrospective cohort analysis. BMJ 2004;328(4754):1463.
7. Kumar A, Soares H, Wells R, Clarke M, Hozo I, Bleyer A, et al. Are experimental treatments for cancer in children superior to established treatments? Observational study of randomised controlled trials by the Children's Oncology Group. BMJ 2005;331(7528):1295.
8. Lathyris DN, Patsopoulos NA, Salanti G, Ioannidis JPA. Industry sponsorship and selection of comparators in randomized clinical trials. European Journal of Clinical Investigation 2010;40(2):172-82.
9. Lexchin J, Bero LA, Djulbegovic B, Clark O. Pharmaceutical industry sponsorship and research outcome and quality: systematic review [Original pharmaceutical industry sponsorship and research outcome and quality: systematic review]. BMJ 2003;326(7400):1167-70.
10. Soares HP, Daniels S, Kumar A, Clarke M, Scott C, Swann S, et al. Bad reporting does not mean bad methods for randomised trials: observational study of randomised controlled trials performed by the Radiation Therapy Oncology Group. BMJ 2004;328:22-5.
11. Yanada M, Narimatsu H, Suzuki T, Matsuo K, Naoe T. Randomized controlled trials of treatments for hematologic malignancies: study characteristics and outcomes. Cancer 2007;110(2):334-9.
12. Als-Nielsen B, Chen W, Gluud C, Kjaergard LL. Association of funding and conclusions in randomized drug trials: a reflection of treatment effect or adverse events?. JAMA 2003;290(7):921-8.
13. Altman D. The scandal of poor medical research. BMJ 1994;308:283-4.
14. Altman D, Bland M. Absence of evidence is not evidence of absence. BMJ 1995;311:485.
15. Atkins H. Conduct of a controlled clinical trial. BMJ 1966;2:377-9.
16. Bradford Hill A. Medical ethics and controlled trials. BMJ 1963;2:1043-9.
17. Bradford Hill A. Clinical trials and the acceptance of uncertainty. BMJ 1987;294:1419.
18. Chalmers I. What is the prior probability of a proposed new treatment being superior to established treatments?. BMJ 1997;314:74-5.
19. Chan AW, Haahr MT, Hróbjartsson A, Gøtzsche PC, Altman DG. Empirical evidence for selective reporting of outcomes in randomized trials: comparison of protocols to published articles. JAMA 2004;291:2547-65.
20. Colditz GA, Miller JN, Mosteller F. How study design affects outcomes in comparisons of therapy.I:medical. Statistics in Medicine 1989;8:441-54.
21. Dickersin K. Why register clinical trials?-Revisited. Controlled Clinical Trials 1992;13:170-7.
22. Dickersin K. How important is publication bias? A synthesis of available data. AIDS Education and Prevention 1997;9:15-21.
23. Djulbegovic B, Bennet CL, Adams JR, Lyman GH. Industry-sponsored research. Lancet 2000;356:2193-4.
24. Djulbegovic B, Lacevic M, Macy T, Adams J, Lyman GH. What is the probability that results of meta-analyses will favor innovative treatments?. VIII Cochrane Colloquium: Evidence for Action. Cape Town, South Africa, 2000.
25. Djulbegovic B, Lacevic M, Cantor A, Fields K, Bennett C, Adams J, et al. The uncertainty principle and industry-sponsored research. Lancet 2000;356:635-8.
26. Djulbegovic B. Acknowledgment of uncertainty: a fundamental means to ensure scientific and ethical validity in clinical research. Current Oncology Reports 2001;3:389-95.
27. Djulbegovic B. Denominator problem needs to be addressed. BMJ 2002;325:1420.
28. Djulbegovic B, Cantor A, Clarke M. The importance of preservation of the ethical principle of equipoise in the design of clinical trials: relative impact of the methodological quality domains on the treatment effect in randomized controlled trials. Accountability in Research 2003;10(4):301-15.
29. Djulbegovic B. Articulating and responding to uncertainties in clinical research. Journal of Medicine and Philosophy 2007;32(2):79-98.
30. Djulbegovic B. The paradox of equipoise: the principle that drives and limits therapeutic discoveries in clinical research. Cancer Control 2009;16(4):342-7.
31. Djulbegovic B. Uncertainty and equipoise: at interplay between epistemology, decision making and ethics. American Journal of the Medical Sciences 2011;342(4):282-9.
32. Dwan K, Altman DG, Creswell L, Blundell M, Gamble CL, Williamson PR. Comparison of protocols and registry entries to published reports for randomised controlled trials. Cochrane Database of Systematic Reviews 2011, Issue 1. [DOI: 10.1002/14651858.MR000031.pub2]
33. Edwards SJL, Lilford RJ, Braunholtz DA, Jackson JC, Hewison J, Thornton J. Ethical issues in the design and conduct of randomized controlled trials. Health Technology Assessment 1998;2(15):1-130.
34. Freedman B. Equipoise and the ethics of clinical research. New England Journal of Medicine 1987;317:141-5.
35. Fries JF, Krishnan E. Equipoise, design bias, and randomized controlled trials: the elusive ethics of new drug development. Arthritis Research & Therapy 2004;6(3):R250-5.
36. Gisbert F, Goerlich J. Weighted samples, kernel density estimators and convergence. Empirical Economics 2003;28:335-51.
37. Gluud LL. Bias in clinical intervention research. American Journal of Epidemiology 2006;163(3):493-501.
38. Hedges LV, Olkin I. Statistical Methods for Meta-analysis. San Diego, CA: Academic Press, 1985.
39. Higgins JPT, Green S, editors. Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 [updated March 2011]. Chichester, UK: John Wiley & Sons Ltd, 2011.
40. Hopewell S, Clarke M, Stewart L, Tierney J. Time to publication for results of clinical trials. Cochrane Database of Systematic Reviews 2007, Issue 2. [DOI: 10.1002/14651858.MR000011.pub2]
41. Hopewell S, Loudon K, Clarke MJ, Oxman AD, Dickersin K. Publication bias in clinical trials due to statistical significance or direction of trial results. Cochrane Database of Systematic Reviews 2009, Issue 1. [DOI: 10.1002/14651858.MR000006.pub3]
42. Juni P, Witschi A, Bloch R, Egger M. The hazards of scoring the quality of clinical trials for meta-analysis. JAMA 1999;282:1054-60.
43. Krzyzanowska MK, Pintilie M, Tannock IF. Factors associated with failure to publish large randomized trials presented at an oncology meeting. JAMA 2003;290(4):495-501.
44. Kumar A, Soares H, Serdarevic F. Totality of evidence: one of the keys to better oncology management. Journal of Oncology Management 2005;14(1):12-4.
45. Lexchin J, Bero LA, Djulbegovic B, Clark O. Pharmaceutical industry sponsorship and research outcome and quality: systematic review. BMJ 2003;326:1167-70.
46. Lilford RJ, Djulbegovic B. Equipoise is essential principle of human experimentation. BMJ 2001;322:299-300.
47. Mandrekar SJ, Sargent DJ. Clinical trial designs for predictive biomarker validation: theoretical considerations and practical challenges. Journal of Clinical Oncology 2009;27(24):4027-34.
48. Mann H, Djulbegovic B. Comparator bias: why comparisons must address genuine uncertainties. James Lind Library (http://www.jameslindlibrary.org/illustrating/articles/comparator-bias-why-comparisons-must-address-genuine-uncertaint) 2012.
49. Monagan MB, Geddes KO, Heal KM, Labahn G, Vorkoetter Sm, McCarron J, et al. Maple 14 Programming Guide. Waterloo ON, Canada: Maplesoft, 2009.
50. Peto R, Baigent C. Trials: the next 50 years. Large scale randomised evidence of moderate benefits. BMJ 1998;317:1170-1.
51. Schulz KF. Subverting randomization in controlled trials. JAMA 1995;274:1456-8.
52. Senn S. Placebo confusion. BMJ eletters 15 August 2000.
53. Silverman BW. Density estimation for statistics and data analysis. Monographs on Statistics and Applied Probability. London: Chapman and Hall, 1986.
54. Soares HP, Daniels S, Kumar A, Clarke M, Scott C, Swann S, et al. Bad reporting does not mean bad methods for randomised trials: observational study of randomised controlled trials performed by the Radiation Therapy Oncology Group. BMJ 2004;328(7430):22-4.
55. Soares HP, Kumar A, Daniels S, Swann S, Cantor A, Hozo I, et al. Evaluation of new treatments in radiation oncology: are they better than standard treatments?. JAMA 2005;293(8):970-8.
56. Weijer C, Shapiro SH, Cranley Glass K. For and against: clinical equipoise and not the uncertainty principle is the moral underpinning of the randomised controlled trial. BMJ 2000;321(7263):756-8.
57. Wood L, Egger M, Gluud LL, Schulz KF, Juni P, Altman DG, et al. Empirical evidence of bias in treatment effect estimates in controlled trials with different interventions and outcomes: meta-epidemiological study. BMJ 2008;336(7644):601-5.