When to randomize, or 'Evidence-based medicine needs Medicine-based evidence'

Pharmacoepidemiology and Drug Safety

Volumn 21, Issue SUPPL.2, 2012, Pages 6-12

  Concato, John ^a,b  

^a VA CONNECTICUT HEALTHCARE SYSTEM   (United States)

^b YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CANCER MORTALITY; CANCER PREVENTION; CANCER SCREENING; CLINICAL DECISION MAKING; COMPARATIVE EFFECTIVENESS; CONCEPTUAL FRAMEWORK; COST EFFECTIVENESS ANALYSIS; DRUG COST; DRUG EFFICACY; DRUG SAFETY; EVIDENCE BASED MEDICINE; EVIDENCE BASED PRACTICE; HEALTH CARE; HUMAN; INTELLIGENCE; MEDICAL ETHICS; MEDICAL RESEARCH; MEDICINE BASED EVIDENCE; METHODOLOGY; NOTE; OBSERVATIONAL STUDY; PRIORITY JOURNAL; PROSTATE CANCER; RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); SCIENTIFIC LITERATURE; TREATMENT OUTCOME; TREATMENT RESPONSE;

COST-BENEFIT ANALYSIS; ETHICS, MEDICAL; EVIDENCE-BASED MEDICINE; HUMANS; OUTCOME AND PROCESS ASSESSMENT (HEALTH CARE); RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; UNITED STATES;

EID: 84860548638     PISSN: 10538569     EISSN: 10991557     Source Type: Journal    
DOI: 10.1002/pds.3245     Document Type: Note

References (50)

1. Agency for Healthcare Research and Quality. Effective Health Care Program (DEcIDE Research Network): Third Symposium on Comparative Effectiveness Research Methods. Methods for developing and analyzing clinically rich data for patient-centered outcomes research, June 6-7, 2011. [8 September 2011].
2. National Institutes of Health. Definition of Clinical Research: Patient-oriented research. [8 September 2011].
3. Evidence-Based Medicine Working Group. Evidence-based medicine: A new approach to teaching the practice of medicine. JAMA 1992; 268: 2420-2425.
4. Guyatt GH, Sackett DL, Sinclair JC, Haywar R, Cook DJ, Cook RJ. Users' guide to the medical literature: IX. A method for grading health care recommendations. JAMA 1995; 274: 1800-1804.
5. Sackett DL, Straus SE, Richardson WS, et al. Evidence-based medicine: How to practice and teach EBM (2nd edn). Edinburgh: Churchill Livingstone, 2000.
6. Swales JD. Evidence-based medicine and hypertension. J Hypertens 1999; 17: 1511-1516.
7. Sehon SR, Stanley DE. A philosophical analysis of the evidence-based medicine debate. BMC Health Serv Res 2003; 3: 14-23.
8. Adams CP, Brantner VV. Estimating the cost of new drug development: Is it really $802 million? Health Aff 2006; 25: 420-428.
9. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: New estimates of drug development costs. J Health Econ 2003; 22: 151-185.
10. Feinstein AR. Current problems and future challenges in randomized clinical trials. Circulation 1984; 70: 767-774.
11. Ziemer DC, Phillips LS. Re: The dogma of "tight control": Beyond the limits of evidence (letter). Arch Intern Med 2006; 166: 1671-1672.
12. Miller FG, Joffe S. Equipose and the dilemma of randomized clinical trials. N Engl J Med 2001; 364: 476-480.
13. Horwitz RI. Complexity and contradiction in clinical trial research. Am J Med 1987; 82: 498-510.
14. LeLorier J, Gregoire G, Benhaddad A, Lapierre J, Derderian F. Discrepancies between meta-analyses and subsequent large randomized, controlled trials. N Engl J Med 1997; 337: 536-542.
15. Coca SG, Krumholz HM, Garg AX, Parikh CR. Underrepresentation of renal disease in randomized controlled trials of cardiovascular disease. JAMA 2006; 296: 1377-1384.
16. Caplan LR. Treatment of acute stroke. Still struggling. JAMA 2004; 292: 1883-1885.
17. Weintraub MI. Thrombolysis (tissue plasminogen activator) in stroke. A medicolegal quagmire. Stroke 2006; 37: 1917-1922.
18. Horwitz RI, Viscoli CM, Clemens JD, Sadock RT. Developing improved observational methods for evaluating therapeutic effectiveness. Am J Med 1990; 89: 630-638.
19. McKee M, Britton A, Black N, McPherson K, Sanderson C, Bain C. Methods in health services research: Interpreting the evidence: choosing between randomised and non-randomised studies. BMJ 1999; 319: 312-315.
20. Benson K, Hartz AJ. A comparison of observational studies and randomized, controlled trials. N Engl J Med 2000; 342: 1878-1886.
21. Concato J, Shah N, Horwitz RI. Randomized, controlled trials, observational studies, and the hierarchy of research designs. N Engl J Med 2000; 342: 1887-1892.
22. Shrier I, Boivin JF, Steele RJ, et al. Should meta-analyses of interventions include observational studies in addition to randomized controlled trials? A critical examination of underlying principles. Am J Epidemiol 2007; 166: 1203-1209.
23. Golder S, Loke YK, Bland M. Meta-analyses of adverse effects data derived from randomized controlled trials as compared to observational studies: Methodological overview. PLoS Med 2011; 8: e1001026. DOI:10.1371/journal.pmed.1001026.
24. Tsimberidou AM, Braiteh F, Stewart DJ, Kurzrock R. Ultimate fate of oncology drugs approved by the US Food and Drug Administration without a randomized trial. J Clin Oncol 2009; 27: 6243-6250.
25. Connors AR, Speroff T, Dawson NV, et al. The effectiveness of right heart catheterization in the initial care of critically ill patients. JAMA 1996; 276: 889-897.
26. McCellan M, McNeill BJ, Newhouse JP. Does more intensive treatment of acute myocardial infarction in the elderly reduce mortality? JAMA 1994; 272: 859-866.
27. D'Agostino RB Jr., D'Agostino RB Sr. Estimating treatment effects using observational data. JAMA 2007; 297: 314-316.
28. Tannen RL, Weiner MG, Xie D. Use of primary care electronic medical record database in drug efficacy research on cardiovascular outcomes: Comparison of database and randomized controlled trial findings. BMJ 2009; 338: b81.
29. Concato J, Horwitz RI. Beyond randomized versus observational studies. Lancet 2004; 363: 1660-1661.
30. Rothwell PM. External validity of randomized controlled trials: "To whom do the results of this trial apply?" Lancet 2005; 365: 82-83.
31. Feinstein AR, Horwitz RI. Problems in the "evidence" of "evidence-based medicine". Am J Med 1997; 103: 529-535.
32. Ligthelm RJ, Borzi V, Gumprecht J, Kawamori R, Wenying Y, Valensi P. Importance of observational studies in clinical practice. Clin Ther 2007; 29: 1284-1292.
33. Borgerson K. Valuing evidence. Bias and the evidence hierarchy of evidence-based medicine. Perspect Biol Med 2009; 52: 218-233.
34. Concato J, Lawler EV, Lew RA, Gaziano JM, Aslan M, Huang GD. Observational methods in comparative effectiveness research. Am J Med 2010; 123: e16-e23.
35. Garbe E, Suissa S. Issues to debate on the Women's Health Initiative (WHI) study. Hormone replacement therapy and acute coronary outcomes: Methodological issues between randomized and observational studies. Hum Reprod 2004; 19: 8-13.
36. Tannen RL, Weiner MG, Xie D, Barnhart K. A stimulation using data from a primary care practice database closely replicated the women's health initiative trial. J Clin Epidemiol 2007; 60: 686-695.
37. Vanderbroucke JP. The HRT controversy: Observational studies and RCTs fall in line. Lancet 2009; 373: 1233-1235.
38. Harris R, Lohr KN. Screening for prostate cancer: An update of the evidence for the U.S. preventive services task force. Ann Intern Med 2002; 137: 917-929.
39. Lin K, Lipsitz R, Miller T, Janakiraman S. Benefits and harms of prostate-specific antigen screening for prostate cancer: An evidence update for the U.S. Preventive Services Task Force. Ann Intern Med 2008; 149: 192-199.
40. Djulbegovic M, Beyth RJ, Neuberger MM, et al. Screening for prostate cancer: Systematic review and meta-analysis of randomized controlled trials. BMJ 2010; 341: 4543-4551.
41. Labrie F, Candas B, Dupont A, et al. Screening decreases prostate cancer death: First analysis of the 1988 Quebec prospective randomized controlled trial. Prostate 1999; 38: 83-91.
42. Adriole GL, Crawford ED, Grubb RL, III, et al. Mortality results from a randomized prostate-cancer screening trial. N Engl J Med 2009; 360: 1310-1319.
43. Schroder FH, Hugosson J, Roobol MG, et al. Screening and prostate-cancer mortality in a randomized European study. N Eng J Med 2009; 360: 1320-1328.
44. Barry MJ. Screening for prostate cancer-the controversy that refuses to die. N Engl J Med 2009; 360: 1351-1354.
45. Concato J. What will the emperor say? Screening for prostate cancer as of 2008. Cancer J 2009; 15: 7-12.
46. Consolidated Standards of Reporting Trials. The CONSORT 2010 Statement. [9 September 2011].
47. Bulletin of the World Health Organization. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: Guidelines for reporting observational studies. [9 September 2011].
48. Pocock SJ, Elbourne DR. Randomized trials or observational tribulations? N Engl J Med (Editorial) 2000; 342: 1907-1909.
49. Worrall J. Why there's no cause to randomize. Br J Philos Sci 2007; 58: 451-488.
50. Rawlings M. De Testimonio: On the evidence for decisions about the use of therapeutic interventions. Lancet 2008; 372: 2152-2161.