Skip to main content
Erschienen in: European Journal of Epidemiology 8/2019

11.04.2019 | ESSAY

Generalizing from the results of randomized studies of treatment: Can non-randomized studies be of help?

verfasst von: Noel S. Weiss

Erschienen in: European Journal of Epidemiology | Ausgabe 8/2019

Einloggen, um Zugang zu erhalten

Excerpt

Different patients who have the same illness may receive different forms of treatment. By documenting their treatment and the progression and complications of their illness, an attempt can be made to draw inferences regarding the relative impact of these different treatments. However, the interpretation of such comparisons can be compromised by underlying differences in the likelihood of the various outcome events among the patient treatment groups. Differences of opinion exist with respect to the frequency and magnitude of the confounding that may arise from these underlying differences. Benson and Hartz [1] concluded that “misuse of observational studies [of the efficacy of therapy] does not often occur in the recent literature”, and that nonrandomized studies of therapy “usually do provide valid information.” On the other hand, Pocock and Elbourne [2] state that greater reliance on the results of nonrandomized studies of therapeutic efficacy could lead to “considerable dangers to clinical research and even to the well-being of patients.” Particularly strident on this point was an editorial [3] which opined that “In an era of evidence-based medicine, controlled clinical trials are required to introduce new therapies into the clinic.” …
Literatur
1.
Zurück zum Zitat Benson K, Hartz AJ. A comparison of observational studies and randomized controlled trials. N Engl J Med. 2000;342:1878–86.CrossRefPubMed Benson K, Hartz AJ. A comparison of observational studies and randomized controlled trials. N Engl J Med. 2000;342:1878–86.CrossRefPubMed
2.
Zurück zum Zitat Pocock SJ, Elbourne DR. Randomized trials or observational tribulations? N Engl J Med. 2000;342:1907–9.CrossRefPubMed Pocock SJ, Elbourne DR. Randomized trials or observational tribulations? N Engl J Med. 2000;342:1907–9.CrossRefPubMed
4.
Zurück zum Zitat Weiss NS, Clinical epidemiology: the study of the occurrence of illness. Oxford, New York; 2006. Weiss NS, Clinical epidemiology: the study of the occurrence of illness. Oxford, New York; 2006.
6.
Zurück zum Zitat Weiss NS, Koepsell TD, Psaty BM. Generalizability of the results of randomized trials. Arch Intern Med. 2008;168:133–5.CrossRefPubMed Weiss NS, Koepsell TD, Psaty BM. Generalizability of the results of randomized trials. Arch Intern Med. 2008;168:133–5.CrossRefPubMed
7.
Zurück zum Zitat Pitt B, Zannad F, Remme WJ, et al. The effect of spironolactone on morbidity and mortality in patients with severe heart failure. N Engl J Med. 1999;341:709–17.CrossRefPubMed Pitt B, Zannad F, Remme WJ, et al. The effect of spironolactone on morbidity and mortality in patients with severe heart failure. N Engl J Med. 1999;341:709–17.CrossRefPubMed
8.
Zurück zum Zitat Juurlink DN, Mamdani M, Kopp A, et al. Drug–drug interactions among elderly patients hospitalized for drug toxicity. JAMA. 2003;289:1652–8.CrossRefPubMed Juurlink DN, Mamdani M, Kopp A, et al. Drug–drug interactions among elderly patients hospitalized for drug toxicity. JAMA. 2003;289:1652–8.CrossRefPubMed
9.
Zurück zum Zitat Grant RM, Lama JR, Anderson PI, et al. Pre-exposure prophylaxis for HIV prevention in men who have sex with men. N Engl J Med. 2010;363:2587–99.CrossRefPubMedPubMedCentral Grant RM, Lama JR, Anderson PI, et al. Pre-exposure prophylaxis for HIV prevention in men who have sex with men. N Engl J Med. 2010;363:2587–99.CrossRefPubMedPubMedCentral
10.
Zurück zum Zitat McCormack S, Dunn DT, Sesai M, et al. Pre-exposure prophylaxis to prevent the acquisition of HIV-1 infection (PROUD): effectiveness results from the pilot phase of a pragmatic open-label randomized trial. Lancet. 2016;387:53–60.CrossRefPubMedPubMedCentral McCormack S, Dunn DT, Sesai M, et al. Pre-exposure prophylaxis to prevent the acquisition of HIV-1 infection (PROUD): effectiveness results from the pilot phase of a pragmatic open-label randomized trial. Lancet. 2016;387:53–60.CrossRefPubMedPubMedCentral
11.
Zurück zum Zitat Elting LS, Cooksley C, Bekele BN, et al. Generalizability of cancer clinical trial results: prognostic differences between participants and nonparticipants. Cancer. 2006;106:2452–8.CrossRefPubMed Elting LS, Cooksley C, Bekele BN, et al. Generalizability of cancer clinical trial results: prognostic differences between participants and nonparticipants. Cancer. 2006;106:2452–8.CrossRefPubMed
12.
Zurück zum Zitat Van de Water W, Kiderlen M, Bastiaannet F, et al. External validity of a trial comprised of elderly patients with hormone receptor-positive breast cancer. JNCI. 2014;106:1–8.CrossRef Van de Water W, Kiderlen M, Bastiaannet F, et al. External validity of a trial comprised of elderly patients with hormone receptor-positive breast cancer. JNCI. 2014;106:1–8.CrossRef
13.
Zurück zum Zitat Atrial Fibrillation Investigators. Risk factors for stroke and efficacy of antithrombotic therapy in atrial fibrillation: analysis of pooled data from five randomized controlled trials. Arch Intern Med. 1994;154:1449–57.CrossRef Atrial Fibrillation Investigators. Risk factors for stroke and efficacy of antithrombotic therapy in atrial fibrillation: analysis of pooled data from five randomized controlled trials. Arch Intern Med. 1994;154:1449–57.CrossRef
14.
Zurück zum Zitat Stern S, Altkorn D, Levinson W. Anticoagulation for chronic atrial fibrillation. JAMA. 2000;283:2901–3.CrossRefPubMed Stern S, Altkorn D, Levinson W. Anticoagulation for chronic atrial fibrillation. JAMA. 2000;283:2901–3.CrossRefPubMed
15.
Zurück zum Zitat Horwitz RI, Singer BH. Why evidence-based medicine failed in patient care and medicine-based evidence will succeed. J Clin Epidemiol. 2017;84:14–7.CrossRefPubMed Horwitz RI, Singer BH. Why evidence-based medicine failed in patient care and medicine-based evidence will succeed. J Clin Epidemiol. 2017;84:14–7.CrossRefPubMed
16.
Zurück zum Zitat Lauer MS, Collins FS. Using science to improve the nation’s health system: NIH’s commitment to comparative effectiveness research. JAMA. 2010;303:2182–3.CrossRefPubMed Lauer MS, Collins FS. Using science to improve the nation’s health system: NIH’s commitment to comparative effectiveness research. JAMA. 2010;303:2182–3.CrossRefPubMed
17.
Zurück zum Zitat Goldman B. Treatments that work for people just like you. Winter: Stanford Medicine; 2016. p. 32. Goldman B. Treatments that work for people just like you. Winter: Stanford Medicine; 2016. p. 32.
18.
Zurück zum Zitat Weiss NS. The new world of data linkages in clinical epidemiology: Are we being brave or foolhardy? Epidemiology. 2011;22:292–4.CrossRefPubMed Weiss NS. The new world of data linkages in clinical epidemiology: Are we being brave or foolhardy? Epidemiology. 2011;22:292–4.CrossRefPubMed
19.
Zurück zum Zitat Baxter NN, Goldwasser MA, Paszat LF, et al. Association of colonoscopy and death from colorectal cancer. Ann Intern Med. 2009;150:1–8.CrossRefPubMed Baxter NN, Goldwasser MA, Paszat LF, et al. Association of colonoscopy and death from colorectal cancer. Ann Intern Med. 2009;150:1–8.CrossRefPubMed
20.
Zurück zum Zitat Weiss NS, Dhillon PK, Etzioni R. Case-control studies of the efficacy of cancer screening: overcoming bias from non-random patterns of screening. Epidemiology. 2004;15:409–13.CrossRefPubMed Weiss NS, Dhillon PK, Etzioni R. Case-control studies of the efficacy of cancer screening: overcoming bias from non-random patterns of screening. Epidemiology. 2004;15:409–13.CrossRefPubMed
21.
Zurück zum Zitat Ray WA, Griffin MR, Schaffner W, et al. Psychotropic drug use and risk of fracture. N Engl Med. 1987;316:363–9.CrossRef Ray WA, Griffin MR, Schaffner W, et al. Psychotropic drug use and risk of fracture. N Engl Med. 1987;316:363–9.CrossRef
22.
Zurück zum Zitat Gerstman BB, Friesman JP, Hine LK. Use of subsequent anticoagulants to increase the predictive value of Medicaid deep venous thromboembolism diagnosis. Epidemiology. 1990;1:122–7.CrossRefPubMed Gerstman BB, Friesman JP, Hine LK. Use of subsequent anticoagulants to increase the predictive value of Medicaid deep venous thromboembolism diagnosis. Epidemiology. 1990;1:122–7.CrossRefPubMed
23.
Zurück zum Zitat Jackson LA, Jackson ML, Nelson JC, et al. Evidence of bias in estimates of influenza vaccine effectiveness in seniors. Int J Epidemiol. 2006;35:337–44.CrossRefPubMed Jackson LA, Jackson ML, Nelson JC, et al. Evidence of bias in estimates of influenza vaccine effectiveness in seniors. Int J Epidemiol. 2006;35:337–44.CrossRefPubMed
24.
Zurück zum Zitat Pignon JP, Tribodet H, Scagliotti GV, et al. Lung adjuvant cisplatin evaluation: a pooled analysis by the LACE Collaborative Group. J Clin Oncol. 2008;26:3552–9.CrossRefPubMed Pignon JP, Tribodet H, Scagliotti GV, et al. Lung adjuvant cisplatin evaluation: a pooled analysis by the LACE Collaborative Group. J Clin Oncol. 2008;26:3552–9.CrossRefPubMed
25.
Zurück zum Zitat Ganti AK, Williams CD, Gajra A, et al. Effect of age on the efficacy of adjuvant chemotherapy for resected non-small cell lung cancer. Cancer. 2015;121:2578–85.CrossRefPubMed Ganti AK, Williams CD, Gajra A, et al. Effect of age on the efficacy of adjuvant chemotherapy for resected non-small cell lung cancer. Cancer. 2015;121:2578–85.CrossRefPubMed
Metadaten
Titel
Generalizing from the results of randomized studies of treatment: Can non-randomized studies be of help?
verfasst von
Noel S. Weiss
Publikationsdatum
11.04.2019
Verlag
Springer Netherlands
Erschienen in
European Journal of Epidemiology / Ausgabe 8/2019
Print ISSN: 0393-2990
Elektronische ISSN: 1573-7284
DOI
https://doi.org/10.1007/s10654-019-00516-3

Weitere Artikel der Ausgabe 8/2019

European Journal of Epidemiology 8/2019 Zur Ausgabe