The Duty to Rescue and Randomized Controlled Trials Involving Serious Diseases

2018 ◽  
Vol 15 (3) ◽  
pp. 298-323
Author(s):  
Joseph Millum ◽  
David Wendler
Keyword(s):  
Medical Research ◽  
Randomized Controlled Trials ◽  
Experimental Treatment ◽  
Controlled Trials ◽  
Potential Treatment ◽  
Randomized Controlled ◽  
Experimental Treatments ◽  
Effective Treatments

During the recent Ebola epidemic, some commentators and stakeholders argued that it would be unethical to carry out a study that withheld a potential treatment from affected individuals with such a serious, untreatable disease. As a result, the initial trials of experimental treatments did not have control arms, despite important scientific reasons for their inclusion. In this paper, we consider whether the duty to rescue entails that it would be unethical to withhold an experimental treatment from patient-participants with serious diseases for which there are no effective treatments, even when doing so is scientifically necessary to test the effectiveness of the treatment. We argue that the duty to rescue will rarely apply. The context of medical research also throws new light on the content of the duty to rescue, since the interests of future patients—who stand to benefit from the fruits of medical research—are relevant to whether the duty applies.

Impact of Blinding on Patient-Reported Outcome Differences Between Treatment Arms in Cancer Randomized Controlled Trials

2021 ◽  
Author(s):  
Fabio Efficace ◽  
David Cella ◽  
Neil K Aaronson ◽  
Melanie Calvert ◽  
Francesco Cottone ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Experimental Treatment ◽  
Patient Reported Outcome ◽  
Controlled Trials ◽  
Open Label ◽  
Multivariable Logistic Regression Model ◽  
Randomized Controlled ◽  
Patient Reported ◽  
Independent Variable ◽  
Outcome Differences

Abstract Some concerns have been raised about potential bias in patient-reported outcome (PRO) results from open-label cancer randomized controlled trials (RCTs). We investigated if open-label trials favor the experimental treatment over the standard treatment more frequently than blinded trials. We also examined if the effect of blinding differs for distal vs more proximal PROs. We assessed 538 RCTs with a PRO endpoint conducted in the most prevalent cancers, of which 366 (68.0%) were open-label, 148 (27.5%) were blinded, and 24 (4.5%) were categorized as unclear. In our multivariable logistic regression model, we did not observe a statistically significant association of the independent variable treatment concealment (blinded vs open-label) on the dependent variable measuring the proportion of trials favoring the experimental treatment (adjusted odds ratio = 1.19, 95% confidence interval = 0.79 to 1.79; 2-sided P = .40). This was also the case when comparing distal and proximal PROs. Our findings provide novel evidence-based data that support the validity of PRO results from open-label cancer RCTs.

scholarly journals Efficacy of experimental treatments compared with standard treatments in non-inferiority trials: a meta-analysis of randomized controlled trials

2010 ◽  
Vol 39 (6) ◽  
pp. 1567-1581 ◽  
Author(s):  
Darius Soonawala ◽  
Rutger A Middelburg ◽  
Matthias Egger ◽  
Jan P Vandenbroucke ◽  
Olaf M Dekkers
Keyword(s):  
Randomized Controlled Trials ◽  
Meta Analysis ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Experimental Treatments

Forest plot: An indispensable segment of meta-analysis

2021 ◽  
Vol 3 (1) ◽  
pp. 3-8
Author(s):  
Vipin Ahuja ◽  
Annapurna Ahuja
Keyword(s):  
Medical Research ◽  
Randomized Controlled Trials ◽  
Graphical Representation ◽  
Meta Analysis ◽  
Review Article ◽  
Forest Plot ◽  
Controlled Trials ◽  
Graphical Display ◽  
Randomized Controlled ◽  
The Individual

Forest plot is the graphical display of estimated results from a number of scientific studies included in Meta-Analysis. The name refers to the forest of lines produced. It is also known as a blobbogram and is a graphical representation of data from studies addressing the same question, along with the overall results. It was developed for use in medical research as a means of graphically representing a meta-analysis of the results of randomized controlled trials. One of the foremost advantages of these plots is that one can see and interpret the information from the individual studies that went into meta-analysis at a glance. It also highlights the amount of variation between the studies and an estimate of the overall result. This review article throws a light on the importance of forest plots and their interpretation in the field of dental research.

Impact of Spin in the Abstracts of Articles Reporting Results of Randomized Controlled Trials in the Field of Cancer: The SPIIN Randomized Controlled Trial

2014 ◽  
Vol 32 (36) ◽  
pp. 4120-4126 ◽  
Author(s):  
Isabelle Boutron ◽  
Douglas G. Altman ◽  
Sally Hopewell ◽  
Francisco Vera-Badillo ◽  
Ian Tannock ◽  
...  
Keyword(s):  
Beneficial Effect ◽  
Randomized Controlled Trials ◽  
Primary Outcome ◽  
Controlled Trial ◽  
Experimental Treatment ◽  
Mean Difference ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Significant Difference ◽  
The Impact

Purpose We aimed to assess the impact of spin (ie, reporting to convince readers that the beneficial effect of the experimental treatment is greater than shown by the results) on the interpretation of results of abstracts of randomized controlled trials (RCTs) in the field of cancer. Methods We performed a two-arm, parallel-group RCT. We selected a sample of published RCTs with statistically nonsignificant primary outcome and with spin in the abstract conclusion. Two versions of these abstracts were used—the original with spin and a rewritten version without spin. Participants were clinician corresponding authors of articles reporting RCTs, investigators of trials, and reviewers of French national grants. The primary outcome was clinicians' interpretation of the beneficial effect of the experimental treatment (0 to 10 scale). Participants were blinded to study hypothesis. Results Three hundred clinicians were randomly assigned using a Web-based system; 150 clinicians assessed an abstract with spin and 150 assessed an abstract without spin. For abstracts with spin, the experimental treatment was rated as being more beneficial (mean difference, 0.71; 95% CI, 0.07 to 1.35; P = .030), the trial was rated as being less rigorous (mean difference, −0.59; 95% CI, −1.13 to 0.05; P = .034), and clinicians were more interested in reading the full-text article (mean difference, 0.77; 95% CI, 0.08 to 1.47; P = .029). There was no statistically significant difference in the clinicians' rating of the importance of the study or the need to run another trial. Conclusion Spin in abstracts can have an impact on clinicians' interpretation of the trial results.

Treatment efficacy for pharyngeal Neisseria gonorrhoeae: a systematic review and meta-analysis of randomized controlled trials

2020 ◽  
Vol 75 (11) ◽  
pp. 3109-3119 ◽  
Author(s):  
Fabian Y S Kong ◽  
Christina L Hatzis ◽  
Andrew Lau ◽  
Deborah A Williamson ◽  
Eric P F Chow ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Treatment Efficacy ◽  
Meta Analysis ◽  
Small Sample ◽  
Bibliographic Databases ◽  
Controlled Trials ◽  
Sample Sizes ◽  
Treatment Regimens ◽  
Randomized Controlled ◽  
Effective Treatments

Abstract Background Rising gonorrhoea rates require highly effective treatments to reduce transmission and prevent development of antimicrobial resistance. Currently the most effective treatments for pharyngeal gonorrhoea remain unclear. This review aimed to estimate treatment efficacy for pharyngeal gonorrhoea. Methods Online bibliographic databases were searched for the period 1 January 2000 to 17 September 2019 for treatments of gonorrhoea. All randomized controlled trials (RCTs) with data on pharyngeal gonorrhoea among participants aged 15 years or above, published in English, were included. Meta-analyses (random effects) were used to estimate the treatment efficacy, defined as microbiological cure, among currently recommended monotherapies and dual therapies, previously recommended but no longer used regimens and emerging drugs under evaluation. Side effects were also summarized. The study protocol was registered on PROSPERO (CRD42020149278). Results There were nine studies that included 452 participants studying 19 treatment regimens. The overall treatment efficacy for pharyngeal gonorrhoea was 98.1% (95% CI: 93.8%–100%; I2 = 57.3%; P < 0.01). Efficacy was similar for single (97.1%; 95% CI: 90.8%–100.0%; I2 = 15.6%; P = 0.29) and dual therapies (98.0%; 95% CI: 91.4%–100%; I2 = 79.1%; P < 0.01). Regimens containing azithromycin 2 g or ceftriaxone were similarly efficacious. The summary efficacy estimate for emerging drugs was 88.8% (95% CI: 76.9%–97.5%; I2 = 11.2%; P = 0.34). Small sample sizes in each trial was a major limitation. Conclusions Regimens containing ceftriaxone or azithromycin 2 g, alone or as part of dual therapies are the most efficacious for pharyngeal gonorrhoea. Further pharyngeal-specific RCTs with adequate sample sizes are needed.

The Need for Randomized Controlled Trials in Podiatric Medical Research

2004 ◽  
Vol 94 (3) ◽  
pp. 221-228 ◽  
Author(s):  
Jill Porthouse ◽  
David J. Torgerson
Keyword(s):  
Health Care ◽  
Medical Research ◽  
Randomized Controlled Trials ◽  
Controlled Trial ◽  
Controlled Trials ◽  
High Quality ◽  
Randomized Controlled ◽  
Cochrane Database ◽  
Key Features

The randomized controlled trial is the most robust method available to evaluate health-care treatments. If podiatric medical practice is to be based on rigorous evidence, then high-quality randomized controlled trials are needed to inform that practice. In this article, we examine the extent to which randomized controlled trials are used in recent podiatric medical research and appraise the quality of those that are available. Using the Cochrane database of all randomized controlled trials in health care, we found only six relevant trials undertaken in podiatric medicine since 1997. These studies were of variable quality. We also discuss the key features of a rigorous trial design. To date, the clinical practice of podiatric medicine is not adequately informed by the best available evidence. We call for more high-quality randomized controlled trials to be undertaken in podiatric medical research. (J Am Podiatr Med Assoc 94(3): 221–228, 2004)

scholarly journals The Potential Influence of Absence of Blinding Method: Secondary Analysis From Randomized Controlled Trials Concerned Acupuncture for Depression

2020 ◽  
Author(s):  
Rui-Ting Wang ◽  
Ling-Jun Cui ◽  
Jian-Ping Liu ◽  
Mei Han ◽  
Yu-Tong Fei ◽  
...  
Keyword(s):  
Randomized Controlled Trials ◽  
Meta Analysis ◽  
Depression Scale ◽  
Experimental Treatment ◽  
Controlled Trials ◽  
Hamilton Depression Scale ◽  
China National Knowledge Infrastructure ◽  
Detection Bias ◽  
Randomized Controlled ◽  
And Control

Abstract Background To evaluate the possibility and impact of performance and detection bias in randomized controlled trials due to lack of the blinding method. MethodsTrials assessing acupuncture for depression published by March 2020 were searched from China National Knowledge Infrastructure database. Through matching, we reassembled the trials of comparing experimental acupuncture and control acupuncture. Meta-analysis with post-treatment HAMD (Hamilton Depression Scale) of the newly reassembled trials was conducted. Changes of HAMD of all included groups were also analyzed, the between groups difference was then analyzed by t-test. All statistical analyses were performed using RevMan 5.3 software. ResultsThirteen pairs of studies were matched from 63 included trials. Re-analysis from the paired studies showed obvious difference of HAMD scales between the experimental and control acupuncture (Mean Difference was -2.95 for HAMD-17, -5.55 for HAMD-24). Re-analysis from all the included groups also showed the statistical difference between groups at four weeks of HAMD-17 (P=0.01), and four/six weeks of HAMD-24 (P<0.01).Conclusion Effectiveness of acupuncture for decreasing the severity of depression was superior in trials that considered acupuncture as experimental treatment, showing performance and detection bias caused by the absence of blinding method is of great significance in acupuncture trials.

Most randomized controlled trials for psoriasis used placebo comparators despite the availability of effective treatments

2021 ◽  
Vol 133 ◽  
pp. 72-79
Author(s):  
Sivem Afach ◽  
Theodoros Evrenoglou ◽  
Nadia Oubaya ◽  
Laurence Le Cleach ◽  
Emilie Sbidian
Keyword(s):  
Randomized Controlled Trials ◽  
Controlled Trials ◽  
Randomized Controlled ◽  
Effective Treatments
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