scholarly journals Should the Randomistas (Continue to) Rule?

2020 ◽  
pp. 47-78
Author(s):  
Martin Ravallion
Keyword(s):  
Observational Study ◽  
Randomized Controlled Trials ◽  
Selection Bias ◽  
Gold Standard ◽  
Policy Making ◽  
A Priori ◽  
Evidence Base ◽  
Controlled Trials ◽  
Development Applications ◽  
Unconditional Preference

The rising popularity of randomized controlled trials (RCTs) in development applications has come with continuing debates about the merits of this approach. The chapter takes stock of the issues. It argues that an unconditional preference for RCTs is questionable on three main counts. First, the case for such a preference is unclear on a priori grounds. For example, with a given budget, even a biased observational study can come closer to the truth than a costly RCT. Second, the ethical objections to RCTs have not been properly addressed by advocates. Third, there is a risk of distorting the evidence-base for informing policy-making, given that an insistence on RCTs generates selection bias in what gets evaluated. Going forward, pressing knowledge gaps should drive the questions asked and how they are answered, not the methodological preferences of some researchers. The gold standard is the best method for the question at hand.

scholarly journals An update around the evidence base for the lower extremity ultrasound regional block technique

F1000Research ◽  
2016 ◽  
Vol 5 ◽  
pp. 104 ◽  
Author(s):  
Andrea Fanelli ◽  
Daniela Ghisi ◽  
Rita Maria Melotti
Keyword(s):  
Lower Extremity ◽  
Randomized Controlled Trials ◽  
Regional Anesthesia ◽  
Current Literature ◽  
Gold Standard ◽  
Ultrasound Guidance ◽  
Evidence Base ◽  
Controlled Trials ◽  
Modest Improvement ◽  
Randomized Controlled

Ultrasound guidance currently represents the gold standard for regional anesthesia. In particular for lower extremity blocks, despite the heterogeneity and the lack of large randomized controlled trials, current literature shows a modest improvement in block onset and quality compared with other localization techniques. This review aims to present the most recent findings on the application of ultrasound guidance for each single lower extremity approach.

Making a Difference

2018 ◽  
Author(s):  
Rani Lill Anjum ◽  
Stephen Mumford
Keyword(s):  
Randomized Controlled Trials ◽  
Comparative Studies ◽  
Gold Standard ◽  
Controlled Trials ◽  
Positive Difference ◽  
Randomized Controlled ◽  
Difference Making ◽  
Making A Difference ◽  
Difference Makers ◽  
Trial Intervention

One view of what links a cause to an effect is that causes make a difference to whether or not the effect is produced. This assumption is behind comparative studies, such as the method of randomized controlled trials, aimed at showing whether a trial intervention makes a positive difference to outcomes. Comparative studies are regarded as the gold standard in some areas of research but they are also problematic. There can be causes that make no difference and some difference-makers that are not causes. This indicates that difference-making should be taken as a symptom of causation: a feature that accompanies it in some, though not all, cases. Symptoms can be useful in the discovery of causes but they cannot be definitive of causation.

Trends in randomized controlled trials in ENT: a 30-year review

1997 ◽  
Vol 111 (7) ◽  
pp. 611-613 ◽  
Author(s):  
K. W. Ah-See ◽  
N. C. Molony ◽  
A. G. D. Maran
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Randomized Controlled Trials ◽  
Gold Standard ◽  
Controlled Trials ◽  
Evidence Based ◽  
Controlled Clinical Trials ◽  
Medline Search ◽  
Randomized Controlled Clinical Trials ◽  
Randomized Controlled

AbstractThere is a growth in the demand for clinical practice to be evidence based. Recent years have seen a rise in the number of randomized controlled clinical trials (RCTS). Such trials while acknowledged as the gold standard for evidence can be difficult to perform in surgical specialities. We have recently identified a low proportion of RCTS in the otolaryngology literature. Our aim was to identify any trend in the number of published RCTS within the ENT literature over a 30-year period and to identify which areas of our speciality lend themselves to this form of study design. A Medline search of 10 prominent journals published between 1966 and 1995 was performed. Two hundred and ninety-six RCTS were identified. Only five were published before 1980. Two hundred (71 per cent) of RCTS were in the areas of otology and rhinology. An encouraging trend is seen in RCTS within ENT literature.

scholarly journals Critical analysis of the drafting of physical therapy randomized controlled trials published in Portuguese

2016 ◽  
Vol 29 (2) ◽  
pp. 421-427
Author(s):  
Ercole da Cruz Rubini ◽  
Fabio Dutra Pereira ◽  
Renato Sobral Monteiro-Junior ◽  
Patricia Zaidan ◽  
Cintia Pereira de Souza ◽  
...  
Keyword(s):  
Physical Therapy ◽  
Randomized Controlled Trials ◽  
Gold Standard ◽  
Critical Analysis ◽  
Physical Therapists ◽  
Controlled Trials ◽  
High Quality ◽  
Randomized Controlled ◽  
Methodological Procedures

Abstract Introduction: randomized controlled trials are high quality studies. Many problems related to the drafting of these studies have been identified and consequently various national and international journals, in an attempt to improve this writing, have adopted the Consolidated Standards of Reporting Trials recommendations. Regarding the studies written specifically by physical therapists, until now, the quality of the drafting in Portuguese has been unknown. Aim: To critically analyze the drafting of RCTs in the area of physical therapy, published in Portuguese, in relation to the CONSORT recommendations. Materials and Methods: On 17th Oct, 2012, 548 RCTs in Portuguese were recovered from the MEDLINE and PEDro databases, which were divided among four evaluators who, after reading the abstracts, selected those related to physical therapy. Of these studies, 78 RCTs were related to physical therapy, which were divided among the four evaluators for the analysis of the drafting according to the CONSORT recommendations. The four evaluators who participated in this study previously obtained a median kappa above 70% when their analyses were compared to the analyses of the evaluator considered the gold standard due to having greater experience. Results: The quantity of items of the CONSORT recommendations according to year of publication was very small, corresponding to a mean of 43% of the items in the articles analyzed. Conclusion: The results make very clear the need to improve the quality of the drafting of the RCTs related to physical therapy in Portuguese and to include more rigorous methodological procedures, such as sample size, randomization and blinding. The dissemination and adoption of the CONSORT recommendations by physical therapy researchers would, without doubt, be a big step towards improving this quality.

scholarly journals Comparison of Aggregated N-of-1 Trials with Parallel and Crossover Randomized Controlled Trials Using Simulation Studies

Healthcare ◽  
2019 ◽  
Vol 7 (4) ◽  
pp. 137 ◽  
Author(s):  
J. Blackston ◽  
Andrew Chapple ◽  
James McGree ◽  
Suzanne McDonald ◽  
Jane Nikles
Keyword(s):  
Randomized Controlled Trials ◽  
Sample Size ◽  
Selection Bias ◽  
Clinical Care ◽  
Controlled Trials ◽  
Type I ◽  
Operating Characteristics ◽  
Crossover Designs ◽  
Carryover Effects ◽  
Randomized Controlled

Background: N-of-1 trials offer an innovative approach to delivering personalized clinical care together with population-level research. While increasingly used, these methods have raised some statistical concerns in the healthcare community. Methods: We discuss concerns of selection bias, carryover effects from treatment, and trial data analysis conceptually, then rigorously evaluate concerns of effect sizes, power and sample size through simulation study. Four variance structures for patient heterogeneity and model error are considered in a series of 5000 simulated trials with 3 cycles, which compare aggregated N-of-1 trials to parallel randomized controlled trials (RCTs) and crossover trials. Results: Aggregated N-of-1 trials outperformed both traditional parallel RCT and crossover designs when these trial designs were simulated in terms of power and required sample size to obtain a given power. N-of-1 designs resulted in a higher type-I error probability than parallel RCT and cross over designs when moderate-to-strong carryover effects were not considered or in the presence of modeled selection bias. However, N-of-1 designs allowed better estimation of patient-level random effects. These results reinforce the need to account for these factors when planning N-of-1 trials. Conclusion: N-of-1 trial designs offer a rigorous method for advancing personalized medicine and healthcare with the potential to minimize costs and resources. Interventions can be tested with adequate power with far fewer patients than traditional RCT and crossover designs. Operating characteristics compare favorably to both traditional RCT and crossover designs.

scholarly journals Are Randomized Controlled Trials Controlled? Patient Preferences and Unblind Trials

1997 ◽  
Vol 90 (12) ◽  
pp. 652-656 ◽  
Author(s):  
Klim Mcpherson ◽  
Annie R Britton ◽  
John E Wennberg
Keyword(s):  
Randomized Controlled Trials ◽  
Patient Preferences ◽  
Treatment Effects ◽  
Evidence Base ◽  
Modern Medicine ◽  
Treatment Preferences ◽  
Controlled Trials ◽  
True Nature ◽  
Randomized Controlled ◽  
Catch 22

The most reliable information about treatment effects comes from randomized controlled trials (RCTs). However, the possibility of subtle interactions—for example, between treatment preferences and treatment effects—is generally subordinated in the quest for evidence about main treatment effects. If patient preferences can influence the effectiveness of treatments through poorly understood (psychological) pathways, then RCTs, particularly when unblinded, may wrongly attribute effects solely to a treatment's physiological/pharmacological properties. To interpret the RCT evidence base it is important to know whether any preference effects exist and, if so, by how much they affect outcome. Reliable measurement of these effects is difficult and will require new approaches to the conduct of trials. In view of the fanciful image with which such effects are portrayed and the uncertainties about their true nature and biological mechanisms, existing evidence is unlikely to provide sufficient justification for investment in trials. This is a Catch 22. Until an escape is found we might never know, even approximately, how much of modern medicine is attributable to psychological processes.

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