Rethinking the pros and cons of randomized controlled trials and observational studies in the era of big data and advanced methods: a panel discussion

Abstract

Randomized controlled trials (RCTs) have traditionally been considered the gold standard for medical evidence. However, in light of emerging methodologies in data science, many experts question the role of RCTs. Within this context, experts in the USA and Canada came together to debate whether the primacy of RCTs as the gold standard for medical evidence, still holds in light of recent methodological advances in data science and in the era of big data. The purpose of this manuscript, aims to raise awareness of the pros and cons of RCTs and observational studies in order to help guide clinicians, researchers, students, and decision-makers in making informed decisions on the quality of medical evidence to support their work. In particular, new and underappreciated advantages and disadvantages of both designs are contrasted. Innovations taking place in both of these research methodologies, which can blur the lines between the two, are also discussed. Finally, practical guidance for clinicians and future directions in assessing the quality of evidence is offered.

Could Insulin Be a Better Regulator of Appetite/Satiety Balance and Body Weight Maintenance in Response to Glucose Exposure Compared to Sucrose Substitutes? Unraveling Current Knowledge and Searching for More Appropriate Choices

“…Analogously, beneficial effects were noted in randomized, controlled surveys, whereas longitudinal cohorts showed harmful effects [ 93 ]. The justification for the above deviation among longitudinal cohorts and randomized, controlled surveys seems to mainly be ascribed to the controls [ 94 ]. Regarding longitudinal cohorts, the enrolled individuals lived freely, and further variables were included in the randomized, controlled surveys [ 94 ].…”

“…The justification for the above deviation among longitudinal cohorts and randomized, controlled surveys seems to mainly be ascribed to the controls [ 94 ]. Regarding longitudinal cohorts, the enrolled individuals lived freely, and further variables were included in the randomized, controlled surveys [ 94 ]. The above findings were compared using two very different strategies [ 93 , 94 ].…”

“…Regarding longitudinal cohorts, the enrolled individuals lived freely, and further variables were included in the randomized, controlled surveys [ 94 ]. The above findings were compared using two very different strategies [ 93 , 94 ].…”

To Error Is Human, to Control Is Divine!

“…The difficulty of obtaining sufficiently large sample sizes in rehabilitation research has been noted, and several solutions have been offered. 11,12 Other authors argue the merits of small sample studies with alternative analytical methods that are less reliant on sample size for statistical power. 13 Multisite studies and multicenter collaborative studies can and have produced adequately powered multivariate rehabilitation intervention studies.…”

“…In study after study, decade after decade, study conclusions continue to read “Further studies with larger sample sizes are needed.” We know this is a persistent problem but we haven’t solved it, and this chronic failure to produce adequately powered studies continues to limit the development of evidence that could improve clinical practice. The difficulty of obtaining sufficiently large sample sizes in rehabilitation research has been noted, and several solutions have been offered 11,12. Other authors argue the merits of small sample studies with alternative analytical methods that are less reliant on sample size for statistical power 13…”