Evidence for Health Decision Making — Beyond Randomized, Controlled Trials

Frieden, Thomas R.

doi:10.1056/nejmra1614394

Cited by 609 publications

(522 citation statements)

References 78 publications

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“…Randomised controlled trials are generally regarded as the highest quality source of data on the effects of medical treatment 20. Early randomised controlled trials showed that thiopurines have a steroid-sparing effect in UC and are effective in maintaining clinical remission 21–24.…”

Section: Discussionmentioning

confidence: 99%

Impact of thiopurines on the natural history and surgical outcome of ulcerative colitis: a cohort study

Eriksson

Rundquist

Cao

et al. 2018

Gut

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Section: Discussionmentioning

confidence: 99%

Impact of thiopurines on the natural history and surgical outcome of ulcerative colitis: a cohort study

Eriksson

Rundquist

Cao

et al. 2018

Gut

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“…HRT had previously been supported by positive results from a highquality and long-running observational study, but the RCT was stopped in the face of excess deaths in the treatment group. The negative result of the RCT led to widespread abandonment of the therapy, which might have been a mistake (see Vandenbroucke (2009) andFrieden (2017)). Yet the medical and popular literature routinely states that the RCT was right and the earlier study wrong, simply because the earlier study was not randomized.…”

Section: Misunderstandings: Claiming Too Muchmentioning

confidence: 99%

“…for drug trials by the FDA; a notable exception is the recent paper by Frieden (2017), ex-di-rector of the U.S. Centers for Disease Control and Prevention, who lists key limitations of RCTs as well as a range of contexts where RCTs, even when feasible, are dominated by other methods.…”

Section: Introductionmentioning

confidence: 99%

Understanding and Misunderstanding Randomized Controlled Trials

Deaton¹,

Cartwright²

2016

167

151

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RCTs would be more useful if there were more realistic expectations of them and if their pitfalls were better recognized. For example, and contrary to many claims in the applied literature, randomization does not equalize everything but the treatment across treatments and controls, it does not automatically deliver a precise estimate of the average treatment effect (ATE), and it does not relieve us of the need to think about (observed or unobserved) confounders. Estimates apply to the trial sample only, sometimes a convenience sample, and usually selected; justification is required to extend them to other groups, including any population to which the trial sample belongs. Demanding "external validity" is unhelpful because it expects too much of an RCT while undervaluing its contribution. Statistical inference on ATEs involves hazards that are not always recognized. RCTs do indeed require minimal assumptions and can operate with little prior knowledge. This is an advantage when persuading distrustful audiences, but it is a disadvantage for cumulative scientific progress, where prior knowledge should be built upon and not discarded. RCTs can play a role in building scientific knowledge and useful predictions but they can only do so as part of a cumulative program, combining with other methods, including conceptual and theoretical development, to discover not "what works," but "why things work".

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“…Other aspects of management, including mechanical ventilation conditions, anticoagulation, drug management, catheter maintenance, management of infections, and sedation, also vary. Overall patient management and technology are continuously improving, making it difficult to interpret the results of RCTs, which often take years to design and execute [5]. The suggestion that ECMO should only be conducted in expert centers [6] is also based on a wish to harmonize practice in the context of clinical trials and high-quality research.…”

Section: Variability Among Centersmentioning

confidence: 99%