I am working on revising a paper that currently has the title “FDA-Mandated “Gold Standard” RCTs Hike Costs and Block Cures.” In the paper I state that a ubiquitous assertion is that “the gold standard” method for learning the safety and efficacy of a medical treatment is a randomized controlled trial (RCT). I have accumulated many examples to support my statement, but to save space in the paper, I am going to list the examples in this blog entry. As time permits, I will update this list as I encounter added examples.
This matters because I believe that calling RCTs “the gold standard,” functions to shut down any discussion of whether any other methods, such as well-done observational studies, might be cheaper, more ethical, or even more sound, than RCTs. (Calling RCTs “the gold standard” serves to locate such a discussion outside the Overton window of politely acceptable discourse.) Arguments and evidence are thus ignored that deserve to be heard and pondered.
Here are a few quotations supporting my claim that RCTs are ubiquitously viewed as the “gold standard” of medical research.
“Generations of medical students have been taught, quite rightly, that the double-blind, randomized [sic], placebo-controlled trial is the gold standard of evidence for new treatments” (Ritchie 2020, 110).
“The purists of evidence-based medicine demand data from randomized controlled trials (RCTs) before doing anything. Those trials are the gold standard of medical evidence, . . .” (Attia 2023, 50; italics in original).
“. . . the results of preliminary clinical trials, the gold standard in medicine, are compelling, despite the small sample sizes” (Whiteman 2023, 167).
“. . . a randomised [sic] clinical trial – medicine’s gold standard for sorting genuine treatments from quackery” (Yong 2016, 231).
“It was the only randomized controlled trial, the gold standard test of efficacy in medicine, ever done in iMCD” (Fajgenbaum 2019, 189).
“Some of these papers are randomized controlled trials (RCTs), which are considered “the gold standard” because they randomly separate people into a test and a control group. If the test group gets the effect and the control group doesn’t, it suggests a cause because the test food is the only difference” (Williams 2021, 204).
“The randomized controlled trial is the gold standard for evidence in clinical medicine” (Prasad and Cifu 2015, 119).
“First devised in the late 1940s (in a study of tuberculosis treatments), the RCT is arguably the most important medical technology of the 20th century. When large and well designed, it is the most powerful way to elucidate the truth in all of medical science. And though it cannot tell you about the mechanism of how a treatment works biologically, it can show conclusively whether a given intervention accomplishes a given goal. In medicine, it is the gold standard for proving a claim” (Prasad and Cifu, 2015, 124).
Without providing you the passages, here are some additional sources that support the ubiquity of the claim that RCTs are the “gold standard” of medical research: Bothwell et al. (2016); Concato and Horwitz (2018); Cook (2018); Hariton and Locascio (2018); Jones and Steel (2018); Mowat et al. (2018).
The full citations for the publications briefly cited above are:
Attia, P. 2023. Outlive: The Science and Art of Longevity. Harmony.
Bothwell, L. E., J. A. Greene, S. H. Podolsky, and D. S. Jones. 2016. “Assessing the Gold Standard — Lessons from the History of RCTs.” New England Journal of Medicine 374, no. 22 (June 2): 2175–81.
Concato, J., and R. I. Horwitz. 2018. “Randomized Trials and Evidence in Medicine: A Commentary on Deaton and Cartwright.” Social Science & Medicine 210 (Aug.): 32–36.
Cook, T. D. 2018. “Twenty-Six Assumptions That Have to Be Met If Single Random Assignment Experiments Are to Warrant “Gold Standard” Status: A Commentary on Deaton and Cartwright.” Social Science & Medicine 210 (Aug.): 37–40.
Fajgenbaum, D. 2019. Chasing My Cure: A Doctor’s Race to Turn Hope into Action; A Memoir. Ballantine Books.
Hariton, Eduardo, and Joseph J. Locascio. 2018. “Randomised Controlled Trials – the Gold Standard for Effectiveness Research: Study Design: Randomised Controlled Trials.” BJOG: an international journal of obstetrics and gynaecology 125, no. 13 (Dec.): 1716–16.
Jones, A., and D. Steel. 2018. “A Combined Theoretical and Empirical Approach to Evidence Quality Evaluation: A Commentary on Deaton and Cartwright.” Social Science & Medicine 210 (Aug.): 74–76.
Mowat, Ryan, S. V. Subramanian, and Ichiro Kawachi. 2018. “Randomized Controlled Trials and Evidence-Based Policy: A Multidisciplinary Dialogue.” Social Science & Medicine 210 (Aug.): 1.
Prasad, V. K., and A. S. Cifu. 2015. Ending Medical Reversal: Improving Outcomes, Saving Lives. Johns Hopkins University Press.
Ritchie, S. 2020. Science Fictions: How Fraud, Bias, Negligence, and Hype Undermine the Search for Truth. Metropolitan Books.
Whiteman, N. 2023. Most Delicious Poison: The Story of Nature’s Toxins—from Spices to Vices. Little, Brown Spark.
Williams, R. A. 2021. Fixing Food: An FDA Insider Unravels the Myths and the Solutions. Post Hill Press.
Yong, E. 2016. I Contain Multitudes: The Microbes within Us and a Grander View of Life. Ecco.