Central Problem is Equipoise
Randomized controlled trials (RCTs) are the gold standard for clinical research, as the authors point out (1). Informed consent as well as consent to randomization are the ethical premises for most RCTs (2). Designs that completely eliminate informed consent are the low-risk pragmatic RCTs and the so-called prompted optional randomization trials (PORT).
A central problem in the ethics of clinical research is equipoise (3). Among other things, this dilemma explains why currently only 3–5% of potential patients are included in RCTs.
Interestingly, in the United States from 1973 to 2006, 68 anticancer drugs were approved by the FDA without evidence from RCTs. A further problem in oncology are negative studies as well as phase II or phase III studies that were discontinued or failed. Indeed, from 2013 to 2015, 32% of 5821 RCTs were discontinued or failed.
Additional references available by request to the author.
Dr. med. Antonis G. Tsamaloukas
Conflict of interest statement
The author declares that no conflict of interest exists.
|1.||Lange S, Sauerland S, Lauterberg J, Windeler J: The range and scientific value of randomized trials—part 24 of a series on evaluation of scientific publications. Dtsch Arztebl Int 2017; 114: 635–40 VOLLTEXT|
|2.||Grady C, Cummings SR, Rowbotham MC, McConnell MV, Ashley EA, Kang G: Informed consent. N Engl J Med 2017; 376: 856–67 CrossRef MEDLINE|
|3.||Joffe S, Miller FG: Equipoise: asking the right questions for clinical trial design. Nat Rev Clin Oncol 2012: 9: 230–5 CrossRef MEDLINE|