Common misconceptions of randomized controlled trials in oncology

Abstract

In biomedicine, randomized controlled trials are regarded as the gold standard of evidence owing to their ability to minimize confounding factors that may influence results. Randomized trials that are properly designed serve as a basis for drug regulation and national guideline development. Despite the many advantages of the study design, there are several misconceptions regarding randomized trials, particularly in oncology. These misconceptions include: the difficulty of designing and conducting a trial, the length of time necessary to complete a trial, the expense, appraisal and critique, pharmaceutical industry influence, and ethical standards. Furthermore, developing regulatory and strategic frameworks has the potential to enhance the randomized trial landscape. Such initiatives will focus on relevant clinical issues that persist in oncology, reducing duplicative and unethical trials and maximizing value‐based healthcare. Here, we address several misconceptions regarding randomized controlled trials and provide potential solutions to enhance their methodology and implementation.

Insurance Denial of Care for Randomized Controlled Trial-Eligible Patients

“…(Am J Clin Oncol 2024;47:56-57) W ell-designed, ethically conducted prospective randomized clinical trials remain the primary means to evaluate medical interventions for patient care, providing Level 1 evidence due largely to their ability to minimize confounding factors that can influence results in lower-quality levels of evidence. 1,2 For patients eligible to receive care on a clinical trial, insurance denial of therapy serves as a pertinent barrier for patients to remain trial-eligible, thus hindering the development of therapies and the overall advancement of health care. Furthermore, insurance denials increase the risk of bias and loss of statistical power for clinical trials in which such denials are not infrequent.…”