Randomized controlled trials (RCTs) are the most rigorous way of determining whether a cause-effect relation exists between treatment and outcome and for assessing the cost-effectiveness of a treatment. For many patients, cancer is a chronic illness; RCTs evaluating treatments for indolent cancers must evolve to facilitate medical decision-making, as "concrete" patient outcomes (eg, survival) will likely be excellent independent of the intervention, and detecting a difference between trial arms may be impossible. In this commentary, we articulate 9 recommendations that we hope future clinical trialists and funding agencies (including those under the National Cancer Institute) will take into consideration when planning RCTs to help guide subsequent interpretation of results and clinical decision making, based on RCTs of external beam radiation therapy dose escalation for the most common indolent cancer in men, that is, prostate cancer. We recommend routinely reporting: (1) race; (2) medical comorbidities; (3) psychiatric comorbidities; (4) insurance status; (5) education; (6) marital status; (7) income; (8) sexual orientation; and (9) facility-related characteristics (eg, number of centers involved, type of facilities, yearly hospital volumes). We discuss how these factors independently affect patient outcomes and toxicities; future clinicians and governing organizations should consider this information to plan RCTs accordingly (to maximize patient accrual and total n), select appropriate endpoints (eg, toxicity, quality of life, sexual function), actively monitor RCTs, and report results so as to identify the optimal treatment among subpopulations.
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American journal of clinical oncology. 2016 Jun 17 [Epub ahead of print]
Nicholas G Zaorsky, Brian L Egleston, Eric M Horwitz, Adam P Dicker, Paul L Nguyen, Timothy N Showalter, Robert B Den
*Department of Radiation Oncology, Fox Chase Cancer Center †Department of Radiation Oncology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA ‡Department of Radiation Oncology, Brigham and Women's Hospital, Boston, MA §Department of Radiation Oncology, University of Virginia Health System, Charlottesville, VA.