Editorial: The Vital Role of Randomized Controlled Trials in Arthritis & Rheumatology

In this issue of Arthritis & Rheumatology (A&R), Sieper and colleagues report the results of an important trial demonstrating the benefits of a tumor necrosis factor (TNF) antagonist for the treatment of nonradiographic spondyloarthritis (1). As optimal treatment for this condition evolves, well-designed randomized controlled trials (RCTs) will play a pivotal role in defining the standard of care for one of the more common inflammatory arthritides. We highlight this study not only because of its importance to rheumatology but also because of the vital role of RCTs in A&R. Throughout the 57-year history of A&R, RCTs have always played a prominent role. Major advances in our understanding of treatment, pathobiology, the mechanisms of action of a treatment, and new avenues of scientific investigation are facilitated by RCTs. Some of the major RCTs published in A&R throughout the many decades document the roles of TNF antagonists in rheumatoid arthritis (2,3), cyclophosphamide and belimumab in systemic lupus erythematosus (4,5), nonpharmacologic therapies in osteoarthritis (6,7), novel therapies in gout (8,9), and various treatments in vasculitis (10,11) With this issue, which begins the tenure of the new editorial team, we rededicate Arthritis & Rheumatology to the goal of publishing the most important RCTs in our field. The importance of RCTs in A&R is reflected by a new section of the journal that is dedicated to RCTs, with one of us (MEW), a senior clinical trials investigator, as the section advisor. As with the RCT published in the current issue (1), A&R will now offer fast-track reviews. For example, the total review time for the article by Sieper and colleagues (1), from submission to acceptance, was 20 days. Authors of RCTs desiring a fast-track review need to contact the Editor in Chief (RJB) or Deputy Editor (DHS) at least 14 days prior to submission to facilitate such a review. The new section will focus on phase I, phase II, and phase III RCTs. A phase III RCT using approved drugs for new indications, such as that reported by Sieper and colleagues, will also be considered for this section and for fast-track review. Nonrandomized phase IV studies will be published in other sections of the journal, and phase I and phase II studies will typically not receive fast-track status, except under circumstances in which the findings are exceptionally noteworthy. In addition, A&R embraces the important role of effectiveness studies in the current research milieu. Efficacy studies (how interventions work in idealized settings, such as an RCT) and effectiveness studies (how interventions work in typical settings, such as registries and pragmatic controlled clinical trials) contribute complementary and critical information for determining best practice. RCTs with negative results also provide important information for drug developers, providers, and patients that will be embraced by this journal. Dr. Solomon’s work was supported in part by research grants to Brigham and Women’s Hospital from Amgen, Lilly, Pfizer, AstraZeneca, and CORRONA. Daniel H. Solomon, MD, MPH (Deputy Editor, A&R), Michael E. Weinblatt, MD (Clinical Trials Advisor, A&R): Brigham and Women’s Hospital, Boston, Massachusetts; Richard J. Bucala, MD, PhD (Editor in Chief, A&R): Yale University School of Medicine, New Haven, Connecticut. Dr. Weinblatt has received consulting fees from AbbVie, Ablynx, Amgen, AstraZeneca, Bristol-Myers Squibb, Can-Fite BioPharma, Catabasis, Compugen, CORRONA, Crescendo Bioscience, Ensemble Therapeutics, Exagen Diagnostics, Five Prime Therapeutics, Genentech/Roche, Idera, Infinity, Lycera, Lilly, MedImmune, Merck, Novartis, Pfizer, Principia Biopharma, Receptos, Regeneron, Samsung, Sanofi, and UCB (less than $10,000 each) and grants from Bristol-Myers Squibb, Crescendo Bioscience, and UCB. Dr. Bucala is a named inventor on, and has received royalties from, licensed patents in the area of macrophage migration inhibitory factor therapeutics and owns equity in Promedior and MIFCOR. Address correspondence to Daniel H. Solomon, MD, MPH, Division of Rheumatology, Immunology, and Allergy, Brigham and Women’s Hospital, 75 Francis Street, PBB-B3, Boston, MA 02115. E-mail: dsolomon@partners.org. Submitted for publication June 15, 2015; accepted in revised form June 18, 2015.