The label academic research organization (ARO), used broadly within the clinical and drug development industries, primarily refers to an academic and/or nonprofit institution that performs one or more functions in the conduct of clinical trials. The services that an ARO provides can range from academic leadership to full-service clinical trial management capabilities, including site monitoring, data management, statistical analysis, safety monitoring, and clinical events classification, in addition to clinical expertise.

The concept of an ARO dates back several decades, when researchers recognized the need for large global clinical trials to answer important medical questions. Clinical scientists from several of the world’s leading academic institutions formed teams of like-minded investigators with the goal of developing and conducting global clinical studies to improve patient care. AROs are focused on developing and sharing knowledge with the end goal of improving patient care. They accomplish this goal not only by leading and conducting multinational clinical trials but also by ensuring that the results from these trials are published and presented. These groups also focus on managing major national patient registries designed to collect data and determine best practices, which can then be incorporated into clinical practice guidelines. Education and development of clinical investigators is also a focus, with many of the leading AROs having fellowship programs whose influence extends around the globe.

The concept of the ARO dates back to the late 1980s, when several groups of physician investigators first came together to address unmet clinical needs by organizing and centralizing the operational efforts associated with conducting large multicenter clinical trials, so-called “megatrials.” The first such group, located at the University of Oxford in the United Kingdom, developed in 1975 from a single research team. It began enrollment of the then-largest trial in history—6,027 patients with suspected acute myocardial infarction (MI)—in mid-1981, completed enrollment at the 245 participating coronary care units in 1985, and published the results the next year (1). Similarly, the Gruppo Italiano per lo Studio della Sopravvivenza nell’Infarto Miocardico (GISSI), formed from a collaboration between the Mario Negri Institute and the Associazione Nazionale dei Medici Cardiologi Ospedalieri, began enrolling 11,806 patients with acute MI in 176 Italian intensive care units in 1984. It published the study results 2 years later (2). Both groups have since continued to perform large, important multicenter trials.

Two groups in particular have paved the way for what we now know as AROs. The Thrombolysis In Myocardial Infarction (TIMI) study group, based in Boston, Massachusetts, and affiliated with Brigham and Women’s Hospital and Harvard Medical School, was among the first groups to take on the challenges associated with organizing and implementing global clinical trials (3). The TIMI series of trials began in 1984, initially sponsored by the National Heart, Lung, and Blood Institute (NHLBI). At first, the trials studied thrombolytic and antithrombotic therapies in patients with acute MI and unstable angina, but they now cover many aspects of cardiovascular disease.

Around the same time, other groups of academic investigators were beginning to organize their efforts to address important clinical questions, and thought leaders from around the world formed an alliance to facilitate the conduct of pivotal cardiovascular clinical trials. The VIrtual Coordinating Centre for Global CollabOrative CardiovascUlar Research (VIGOUR) is a working group of global thought leaders in cardiovascular clinical medicine who have achieved academic and industry recognition for performing large multinational clinical trials. The alliance developed from a group of academic investigators participating in the international Global Utilization of Streptokinase and TPA (alteplase) for Occluded Coronary Arteries (GUSTO-1) trial, which began enrollment in 1990 (4). VIGOUR members have since conducted many large international trials, all according to the group’s philosophical perspectives on clinical trial design and methods (5). The VIGOUR organization is committed to improving the practice of cardiovascular medicine and patient outcomes (6).

During the 1990s and into the new millennium, many more now-prominent AROs began to formalize their efforts to lead the development of new therapeutics and shape the future of clinical practice across the globe. Table 3–1 lists some of the major AROs currently leading clinical trials (7), and Figure 3–1 displays their global distribution.