Clinical Trials: Cornerstone of Biomedical Research and Innovation

The origins of the clinical trial can be traced to the rational mindset that grew from scientific advances during the 18th century Age of Enlightenment and gave rise to the Industrial Revolution. Now, more than two centuries later, a new technological revolution in genomics, proteomics and information technology promises to transform the practice of medicine. Clinical trials still remain the best way to determine the safety, efficacy and societal value of innovative treatments.

Facts and fallacies about clinical trialsThe success of a clinical trial hinges on the researchers' ability to recruit enough suitable patients in an ethical manner so that statistically meaningful results can be obtained within a reasonable time frame. Investigators must often find approaches to make sure of obtaining fully informed consent from trial participants of varying cultural and educational backgrounds. In some cultures, investigators must respect traditions of collective versus individual decision making. Involvement of community leaders at the planning stage can help to avoid misunderstandings and encourage cooperation in the conduct of the trial. Outreach to enroll members of minorities has made trial cohorts more representative of the communities where treatment is required. Page 19

What is a clinical trial?Health care providers once relied on their early training in making clinical decisions, whereas approaches to patient care today are increasingly prompted by treatment guidelines and consensus statements based on evidence from clinical trials. The current approach is termed evidence-based medicine. Randomized, controlled trials, accepted as the gold standard for clinical investigation, are used to confirm safety and efficacy of interventions in humans after basic information has been obtained from exploratory studies. Patients and control subjects are matched, as far as possible, for demographic characteristics, to ensure that the results can be extrapolated to patient care in the community. Page 10

When the trial is overTrial sponsors base global strategies on the principle that participants should be left no worse off as a result of taking part and that there is an obligation to provide participants with the benefits that follow from a successful trial. Although trial follow-up should be addressed at the planning stage, questions of whether a treatment should be provided afterward depend on post trial conditions, including the outcome of treatment, national regulatory processes and infrastructure and the nature of the disorder at issue. Regardless of whether treatment continues, research in and of itself is beneficial, helping to develop capability for the benefit of the local population at the research site and in the host country. Page 32

Looking to the futureThrough clinical trials conducted on a global scale, responsible pharmaceutical companies are developing links with public health communities worldwide, providing important educational resources to developing countries and helping to bridge gaps in health care. Page 36