The efficacy and effectiveness of drugs for diabetes: how do clinical trials and the real world compare?

Excerpt

For many years, the randomised controlled clinical trial (RCT) has been the gold standard for evaluating the efficacy, tolerability and safety of drugs. The modern framework for RCTs was developed in the mid-20th century through the leadership of the UK Medical Research Council and the US National Institutes of Health in collaboration with academic clinical researchers and scientists, among others [1]. Adoption of the RCT by regulatory agencies, including the US Food and Drug Administration (FDA) and the European Medicines Agency, as a requirement for the approval of most new drugs significantly shifted the clinical trial landscape. Today, the majority of patients who participate in RCTs do so in studies sponsored by pharmaceutical and device companies. The advantages of RCTs are well known to most clinical researchers and clinicians. Random assignment to treatment minimises allocation bias, that is, the chance that known and unknown systematic differences between treatment groups affect the outcomes. Masking of treatment assignment when possible ensures that any conscious and unconscious biases of investigators and participants do not alter outcomes. Finally, analysing responses according to treatment assignment (the intention-to-treat principle) minimises the chance that differential follow-up biases the results. …