Randomized Controlled Trials and Naturalistic Data: Time for a Change?

Abstract

Current medical research is over-dependent upon the randomized controlled trial (RCT). There are limitations to this approach and an exclusive reliance upon RCTs at the expense of more naturalistic observation impedes development in clinical understanding. Many questions concerning a drug's usefulness cannot be answered using standard RCT design. It is proposed that the existing four-phase drug-development design be supplemented with naturalistic studies at the phase 4 stage in order to evaluate a drug's true performance in its eventual setting. Naturalistic studies are ideally placed to generate new hypotheses which can then be examined further using RCT design.