Clinical Trials Test Safety, Efficacy, and Effectiveness of Drugs

Robert Gadimian is a regulatory professional with a quarter of a century’s worth of experience in hands-on drug development. Perceptive Regulatory Consulting, Robert Gadimian’s company, helps drugs and related products get approved by the Federal Drug Administration (FDA) by meeting clinical trials requirements post-Investigational New Drug (IND). His company helps make sure the drugs reach the safety, efficacy, and effectiveness benchmark.

After a drug has gotten an IND status, this means that the FDA has approved it to go into clinical trials. The first thing is to demonstrate the safety observed on animal test subjects and a small number of humans. In Phase I clinical trials, the purpose is to “determine dosing, document how a drug is metabolized and excreted, and identify acute side effects.” The Phase II and Phase III clinical trials gather data on the drug’s efficacy and effectiveness in larger and more varied groups of people with a particular condition of interest.

Safety, efficacy, and effectiveness are the three salient elements in the drug-making and regulatory process. Safety is usually measured scientifically by toxicity to discover the overdose line or the optimal dose. The safety test discovers any potential adverse effects. Efficacy represents the health benefit of the drug, while effectiveness refers to other factors. Factors may affect efficacy, such as insufficient dose, duration of use, and interactions with other drugs.