What is a clinical trial?
Before a drug can be used in clinical practice, pharmaceutical and biotechnology companies put the drug through strenuous testing to ensure both its safety and efficacy. Drug candidates are studied pre-clinically, both in the laboratory and in animals, prior to first human administration. If the drug appears to be safe and effective in animal and laboratory models, then clinical testing in humans may begin.
There are three phases in clinical trial development. The first phase is a Phase I study. The main objective in this type of study is to determine if the drug is safe, well tolerated and what dose should be used, and only a small number of healthy volunteers or patients are treated. If results from the Phase I study are positive, then a Phase II study may begin. A larger number of patients who have the targeted disease are included in a Phase II study, which measures efficacy in addition to safety. Sometimes, an initial assessment of the drug’s effect is included in the Phase I study, in which case the term ‘PhaseI/II’ may be applied. Phase III studies contain a larger number of patients and are used to further establish efficacy. If positive results are achieved, the biotech or pharma company may apply for approval to market the drug with the appropriate drug regulation organisation, such as the Food and Drug Administration (FDA) in the United States or the European Medicines Agency (EMA) in Europe. If approved, the companies can then produce and sell the product to the public.
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