The United States Food and Drug Administration (USFDA) must approve new medications for use before they are sent to the market and prescribed by doctors. But in order for this approval to take place, the drugs are tested for both safety and efficacy. Human volunteers are essential to evaluate the drug safety and the effectiveness of the drug. But this doesn’t mean that people must risk their health and safety in these trials.
Earlier phases of human study on the investigational medication involve careful testing and retesting for safety. Later stages of human testing also evaluate safety and efficacy of the investigational medication/device.
Often, the investigational drug will be compared to a placebo, meaning a pill that has no active drug ingredients. This way, researchers can measure how well the new medication works compared to no treatment. In many cases, the participants won’t know if they are receiving the placebo or the drug, which is important for the integrity of the study.
While investigational drugs are tested for safety before human testing, there are always potential risks involved. Those wanting to participate in a clinical study should be well aware of these risks, and potential side effects. All is described in the study information and Informed Consent Form.
Many people take part in trials of this type as it is one manner to obtain novel forms of medication that are not available otherwise, especially if one has tried approved medications without efficacy. For example, if there is an investigational drug for osteoarthritis, a person may want to try the study drug because other currently approved medications don’t work well.
Many of these trials for investigational or experimental drugs require certain participant characteristics. Often, participants must be dealing with a particular health issue, or have recurring problems of a specific kind.