Basic Science
According to the FDA's Protecting America's Health Through Human Drugs, the drug development process begins with basic science research. Basic science is different than clinical research because it focuses on the activity of specific proteins, genes, or cells. Often this work starts with researchers trying to understand how the human body works or how certain diseases progress. As researchers gain a better understanding about some of the underlying processes behind diseases, they can see if changing the body's biological chemistry can affect how these diseases progress or how they come to pass in the first place. Researchers looking to make a new drug will then test out compounds to see what effect they have on proteins or cells to see if they could be useful in treating a disease.
Animal Models
The FDA notes that once a compound has been identified as being potentially useful as a drug, the next step is to try the drug out in animal models. This is because some compounds work differently when they are taken from controlled cell or protein experiments and are given to animals. Researchers make every effort to use as few animals as possible, but often have to try out their drug on more than one animal specie because some drugs work differently in different animals. These animals may be genetically modified or be raised in conditions that allow their bodies to mimic those of humans with the disease for which the drug could be used. The purpose of this stage in the drug development process is to show that the target drug can be of use in treating disease in whole animals without causing significant toxicity.
Human Testing
If a compound makes it through animal testing and is still considered to be a potentially useful drug, the next part of the process is testing in humans. According to the Pharmaceutical Product Development company, testing in humans (often referred to as clinical testing) has three phases. In the first phase healthy human volunteers are given the medication take the compound to evaluate its safety and side effects. These people typically take the drug for a short period of time and their overall health is monitored carefully. Physicians will also examine how the drug is absorbed and distributed throughout the body, as well as how it is metabolized and secreted. Phase II studies involve giving the drug to patients who have the disease for which the drug would be used to determine if it can effectively treat humans. This phase gives effectiveness and safety information for the drug and typically six months to three years. If a drug passes phase II studies, it moves onto phase III studies, which are essentially expansions of the phase II studies. Both phase II and phase III studies are usually performed with patients being randomly assigned to one of two groups: one that receives the investigational drug and one that does not. The results from the two patients are then compared.
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