This past week in lab, I had an interesting discussion with some other lab members about the process behind drug discovery. Because I am not very familiar with the steps necessary for drugs to progress from development to production, I thought I would use this blog to explore what needs to happen in order for a drug to be approved by the FDA.

The Food and Drug Administration (FDA) was founded in the 1920s, and its primary purpose is to monitor public health in the United States by overseeing food safety and prescription & over-the-counter medications. Here is a quick overview of the general process. The obvious first step in drug development is that a molecule or agent has to be identified for its potential to treat a disease or illness. Traditionally, this first step starts in research labs where scientists isolate the drug of interest and test it for its effectiveness. Testing can include in-vitro studies, in which the effects of the drug are studied on bacteria or mammalian cells cultured in petri dishes. Testing can also involve in-vivo studies, in which live animals, such as mice, are treated with the drug. After successful lab testing is done, and the drug is deemed to be safe and effective, clinical studies and trials in human volunteers can begin. Clinical trials typically take place in three phases. In Phase 1 clinical trials, the drug is given to only a few human subjects and the primary purpose of this phase is to find the safest doses of the drug. Approximately 70% of drugs pass this phase. Phase 2 clinical trials involve even more participants, and the main purpose of Phase 2 is to determine whether any severe side effects are produced by the medication. Only about 30% of drugs pass this phase. Finally, in Phase 3 clinical trials, the drug is used in double-blind, placebo control trials, so that neither the scientists nor the patients know if they are getting the drug or a placebo. Only drugs that pass through this rigorous phase are submitted for FDA approval.

After processing through various stages of clinical trials, a company or lab would appeal to the FDA and submit a new drug application (NDA). Following FDA review of the studies and results involving the drug, which can take several months, the drug can finally be approved for marketing and sale.