Bringing New Medication to Market

FDA drug approval process involves extensive research and testing including four phases of clinical trials

/ Author:  / Reviewed by: Robert Carlson, M.D Beth Bolt, RPh

Whether it’s an advertisement for sleep medication, bladder control or seasonal allergies, all medications share one thing in common — extensive research and testing had to be done before they were put on the market.

According to the US Food and Drug Administration (FDA), a drug is a product intended to diagnose, treat, cure or prevent disease. Any product falling under this category must be reviewed by the Center for Drug Evaluation and Research (CDER) to ensure that it lives up to its stated claims and is safe for use.

In the CDER’s review, the drug company that has developed a drug must submit the results of all of the tests they’ve performed.

Initial Review

First, the drug has to be tested in animals. This allows basic information to be obtained about the safety and effectiveness of the drug.

Once the drug has been tested in multiple animal species, the drug company must submit an Investigational New Drug (IND) application to FDA that includes the composition of the drug, their results from the animal studies, and their plans for testing the drug on humans.

Once the FDA approves the IND application, then the drug trials in humans can begin.

Four Phases of Clinical Trials

The first phase of drug trials involves a small number of healthy volunteers, typically between 20 and 80 individuals. The main goal of this phase is to evaluate the safety of the drug as well as determine an appropriate dosage and identify potential side effects.

The second phase of trials involves a larger group of individuals, typically in the hundreds. The primary goal of this phase is to determine the effectiveness of the drug. To do this, researchers recruit individuals who have the disease of interest.

In some cases, patients who receive the drug are compared to patients with the same disease who receive a different medication or a placebo (fake medication). Safety and side effects continue to be monitored and evaluated in this phase.

The third phase involves the largest amount of participants, typically in the thousands. In this phase, researchers gather more data about the effectiveness and safety of the drug. Researchers are also able to study the effects of the drug in different sub-populations to see if these effects vary from group to group.

The Drug Approval Process

Once these three phases have been completed, the drug company submits a New Drug Application (NDA) to the FDA. The NDA includes the findings from all of their studies.

The CDER has a team of doctors, statisticians, chemists, pharmacologists and other scientists review the application and the findings from the research studies.

The labeling for the drug is reviewed to make sure it is accurate and complete for consumers and healthcare providers, and the facility where the drug will be produced is inspected.

After everything has been reviewed, if it is found that the benefits of the drug outweigh the risks, then the drug is approved for sale. 

The fourth and final phase of the drug trial doesn’t happen until after the drug has been released. Once the drug is available to consumers, the FDA monitors its use for any potential complications or adverse reactions (negative or harmful side effects).

This process is not a quick one, and according to Sandra Kweder, MD, FACP, director of the Office of New Drugs in CDER, clinical trials are at least part of the reason. In a statement on the FDA website, Kweder noted, “It's the clinical trials that take so long — usually several years.”

There are some products, however, that are exempt from this extensive review. One of the most notable being dietary supplements. Steve Leuck, President of AudibleRX, a company that provides patient medication education and counseling, commented that, "Based on the Dietary Supplement Health and Education act of 1994, a company does not need to obtain approval from the FDA showing that a product does what it says it does."

In these instances the product should include a statement that notes that it has not been evaluated by the FDA and that it is not intended to diagnose, treat, cure, or prevent any disease.

The FDA can get involved after the product has been released if problems are found with the product or its labeling. Dr. Leuck noted that the FDA would need to " for products being marketed, test them, and then force them off the market if they are proved to be advertised inappropriately."

Review Date: 
June 13, 2014
Last Updated:
June 23, 2014