What Does it Take to Get a Medication Approved Through the FDA?

Do you pick up prescription medications from a pharmacy monthly? You’re in good company: about 66% of adults and about 20% of children in the United States take prescription medications. You may wonder how prescription medications become approved for use. Medications are ultimately approved through the United States Food and Drug Administration (FDA). It can be a long and expensive process to gain FDA approval for a new medication.

The first step in creating a new medication generally begins with finding a gene or protein that plays a role in a specific disease or medical condition. That gene or protein then becomes the therapeutic target for the new medication. Once a new potential medication is identified it will be tested in a laboratory to ensure its safety. This is done long before the medication ever makes its way to human use.

If the laboratory assessments support the safety of the medication, the researcher may submit an Investigational New Drug (IND) application to the FDA to request approval to begin human studies. Each year, the FDA reviews about 1,500 IND applications. In addition to the FDA review, a local Institutional Review Board must approve a request for clinical trials. The researcher can begin clinical trials in humans after approval.

• Phase 1 trials are conducted in approximately 20-100 healthy volunteers or people with the disease being targeted. The purpose of a Phase 1 trial is to determine medication safety and dosing. About 70% of medications will move on to Phase 2 trials.
• Phase 2 trials are conducted in several hundred volunteers who have the disease or medical condition to assess medication safety, performance, and dosing. About 33% of medications will move on to Phase 3 trials.
• Phase 3 trials are conducted in 300-3,000 volunteers who have the disease or medical condition to further assess safety, performance, and side effects. About 25% of medications will perform well enough in Phase 3 trials to move forward in the FDA approval process.

Following successful completion of a Phase 3 trial, a New Drug Application (NDA) can be submitted to the FDA to request review for approval. The FDA will then take 6-10 months to review all the data submitted and either accept or deny the NDA. Once a medication is approved, the FDA requires the manufacturer to continue to monitor safety of its drug. This ensures that any serious or rare side effects that may not have occurred in the clinical trials can be evaluated.

On average, it takes ten years to get a new medication approved by the FDA. Only about ten percent of potential drugs make it through the rigorous process to become FDA approved.

Sometimes new medications, including vaccines, for which there are no alternatives are needed in a public health emergency, and full FDA approval would take too long. In this case, the FDA can use its Emergency Use Authorization (EUA) authority to allow non-FDA approved medications to be used when certain criteria are met.

The FDA requires safety and effectiveness data to review an EUA. The FDA reviews medical records, site visits for manufacturing facilities, previous compliance history, and compliance with good manufacturing practices to ensure safety post-EUA. The FDA looks at the risk versus the benefit of waiting for all the evidence needed for full approval. The FDA does not allow the use of a test, medical supply, or medication if they do not think it is safe.

In addition to the EUA, there are other special approaches to drug review that can shorten the time it takes to get a medication FDA-approved.

• Fast Track and Breakthrough Therapy designations are used to accelerate the development and review of medications that treat an unmet medical need or provide a sizeable improvement over available treatment options.
• Accelerated Approval designation allows medications that treat an unmet medical need for serious conditions to be approved based on less rigorous data than is usually needed for FDA-approval.
• Priority Review designation means the FDA will aim to provide a decision on an NDA within 6 months, as opposed to the 6-10 months that it may otherwise take.

As with EUA, in these special situations the FDA still follows detailed procedures to ensure a medication or vaccine is safe and effective, before approving its use. The FDA website has more information on the FDA approval process and Emergency Use Authorization.