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

If you pick up a prescription medication from the pharmacy each month, you’re not alone. About 66% of adults and about 20% of children in the United States take prescription medications. You may have wondered how a prescription medication becomes approved for use. Medications are ultimately approved through the United States Food and Drug Administration (FDA). The process to gain FDA approval for a new medication can be long and expensive.

Generally, the first step in creating a new medication begins with finding a gene or protein that plays a role in a specific disease or medical condition. The 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. After approval, the researcher can begin clinical trials in humans.

• 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 of 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.

Sometimes new medications for which there are no alternatives are needed for an emergency situation, such as 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. 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.

On average, it takes ten years and hundreds of millions of dollars 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. Despite these challenges, researchers remain hard at work to develop new safe and effective medications.