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

Today is Wear Purple Day, a day that gives us an opportunity to raise awareness about epilepsy – a condition for which there is no cure. Each year, researchers work to develop medications to treat diseases and medical conditions like epilepsy. These medications generally stem from learning how a specific disease or medical condition is triggered, then a medication is created to target and stop these processes from occurring.

First, a medication is tested in the lab to examine its safety. If the medication is successful, the researcher submits an investigational drug application (IND) to the Food and Drug Administration (FDA) to request approval to begin studying this medication in humans. Following initial studies, approximately 5 in 5,000 medications will appear successful enough for a researcher to submit an application to the FDA.

The FDA will review this application and determine if the proposed study appears safe. In addition to the FDA’s approval, a local institutional review board must approve a request for clinical trials. After approval, the researcher can begin the following clinical trials. Each trial must be successful to move on to the next phase.

  1. Phase 1 Trial:
    1. Conducted in approximately 20-80 healthy participants to determine medication safety
  2. Phase 2 Trial:
    1. Conducted in approximately 300 or less participants, who have the disease or medical condition, to assess medication safety and performance as well as establish medication dosing
  3. Phase 3 Trial:
    1. Conducted in approximately 3,000 or less participants, who have the disease or medical condition, to further assess safety, performance and side effects

Following successful completion of the phase 3 trial, the researcher is able to submit a new drug application to the FDA to request review for approval. Once it is submitted, the FDA has 60 days to decide if they wish to file this application to be reviewed or not. After an application is marked for review, the FDA reviews all of the data submitted and has 6-10 months to accept or deny the medication for approval.

Faster approvals are available if the medication is able to meet an unmet medical need for a serious or life-threatening condition. Once a medication is approved, it will be regulated and marketed by the FDA for use by individuals within the general public. The FDA continues to monitor the use of the medication to ensure it remains a safe treatment option for individuals.

The FDA approves medications that have demonstrated safety and capabilities that provide positive benefits and outweigh the potential negative effects, such as side effects; this strives to keep individuals safe while helping them have access to medications that may achieve the best outcomes for their health.

It takes approximately 12 years for a medication to be successfully developed, tested and approved by the FDA, with an overall cost around $359 million. With this in mind, a lot of time, money, and effort is put into developing a new drug and getting it approved, but individuals remain hard at work to develop medications that will safely and effectively treat individuals.

For more information on Nationwide Children’s Hospital’s Pediatric Epilepsy Center, click here or listen to our PediaCast.

Kelly Huston
Kelly Huston is a 2018 PharmD candidate at Cedarville University School of Pharmacy on clinical rotation with Nationwide Children's Hospital. Her interests include geriatrics, psychiatry, and pharmacy informatics. In light of these interests, she will be pursuing a PGY1 residency.

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