The FDA Drug Approval Process
Chris Regal | June 18, 2012
Ever wonder why medications are so expensive? The FDA approval process is long and arduous for many pharmaceutical companies. The investments are massive and the time needed to conduct the appropriate rounds of testing can take years, even decades. Granted, the FDA is invested in the safety and efficacy of medication–and rightly so. Join us on a peek behind the curtain on how a drug gets approved.
First, you need an idea. Where is there a market need? Both private companies and public research institutions (such as the NIH) fund research that can pinpoint the need for a drug and insight into the preliminary development. Once a drug has been developed in a laboratory, it moves on to the next step.
The first stage of testing is performed on animals. The drug is tested on a variety of species to ensure its safety, all while the initial effects are documented by researchers.
An Investigational New Drug application is submitted to the FDA to report the results of the animal testing. This lays out the plan for the next stage of testing: humans.
Phase 1 testing
Phase 1 testing is performed on between 20 and 80 healthy volunteers. The emphasis is on safety in Phase 1; this phase determines potential side effects and how the body reacts to it.
Phase 2 testing
The emphasis of Phase 2 testing is on effectiveness. The drug is given to hundreds of people with a particular condition which the drug intends to treat. Often, the drug is compared to a placebo in other patients in the trial. Before moving to Phase 3, the drug is evaluated by an FDA panel to decide how large Phase 3 testing will be.
Phase 3 testing
Here, the drug reaches thousands of patients, continuing what was learned in Phases 1 and 2 but on a much larger scale. Safety and effectiveness are evaluated, as are side effects and interactions with other medications. The average size of a Phase 3 trial is 4,000 people.
Now that all of the trials have been performed, it’s time for the FDA to discuss the submission of a New Drug Application.
It’s time for the manufacturer to formally request that the FDA consider the new drug. The Application requires details on all Phases of clinical trials and plans for the future. Take a look at the required forms–this application can be thousands of pages long.
The FDA has 60 days after receiving the New Drug Application to be reviewed. In other words, the FDA has 60 days to consider whether or not it is even interested in filing the drug for a potential review. The drug is then reviewed (if passed on to that step) where the Review team dives into all the research and findings from the first seven steps.
The FDA reviews the information provided and devises a drug label for the medication, complete with side effects, warnings, potential interactions, etc.
Before approval, the FDA has to go and inspect the facilities in which the drug will be manufactured to ensure that everything is up to standard with regard to safety, cleanliness, contamination, etc.
If a drug survives all of the steps, it can finally be approved to reach the market. The total cost for a drug to go from an idea to the market is an estimated $802 million. It takes roughly 16.5 years of development for a drug to reach the market. Of every 5,000 that goes to animal trials, five make it to human testing, and only one makes it to market.
The FDA has an Accelerated Approval program designed to expedite the process for an unmet medical need. To qualify for this program, the FDA must be able to see the effectiveness on a blood test or X-ray, for example, which can provide definitive proof of efficacy rather than waiting for clinical trials.
Fast track program
In the late 1900s, AIDS patients were dying before drugs could go through all of the approval steps. In response, the FDA created a Fast Track Program where the FDA reviews products to treat serious life-threatening disease and meet medical needs. It is processed as a “rolling submission” rather than waiting through each step individually.
The FDA continuously monitors the safety and effectiveness of the new drug, even after approval. The manufacturer also has to submit periodic updates on safety and effectiveness of the drug. If problems are found, the FDA may change the labeling, issue a public warning or pull the drug from the market entirely.