5 things to know about the FDA approval process

Whether it’s a cancer drug, vaccine or other medication, medical treatments in the U.S. have to go through a specific process to make sure they’re safe and effective before being released to the public.

The Food and Drug Administration (FDA) oversees the process from start to finish – and even after approval.

To better understand the FDA approval process, we spoke to Vivek Subbiah, M.D. Here’s what he shared.

The FDA carefully reviews all drugs and vaccines

Cancer drugs and vaccines, like the COVID-19 vaccine, follow a similar process for approval.

The first step is called discovery. This is when researchers learn about the disease they’re targeting and make a plan to treat it. With vaccines, they’re trying to mimic a virus’ effects on the body to train the immune system to recognize and defend against it. Cancer drugs may target a specific type of cancer, genetic mutation or protein within the cells.

After discovery, the new treatment goes through three phases of clinical trials before it can be approved. Each phase is designed with different variables to make sure the treatment is effective and safe.

Phase I is the first time the treatment will be given to humans to determine the best dose and preliminary safety data. Phase II clinical trials test for safety and efficacy. Phase III studies test for efficacy and collect more safety data in a larger group of people. If a Phase III clinical trial is successful, the drug or vaccine can be submitted for FDA approval.  

“With cancer drugs, the approval process can take anywhere from six to 12 years,” Subbiah says. Each phase can take around two years, but the timeline depends on how rare the disease being treated is.

For less common conditions, each phase may take longer because there simply aren’t enough patients to work with. This is one of the reasons it is so important for people to enroll in clinical trials, especially if you’re diagnosed with a rare disease or condition. 

The process for vaccine approval is similar, but there’s not a definite timeline, as we’re seeing with COVID-19 vaccine development. In most cases, the better the understanding of a virus and the disease it causes, the more efficient a vaccine will be.

Emergency use authorizations (EUA) may be granted in certain cases if vaccines are effective

Just because a drug or vaccine is developed quickly doesn’t mean researchers are taking shortcuts. “The FDA vaccine development program is one of the most robust in the world,” Subbiah says.

In some cases, if a drug or vaccine is successful in a Phase II or early Phase III study, they may be authorized or approved more quickly. For vaccines, this is called an emergency use authorization (EUA). If a vaccine receives emergency use authorization, that means the results of the clinical trial show the vaccine is working, and side effects are tolerable and the benefits outweigh the risks.

EUAs are only valid for a certain amount of time – for example, throughout a public health crisis like the COVID-19 pandemic. After receiving an EUA, a vaccine or drug still needs to go through the full FDA approval process like the COVID-19 vaccines are. This process can take some time. That’s why clinical trials for many COVID-19 vaccines are continuing, even though they’ve already received EUA.  

Accelerated approval is often used for cancer drugs

The FDA may also approve cancer drugs before a Phase III clinical trial is done. This process is called accelerated approval.

“We’ve seen accelerated approvals with several cancer drugs recently,” Subbiah says.  Sotorasib , selpercatinib and pralsetinib are three drugs that have seen such encouraging results in clinical trials that they’re now available for patients with non-small cell lung cancer with specific mutations through accelerated approval.

"The studies are showing these drugs offer so much benefit to patients that it’s critical we offer them as soon as possible to save as many lives as possible,” Subbiah adds.  

This fast-track to approval is exactly how the FDA process is designed to work, Subbiah says. “In the case of a public health emergency like the COVID-19 pandemic, the FDA pulls all its resources together to quickly and safely find a way to protect as many people as possible,” he explains.

That teamwork gives him hope for the future of drug development. “If we applied the COVID-19 vaccine model to cancer drugs, we could have safe and effective treatments created more quickly,” Subbiah says. 

The FDA continues monitoring after approval to ensure safety

No drug is 100% effective or completely without side effects. So, those side effects and the long-term effectiveness of the drug or vaccine will be monitored by the FDA – even after the drug or vaccine has been approved.

“When you think of the FDA, it’s not just one group that makes the decisions,” Subbiah says. “There are dozens of government agencies involved in making sure these treatments are safe.”

These include the Centers for Disease Control and Prevention, Centers for Medicare and Medicaid Services and others. The FDA also seeks the input of Vaccine and Related Biological Products Advisory Committee.

Before a drug or vaccine can be approved for a different population, like children, those groups would need to be included in a Phase I clinical trial and start the whole process over.

FDA-approved drugs and vaccines are safe and effective

Science has come such a long way in recent years that drug development can happen more quickly now than ever before. “If something’s been approved by the FDA, you can trust that it’s safe,” Subbiah says.

He stresses that this is especially true for COVID-19 vaccines, even though they are new. The technology used for these mRNA vaccines has been around for decades, which is why researchers were able to develop these COVID-19 vaccines so quickly, Subbiah explains.

“Thanks to science and vaccines, we’ve been able to eradicate so many diseases,” he adds. “You should have confidence in the FDA and how it oversees development of these treatments.”

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