How Clinical Studies Get Approved by the FDA and Why That Is Important

Research scientists and doctors are like detectives. They study people to find clues to help them solve health mysteries. Research studies or trials are step-by-step investigations experts use to discover new treatments for diseases. These treatments can be medicines or medical devices developed to help people dealing with health conditions. 

New drugs and treatments travel through steps or phases to ensure they work and are safe before the public can use them. The Food and Drug Administration (FDA) decides if a drug or treatment is approved based on the evidence provided by research studies. Clinical research is essential for medicine to progress, so let’s explain how studies or trials work.

Understanding Clinical Trials

There are four phases a clinical trial completes in order to achieve FDA approval. Early phase or preclinical discovery is the initial action scientists take to start a study.

Phase 1: Testing for Safety and Figuring Out Dosage 

In this phase, a new drug or treatment is tested to learn if it’s safe for people. Phase 1 is also when scientists determine how much, or what dosage, is safe to use.

Phase 2: Does it Work or Have Side Effects?

In phase 2, people with the health condition being studied are given the drug that is being developed for that condition. This phase helps determine if the drug works, and people are watched for side effects.

Phase 3: Confirm Effectiveness and Compare Existing Treatments

Phase 3 involves the largest group of participants with the condition who are given the treatment. This is the phase when a study may end if the drug, device or treatment isn’t working as expected. The FDA reviews phase 3 information or data to decide if the treatment will be approved. 

Phase 4: Treatment Approved, Ready for Sale and Watched

Once the new treatment is approved, it’s made available for the public to purchase. The FDA continues to observe people using the treatment to watch for long-term side effects and learn new benefits.

The FDA Approval Process

Now we understand that the FDA obtains information from clinical research, and it reviews it before approving new treatments. Additionally, the FDA follows specific steps before approving new drugs. 

If pre-clinical trial research looks promising, the sponsors or investigators submit an Investigational New Drug (IND) application. It’s like asking permission to begin a clinical study. The FDA has 30 days to review the application. Once the FDA approves the IND application, the drug goes through clinical testing in Phases 1 to 3. After Phase 3, the FDA takes a closer look before the drug can move to Phase 4:

• The drug company can apply for full approval by submitting a New Drug Application (NDA).
• The FDA has 60 days to decide if the application is ready for a full review.
• A team of experts — including doctors, scientists and statisticians — carefully examines all the research and data.
• FDA inspectors visit the sites that participated in the clinical trial and the places where the drug is made and tested to make sure everything meets standards.
• The FDA also checks the drug’s label to ensure it gives clear and accurate information.
• If everything looks good, the FDA approves the drug, and it moves into Phase 4 (post-approval monitoring).
• If there are problems, the FDA sends a letter (called a Complete Response Letter) explaining what needs to be fixed before the drug can be approved.

An Exception to the Rules

In some cases, a treatment may be pushed through the review process because there is an urgent need. This is called an Emergency Use IND. In these cases, the usual paperwork and waiting time are skipped so the treatment can be used quickly, like during the COVID-19 pandemic. Emergency approval can also be used for people with serious illnesses who have no other treatment options.

Some treatments take decades of research before they’re approved. These long-term efforts can lead to major breakthroughs for diseases that were once untreatable.

The key point is this: Clinical research is essential for medical progress. It helps solve medical mysteries. Without the hard work of researchers, doctors and other health experts constantly testing and improving treatments, we wouldn’t have many of the medicines we rely on today. Clinical research helps answer the questions the FDA needs to approve new treatments.