New Treatments & Clinical Trials

Innovations in treating cardiovascular disease (CVD) continue to develop rapidly. Now more than ever, people with problems affecting their heart or their blood vessels can be assured there are effective treatments available. The medical community is always working toward innovations to improve patient care, especially considering heart disease is the leading cause of death in the U.S., with nearly 700,000 Americans having died from heart disease in 2020.1 You may be eligible to try new treatment options that haven't yet been approved by the U.S. Food and Drug Administration (FDA) by participating in a clinical research trial. However, it's important to learn how the process of developing and approving these new treatments work.

Developing new drugs and medical devices

Products almost always begin development in the laboratory where the concept is tested outside of humans. These are called “preclinical” studies. If these studies look promising, then researchers seek approval for research in humans.

The first studies in humans may be very small. The goal of these studies is to confirm that the drug or device is safe and appears to function as expected. If so, then additional studies are planned.

New products must pass through progressively larger research studies until sufficient evidence has been accumulated. The evidence must show that the drug or device is safe and effective. At that point, an application is made to the FDA for approval to use the product in appropriate patients.

It can be confusing because research about various treatments may be published on the internet before the process is complete. It's important to remember that just because a research study showed a treatment may be beneficial, it's necessary to complete the entire process before we can confidently say it should be used. Unfortunately, the history of medicine is full of many stories about treatments that seemed very promising in initial studies, only to find later that they were either ineffective or even dangerous when larger studies were done.

FDA approval

As consumers of new medicine and other types of treatment, many of us are familiar with the stage that involves approval by the FDA. Although you may be eager to try a new treatment, unless you participate in a clinical trial, you may have to wait until the FDA approves the treatment as safe and effective.

Enrolling in a clinical trial

If you’re interested in trying a treatment in development and not yet approved by the FDA, talk with your doctor about participating in a clinical trial.

Off-label use of drugs and medical devices

In the U.S., FDA regulations permit doctors and other healthcare providers to prescribe approved drugs and medical devices for indications other than the disease or condition for which they’re approved—this is called "off-label" use.

The importance of asking questions

Whether you're learning about a new medical study in the news or are interested in participating in a clinical trial, it's always important to ask questions to get as much accurate information as possible. The more information you have, the better equipped you are to make informed decisions with your doctor regarding your health.