Next time you open your medicine cabinet or fill a prescription, think about this: New drugs go through rigorous testing before they are deemed safe and effective for people to use.
Research is done through clinical studies, which are essential for improving health and medical care.
Clinical studies are proving ground for new or better:
Learn More: Increasing Diversity in Clinical Studies
Without these studies, and the people who volunteer to take part in them, many common medicines including aspirin, statins to lower cholesterol, and medicines for high blood pressure and diabetes would not be available today. Through clinical research, we have developed a deep understanding of diseases, such as heart disease and stroke, and how best to treat and prevent them.
The Food and Drug Administration (FDA) requires studies to prove that a treatment or device is safe and effective before allowing it to be given to patients. More than a hundred FDA-approved drugs and devices are available to treat or prevent heart and blood vessel disease.
Clinical trials are carefully planned research studies that assess new drugs, devices, procedures, vaccines, or tests or screenings.
These studies also test interventions that might help prevent another heart attack or slow the progression of diseases (for example, cardiac rehabilitation or team-based care).
The studies look at specific drugs or treatments to determine:
Cardiovascular studies help answer scientific questions and find new ways to:
Often during research, the best standard treatment or procedure is compared with the best standard treatment + new drug or intervention.
Placebos – a pill or injected medicine that has no therapeutic effect – aren’t used for life-threatening health issues.
Even when new therapies are compared to placebo, both the treatment and control groups are offered standard medications and treatments, laboratory testing and close supervised follow-up.
New medicine and devices have to advance through four phases of study. While each phase has a different goal, each one builds on the next.
Researchers start by studying the treatment in a small group of patients. They will invite more people to enroll once there is clear evidence that the therapy being tested causes no harm.
Participants can drop out of a clinical trial at any time. The study can also be stopped midway through if there is any sign that the study treatment is causing harm.
|Trial Phase ||Main Focus ||People Enrolled * ||How Long |
|Phase 1||Is it safe? What’s the best dose (amount) to give?||20-100 healthy people with the condition||Several months|
|Phase 2||Does it work? Are there side effects?||100-300 people with the disease||Several months to 2 years |
|Phase 3||How well does it work? How does it compare with existing treatments?|| 300-3,000 people|| 1-4 years|
|Phase 4||Post-marketing studies that look at how the drug works in a large population of patients over an extended period of time. Is there continued safety and efficacy?|| Several thousand|
* Studies that enroll people with rare diseases – conditions that affect fewer than 200,000 people in the U.S. – often will include fewer patients at each stage of study.
Some drugs or devices don't progress to the next phase of study because they do harm, are found to be ineffective, or for other reasons.
FDA also has fast-track and breakthrough therapy designations to help speed up the development and study of drugs that treat serious or life-threatening conditions or that fill an unmet medical need.
Another type of clinical research involves researchers gathering information from participants or examining information that has already been collected. This is called an observational study.
In this type of study, researchers do not assign a particular intervention to participants.
These research studies often are designed to look at large populations of people. Observational studies help identify important trends in heart disease over time.
Clinical studies rely on volunteers. Many clinical studies are delayed and some never happen because there aren't enough volunteers.
People might decide to join a clinical study for many reasons. For example, to help:
Historically, certain groups – women, people older than 75, and people from certain racial and ethnic minority groups – have not been well represented in clinical studies. Yet, we know certain treatments work differently in men and women, as well as in people from different racial or ethnic backgrounds.
Many of these groups also have a heavier burden of cardiovascular disease and risk factors. New therapies need to be tested in a wide variety of people to find out how well they work within the general population. If a drug is studied only in adult white men, the research findings may not apply to people of other backgrounds.
“A wide range of people should have the opportunity to participate in trials both to have access to new therapies and to have the chance to contribute to better treatment of everyone,” said Keith C. Ferdinand, MD, professor of medicine, Tulane University, New Orleans. “It’s an important altruistic goal for many Americans.”
Martha Gulati, MD, associate director of the Barbra Streisand Women Heart Center in Los Angeles, stresses that volunteers are the foundation of clinical studies. “We will only know if and how a therapy works in men and women, and people of different races or ethnic groups, if we study these interventions in everyone,” she said.
Only a small fraction of people with heart or blood vessel diseases actually take part in clinical studies. But of those who do, many say they would join one again. Unfortunately, there are barriers or hesitations to participate. People may:
Participants in research studies may receive a small compensation, which can help cover travel or other costs.
"Also, volunteers can benefit from uncovering how new drugs and devices will work for all patients,” Dr. Ferdinand said. “If a major breakthrough therapy is discovered, the new therapy is sometimes offered as compassionate, extended free therapy.”
Depending on your condition, Dr. Gulati says it’s important to ask about clinical studies as a potential treatment path so you know all of your options. These studies aren’t only for people with late stage heart or vascular diseases.