Next time you open your medicine cabinet or fill a prescription, think about this: New drugs go through an average of 12 years of 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 Trials
Without these studies, and the people who volunteer to take part in them, many common medicines including aspirin, statins to lower cholesterol, beta blockers for high blood pressure and the newer diabetes medicines would not be available today. Nor would we have developed such 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 rigorous scientific studies to prove that a treatment or device is both safe and effective before allowing it to be available to patients. Altogether, more than a hundred FDA-approved drugs and devices are available to treat or stave off cardiovascular disease, and they work in different ways.
Clinical trials are carefully planned research studies that assess new drugs, devices, procedures, biologics (for example, vaccines or targeted therapies), diagnostic approaches/tools.
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, etc.).
Research 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.
Research studies also can be designed to look at large populations of people (called observational studies) and to identify important trends in heart disease over time.
Researchers also plan trials to evaluate how certain lifestyle habits such as diet or exercise influence outcomes. For instance, the DASH diet plan was shown in a clinical trial to lower blood pressure, especially among Black patients.
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 indication that the study treatment is harmful
|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 Americans—often will include fewer patients at each stage of study.
Not all drugs or devices progress to the next phase of study either because they do harm, are found to be ineffective or 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.
Clinical trials rely on volunteers. Many clinical trials are delayed and some never happen because there aren't enough volunteers.
People might decide to join a clinical trial for many reasons. For example, to help:
Historically, certain groups—women, people 75+ years old and certain racial/ethnic minorities, in particular—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/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 truly gauge how well they work within the general population. If a drug is studied only in adult white men, it is not necessarily applicable in general to others.
“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, stresses that volunteers are the foundation of clinical studies. “We will only know if and how a therapy works in men and women, and different racial/ethnic groups, if we study these interventions in everyone,” she said.
Only a small fraction of people with heart or
vascular diseases actually take part in clinical trials. But of those who do participate, many say they would join one again. Unfortunately, there are barriers or hesitations to participate. People may:
“Clinical research does not pay participants in research studies but often will help with travel costs, and the volunteers benefit form 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 trials 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.
It’s important to talk with your health care team about how you are feeling and if your current treatment plan is working for you. A lot of research us underway, so taking part in a clinical trial may be or become an option for you in preventing or treating heart disease.
If you’re thinking about participating in a clinical trial, here are a few questions you might want to ask: