Clinical research is medical research involving people. There are two types, observational studies and clinical trials.
Observational studies observe people in normal settings. Researchers gather information, group volunteers according to broad characteristics, and compare changes over time. For example, researchers may collect data through medical exams, tests, or questionnaires about a group of older adults over time to learn more about the effects of different lifestyles on cognitive health. These studies may help identify new possibilities for clinical trials.
Clinical trials are research studies performed in people that are aimed at evaluating a medical, surgical, or behavioral intervention. They are the primary way that researchers find out if a new treatment, like a new drug or diet or medical device (for example, a pacemaker) is safe and effective in people.
Often a clinical trial is used to learn if a new treatment is more effective and/or has less harmful side effects than the standard treatment. Other clinical trials test ways to find a disease early, sometimes before there are symptoms. Still others test ways to prevent a health problem. A clinical trial may also look at how to make life better for people living with a life-threatening disease or a chronic health problem. Clinical trials sometimes study the role of caregivers or support groups.
Before the U.S. Food and Drug Administration (FDA) approves a clinical trial to begin, scientists perform laboratory tests and studies in animals to test a potential therapy’s safety and efficacy. If these studies show favorable results, the FDA gives approval for the intervention to be tested in humans.
Clinical trials advance through four phases to test a treatment, find the appropriate dosage, and look for side effects. If, after the first three phases, researchers find a drug or other intervention to be safe and effective, the FDA approves it for clinical use and continues to monitor its effects.
Clinical trials of drugs are usually described based on their phase. The FDA typically requires Phase I, II, and III trials to be conducted to determine if the drug can be approved for use.
There are many reasons why people choose to join a clinical trial. Some join a trial because the treatments they have tried for their health problem did not work. Others participate because there is no treatment for their health problem. By being part of a clinical trial, participants may find out about new treatments before they are widely available. Some studies are designed for, or include, people who are healthy but want to help find ways to prevent a disease, such as one that may be common in their family.
Many people say participating in a clinical trial is a way to play a more active role in their own health care. Other people say they want to help researchers learn more about certain health problems. Whatever the motivation, when you choose to participate in a clinical trial, you become a partner in scientific discovery. And, your contribution can help future generations lead healthier lives. Major medical breakthroughs could not happen without the generosity of clinical trial participants—young and old.
Here’s what happens in a trial:
1. Study staff explain the trial in detail and gather more information about you.
2. Once you have had all your questions answered and agree to participate, you sign an informed consent form.
3. You are screened to make sure you qualify for the trial.
4. If accepted into the trial, you schedule a first visit (called the “baseline” visit). The researchers conduct cognitive and/or physical tests during this visit.
5. You are randomly assigned to a treatment or control group.
6. You and your family members follow the trial procedures and report any issues or concerns to researchers.
7. You may visit the research site at regularly scheduled times for new cognitive, physical, or other evaluations and discussions with staff. At these visits, the research team collects information about effects of the intervention and your safety and well-being.
8. You continue to see your regular physician for usual health care throughout the study.
The following are some questions to ask the research team when thinking about a clinical trial. Write down any questions you might have and bring your list with you when you first meet with the research team.