Medical studies, or clinical trials, are everywhere in the news and on social media. You may see flyers about medical studies posted at the doctor’s office, hospital, clinic, or even posted on public bulletin boards. You may hear about a patient making an unexpected recovery after receiving an “experimental treatment” in a medical study. So what are these medical studies? Who participates in them? Can a medical study be the right answer for your health care?
What is a clinical trial?
The technical name for a medical research study that involves volunteer human participants is a “clinical trial.” Clinical trials are intended to study new methods to prevent, detect, or treat diseases, known as interventions. An intervention may be a medical product, like a drug or a medical device, or it may be a procedure, like a surgery or an MRI. An intervention can also be a behavioral change, like dietary changes or a new exercise program.
Who runs clinical trials?
Pharmaceutical companies, academic medical centers, universities, and patient groups can sponsor (pay for and conduct) a clinical trial. Government agencies such as the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs often also do so. Every clinical trial has a primary investigator, who is most often a medical doctor. The clinical trial team can include other doctors, nurses, social workers, therapists, statisticians, and clerical staff members.
What is the goal of a clinical trial?
There are many reasons that researchers conduct clinical trials, including:
- To find out if a new intervention, or set of interventions, is more effective or safe than what is already available.
- To show that a new intervention is just as good as what is already available, but might be lower in cost, or easier for patients to tolerate.
- To find out if an intervention can diagnose a condition better and more accurately than what is already available.
- To see if an intervention can improve patient quality of life compared to what is already available.
How are clinical trials conducted?
Every clinical trial has a protocol, which is a detailed plan that explains exactly how the trial will happen. The protocol says which type of people may volunteer for the study by laying out specific inclusion criteria. Inclusion criteria may include age, gender, disease status, stage of disease, etc. There are also exclusion criteria that specify which people may not volunteer for the study. Exclusion criteria often include the prior use of a certain drug or treatment.
The protocol also specifies what the intervention will be compared against. Sometimes, it is compared against a placebo. A placebo can be a fake procedure, a “sugar pill”, or a sham treatment. Study participants who receive the placebo believe that they are receiving medical treatment, but they are not. Sometimes an intervention is compared against an established medical procedure, device, or drug that is already in use by doctors.
Doctors and researchers collect data about the effectiveness and safety of the intervention or interventions during the trial. After the clinical trial is over, doctors, researchers, and statisticians analyze the data and often publish it in a scientific journal for other researchers and doctors to read. If the intervention is promising, it may be tested again in a larger trial with more volunteers, or it may go through an approval process by the US Food and Drug Administration (FDA) to become a marketed drug or device.
What are the benefits and risks of participating in a clinical trial?
Clinical trial researchers cannot be sure whether the new intervention or interventions will be helpful, harmful, or have no effect. Therefore, there can be both risks and benefits to participating in a clinical trial.
Some of the risks may include:
- Experiencing side effects. These may be mild and just a little bothersome, or they may be serious and require stopping the trial. In some rare cases, participants in clinical trials have experienced life-threatening side effects.
- Losing time for visits to the study site, blood tests, treatments, hospital stays, or complex dosage requirements.
- Receiving a placebo when you had hoped to receive the experimental intervention.
Some of the benefits include:
- Gaining access to new treatments or medical interventions before they are widely available.
- More careful monitoring by a research team that includes doctors and other health professionals.
- Helping other people by contributing to medical research.
Any study of a drug, biological product, or medical device that is regulated by the FDA must be reviewed, approved, and monitored by an institutional review board (IRB). This board is made up of doctors, scientists, ethicists, and members of the community. The IRB is tasked with making sure that the study is ethical and that the rights and welfare of participants are protected.
How can I decide if a clinical trial is the right answer for me?
Anyone considering a clinical trial should discuss it with his or her doctor. According to ClinicalTrials.Gov, some questions that patients can ask include:
- What intervention is being studied?
- Has this intervention been tested before?
- Is the intervention being tested against placebo, or against an already available therapy or treatment? How will participants be assigned to the groups?
- Who will know which intervention I receive during the trial? Will I know? Will members of the research team know?
- How do the possible risks, side effects, and benefits of this trial compare with those of my current treatment?
- What tests and procedures are involved?
- How often will I have to visit the hospital or clinic?
- How long will the study last?
- Will I be paid for my participation? Will I be reimbursed for other expenses?
- What type of long-term follow-up care is part of this trial?
- If I benefit from the intervention, will I be allowed to continue receiving it after the trial ends?
- Will results of the study be provided to me?
- Who will oversee my medical care while I am participating in the trial?
- What happens if I experience side effects during the study?
Clinical trials are the most powerful way for scientists and researchers to understand the safety and effectiveness of new drugs, devices, and other medical therapies. Healthy volunteers and those with a disease or condition can pave the way for groundbreaking scientific and medical advances by participating in a clinical trial. However, it is important to consult your doctor and weight the risks and benefits carefully before enrolling.
For Further Reading
ClinicalTrials.gov. Learn About Clinical Studies.
National Institutes of Health. NIH Clinical Research Trials and You – The Basics.
The U.S. Food and Drug Administration. The FDA’s Drug Review Process: Ensuring Drugs Are Safe and Effective.