If and when you decide to join a clinical trial, you should have a discussion with your doctor about the goals of the trial and what you can reasonably expect to get out of it.
Different types of clinical trials
Clinical trials are research studies involving human volunteers that form the basis for how medical care is delivered and improved. Some trials are designed simply to ensure that a treatment is safe to use; others compare interventions to determine whether one is safer or more effective.
The treatment may be a drug for someone with cancer or it might compare surgery with nonsurgical interventions. It might be a behavioral intervention such as meditation in a person with a chronic illness.
In some cases, patients may enter a clinical study to be observed, and no specific treatment is assigned. One observational study that I lead is following 2,000 patients who have been diagnosed with a bone marrow cancer called myelodysplastic syndrome. In this study, treatment decisions are made by patients and their doctors, while my research team will collect data from them to learn more about the natural history of the disease.
If you are considering a clinical trial, it’s important to ask whether it is a Phase 1, 2 or 3 trial.
Phase 1: These trials, sometimes called “first in human” studies, examine the safety of a drug and determine the correct dose and schedule. The focus is on side effects, and only secondarily on whether a drug works. Often, not more than a dozen or two dozen patients enroll in a Phase 1 trial, and everyone typically receives the study drug.
Phase 2: In these trials, the focus is on whether the intervention is effective in participants with the same diagnosis. Safety continues to be determined. Dozens or even a couple hundred people may enroll in these types of studies.
Phase 3: These trials are randomized, meaning some participants are treated with a study intervention, while other participants receive the standard of care that may also include a placebo. Safety and efficacy of the two treatments are compared. Hundreds or even thousands of people may be enrolled in a Phase 3 trial, which may be “blinded,” meaning neither participants, nor sometimes even their doctors, know whether a patient is receiving the study drug or the standard treatment.
Questions to ask before joining a clinical trial
Just as with standard of care treatments, start by asking about the known side effects of the drugs or other interventions being studied in a trial, and the potential to benefit from the trial.
In addition to asking about the phase of the study, ask how many patients with your condition have been treated previously with the study intervention. Ask whether you will receive the intervention, or if you might be randomized to be treated with a standard of care, or a placebo. It is not necessarily a bad thing to receive a placebo — remember, a study intervention may not help, and may have side effects that make you feel worse.
Every trial has a “calendar of events” — required visits, laboratory or radiology tests, and the timing and frequency of the intervention. Ask about it, to make sure you don’t find participating in a trial to be too burdensome, particularly if you live at a distance from the treatment center.
Check on insurance coverage
Ask what parts of a trial will be covered by insurance, what parts are paid for by the study sponsor (such as a pharmaceutical company, foundation or the government) and what will result in out-of-pocket expenses.
Federal law requires most health insurance plans to cover routine patient costs in clinical trials. For example, when I treat a patient who has myelodysplastic syndrome, I will routinely check my patient’s blood counts every week. Thus, weekly blood count checks in a clinical trial for myelodysplastic syndrome should be covered by insurance.
If that same trial required that participants undergo an EKG every week — something I wouldn’t do routinely — those EKGs should be paid for by the sponsor of the trial. Typical out-of-pocket expenses might include parking, paying for gas, or usual co-pays for office visits. Sometimes those can be reimbursed by the trial sponsor or patient advocacy groups, so make sure to ask.
Gaining access to new drugs
“I don’t want to be a guinea pig.” This is how my own mother responded to the question of whether she would enroll in a clinical trial, if given the opportunity. As someone who has devoted his career to designing clinical trials and enrolling patients in these studies, I was a little taken aback by her response. I also recognized that it was a common misconception.
In most clinical trials, participants receive at least the standard of care treatment for their conditions.
Because many trials are designed by or with input from experts in the field, and every investigator participating in the trial is required to follow the same “playbook” for monitoring and treating patients, the level of care received in a trial may be, on average, better.
One of my greatest sources of pride has been the ability to offer my patients a number of clinical trials of drugs that were highly effective and ultimately approved by the FDA. By participating in those trials, many of my patients had access to those drugs and benefited from them before they were approved.
Volunteer vs. paid clinical studies
Patients with a defined medical diagnosis who take part in Phase 1, 2 or 3 clinical trials do so on a voluntary basis and typically aren’t paid to participate. However, sometimes university and drug company researchers recruit healthy volunteers to participate in various types of paid research. For instance, healthy volunteers might get paid for participating in studies designed to gain preliminary information about drug distribution and metabolism in the body, and in these cases some may receive a placebo. Or social science researchers may pay volunteers to take part in a series of experiments to measure the stress response. These paid studies serve a purpose in research but are not typically labeled as a clinical trial.
How to find a clinical trial
Ask your health-care team about clinical trial possibilities. You can also do your own research. My go-to site to search for clinical trials is clinicaltrials.gov. There are over 445,000 studies listed on clinicaltrials.gov — approximately one-third of which are being conducted in the United States. Here, you can enter information about your condition and location as well as other terms such as a specific drug’s name or the name of an investigator. Some patient advocacy groups have clinical trial nurse navigators to help you identify the right clinical trial for your diagnosis.
Mikkael A. Sekeres, MD, MS is chief of the division of hematology at the Sylvester Comprehensive Cancer Center, University of Miami. He is author of When Blood Breaks Down: Life Lessons from Leukemia and Drugs and the FDA: Safety, Efficacy, and the Public’s Trust. Follow him on Twitter @MikkaelSekeres.