Clinical trials are research studies that test the safety of new medical treatments and how well they work. They’re the backbone of medical progress. Every modern treatment available today — from chemotherapy to targeted therapies — exists because people took part in clinical trials.
But they’re also often misunderstood. If you’ve never taken part in one, you may have questions about what they’re like and what they require of the people in them. Understanding how they really work can make it easier to have informed conversations with your care team and to decide whether a clinical trial is right for you.
Developing new drugs is a major focus of clinical trials, but they’re also used to answer other questions, such as who benefits most from existing treatments and how existing treatments work best. The overarching goal is to find ways to improve care.
In a recent WebMD Reddit AMA on clinical trials for cancer, Marina Baretti, MD, a cancer researcher who holds the Jiasheng Professorship in Hepato-Biliary Cancer Research at Johns Hopkins University School of Medicine, shared her expertise and experience with clinical trials to help bring awareness to their importance and clear up common misconceptions.
Myth No. 1: Clinical trials test drugs on healthy people
One of the most widespread misconceptions is that clinical trials are experiments on healthy volunteers. But that’s not how most trials work, especially in cancer research. Who participates depends on the study, but trials require their participants to have specific diagnoses. Even in prevention studies, people who take part may have underlying risk factors, such as genetic predispositions or precancerous conditions, that place them at higher risk of developing cancer.
“Even the early-phase trials are really almost always conducted in patients who have cancer,” Baretti says. “These are not healthy volunteers.”
The reason is that cancer treatments can carry real risks, so exposing healthy people to those risks wouldn’t be ethical. Researchers also need to see how a treatment works in the body when cancer is present. For example, if the goal of the drug is to shrink a tumor, there needs to be a tumor to shrink.
“The overall concept is that these trials are not just designed to assess safety, but also designed to look for early signs of activity against the cancer,” Baretti says. “And this can only be meaningfully evaluated in people who actually have the disease.”
Myth No. 2: Clinical trials make you a “guinea pig” for unsafe treatments
Before any drug reaches human testing, it goes through a long process of lab and animal research. By the time a research team offers a trial to humans, there is already some proof showing the treatment could be safe and potentially helpful. From there, every study must be reviewed and approved by ethics committees that are specifically tasked with protecting your safety and rights.
Also, every clinical trial requires informed consent from the people in it. That means researchers give you all the information you need before a study so you can decide for yourself if you want to do it. Every participant should first know the purpose of the study, what it involves, and the potential risks and benefits before agreeing to take part.
Safety doesn’t stop once the trial begins. They’re tightly regulated as they happen. Researchers track you carefully and watch for side effects in real time. The USDA also oversees clinical trials to ensure they meet strict standards.
“You’re closely monitored to ensure your safety and to make sure the drug is not causing unnecessary toxicity,” Baretti says.
And your participation is always voluntary. You can withdraw from a trial at any time, for any reason. You’re in control throughout the process.
Myth No. 3: You might get a placebo instead of real treatment
Placebos are inactive medications or treatments that look the same as the treatment researchers are studying in a clinical trial. Some studies are designed as “double blind.” This means that neither you nor your medical team know which group you’re in. This helps researchers to make fair, accurate comparisons between the two.
A common fear about clinical trials is that you’ll agree to take part in a clinical trial and then get a placebo and therefore be getting no treatment at all. In most clinical trials, people still get the usual treatment doctors already use. Then they’re randomly given either the new drug or a placebo on top of that. That way no one misses out on care that’s already known to work.
In rare cases where there isn’t a proven treatment yet, some trials use a placebo by itself. But these studies are closely checked and strictly regulated to make sure they’re fair and safe. The goal is to see whether the new option improves outcomes, not to take away all forms of care.
But even if your study uses a placebo (and many do not), every participant gets the current standard treatment as a baseline, Baretti says. The goal is to see whether the new option improves outcomes, not to take away all forms of care.
“The trials will either compare the standard of care with something new or will add new drugs and new medications on top of the standard therapy,” she says.
What’s more, some trials include what’s called a crossover design, where participants in the placebo or standard of care group can switch to the new treatment if the study shows benefit or if their disease progresses, says Baretti.
“There’s also a pathway for what we call accelerated approval, as well as compassionate use programs,” she says. “Accelerated approval is conditional — it allows a drug to become available based on early signs of benefit, but it still requires larger, confirmatory studies over time to ensure that the benefit is sustained in a broader patient population.”
Compassionate use programs can also provide access to promising therapies outside of clinical trials, especially if no other options are available. These pathways are often a way to have treatment accessible earlier on for cases such as rare cancer types.
Myth No. 4: A clinical trial helps only those after you
As a clinical trial participant, you may think that you’re contributing to science but not necessarily helping yourself. While it’s true that trials inform future care, that doesn’t mean you won’t benefit from the treatment, too. You can think of it as “early access.”
When you take part in a clinical trial, you may gain access to promising therapies years before they become widely available. You’ll also get more frequent monitoring and follow-up care, which can lead to earlier detection of complications or changes in your condition.
In fact, multiple studies show that people in clinical trials often have better outcomes than people not in trials. Researchers think this may be because of the extra attention you get as part of a study.
Myth No. 5: Clinical trials are a last resort
You might have heard that clinical trials are something you turn to only after all other treatments have failed. In reality, some clinical trials are designed for people who are newly diagnosed or who haven’t started treatment yet.
“Sometimes having early access to clinical trials might give the opportunity to access drugs that might be a good fit for your tumor type or biology,” Baretti says.
You don't want to miss the window of opportunity to participate in a trial because some trials can have strict criteria in terms of what are the characteristics, the eligibility criteria, and the number of prior lines of therapies, she says. This is why it’s important to ask about clinical trials as part of your initial treatment discussions as one of several paths to consider.
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Photo Credit: DigitalVision/Getty Images
SOURCES:
Marina Baretti, MD, assistant professor of oncology and The Jiasheng Professorship in Hepato-Biliary Cancer Research, Johns Hopkins University School of Medicine.
UCLA Health: “Clinical trials: Myths vs. facts.”
Johns Hopkins Medicine: “Clinical Trials: Separating Fact from Fiction.”
National Cancer Institute: “Placebo.”
University of Utah Health: “Four Common Myths about Clinical Trials—Debunked.”
