What comes to mind when you hear the words “clinical trial”? If it’s placebos and a last resort, then it’s time to set the record straight. While medical dramas might have you believe that clinical trials are only for people who've exhausted all their other options, the truth is that they can offer access to investigative treatment at any point in a cancer journey, and some patients can join a trial from the beginning of their cancer treatment plan.
While every FDA-approved drug has gone through clinical trials, only about seven percent of Americans living with cancer enroll in clinical trials, and even fewer newly diagnosed patients participate as a first line of treatment at the onset of diagnosis. That means many people with cancer may miss out on the potential breakthroughs these trials can lead to. The best time to ask your doctor about clinical trials isn’t after other options are exhausted; it’s at the time of diagnosis.
So, what exactly are cancer clinical trials, how do they work, and who's eligible for them? To answer these questions, we spoke with Arjun Balar, MD, senior vice president and head of oncology global clinical development at Eli Lilly and Company.
What is a clinical trial?
A clinical trial is a research study designed to evaluate the safety and effectiveness of investigational medications, medical devices, diagnostic tools, or behavioral interventions that have not yet been approved by the FDA. Each trial consists of four main phases, which Dr. Balar breaks into early and late phases: “Early-phase trials are what we call ‘open label’ — this means everyone gets the drug being tested, and you know what dose it is.” These early trials are where researchers look at safety signals and tolerability, assess dosing, understand how the body processes the drug, and assess early signals of potential benefit.
In a Phase 3 trial, the investigational treatment being tested has undergone vetting in clinical and lab settings. In cancer Phase 3 clinical trials, the investigative treatment is typically compared to the standard of care to determine if it has the potential to be better than what is currently available. Participants are not given a placebo — they receive active treatment regardless of which group they are assigned, Dr. Balar explains. “In Phase 3 trials, half of the patients get the existing treatment approach, and the other half get the investigative treatment. This means the ‘comparator’ group is actually receiving the exact same treatment as they would have received if they had not participated in the trial.”
What are the benefits of participating in a Phase 3 cancer clinical trial?
If you participate in a Phase 3 cancer clinical trial, no matter which treatment group you fall under, all patients will receive specialized attention and careful monitoring. “Trial participants have a whole team of people, some of whom you see and many of whom you don't, who are keeping an extraordinarily close eye on you,” says Dr. Balar.
For a newly diagnosed patient, that level of dedicated attention can be very meaningful and comforting. Trial participants receive structured, consistent monitoring – testing on schedule, proactive side effect management, and a responsive care team.
Participation in a clinical trial is completely voluntary. In order to obtain your informed consent, prior to joining a trial, the research team will explain the study, including potential risks and benefits associated with your participation
Clinical trial enrollment and cancer treatment innovation
Increasing enrollment in Phase 3 cancer clinical trials specifically is key to developing potential treatments, advancing care for people living with cancer, and advancing medical research.
“There are scientific advancements happening all the time,” says Dr. Balar, “So as technology improves, we are continuing to push the boundaries of cancer treatments.”
This isn’t to say current treatments are ineffective, but that potentially better, more advanced treatments are being studied every day to answer questions to assess their safety and effectiveness before approval. “What is the right dose? What is the right schedule?" says Dr. Balar. "How much do we know about the side effects? These potential treatments are still being developed.
How can you find out about cancer clinical trials you might be eligible for?
Every patient deserves to be fully informed. From the moment you receive a diagnosis, ask your doctor about clinical trials as an option worth exploring from the start. Don’t wait for your doctor to bring it up: ask directly. According to Dr. Balar, “if your physician isn’t running a trial, many times they have relationships with other physicians in their network or community, so they can help you find one.”
Another option is to try a patient advocacy group. Almost every single type of cancer has a corresponding advocacy group, composed of people who are knowledgeable about your type of cancer and can offer support resources.
Clinical trials aren’t a last-ditch effort or a leap into the unknown — they’re a regulated, closely monitored pathway to developing potential treatments, and often, additional support from the care team. By understanding how they actually work and asking questions, patients can move past fears and gain a better understanding of what they truly are: an opportunity to play a role in shaping the future of medicine and access to potential investigational treatments. And whether you’re newly diagnosed or exploring next steps, asking about clinical trials could help open the door to potential options you didn’t know existed.
To learn more about cancer clinical trial options, visit cancerclinicaltrials.lilly.com.
