Clinical trials assess the safety and efficacy of new cancer drugs or drug combinations. The data and learnings these studies collect help regulators determine if the treatments should be approved and made broadly available to patients.
Clinical researchers conduct clinical trials at cancer institutes, medical centers, clinics, and hospitals worldwide, all under strict guidelines. Many oncologists at Dana-Farber are specialists in both cancer care and in clinical research because they want to do all they can to help improve outcomes for patients with cancer.
“Clinical trials are done with a great deal of care, and generate the data needed for analyzing whether a drug is safe for patients, what the right dosage is, and how effective the drug is either alone, in combination, or in comparison with standard treatment,” says Jeffrey Meyerhardt, MD, MPH, Chief Clinical Research Officer at Dana-Farber. “They are essential for moving cancer treatment forward and bringing new and improved cancer therapies to our patients.”
How do clinical trials move cancer treatment forward?
Clinical trials are designed to rigorously gather evidence about whether a new approach to cancer treatment works, who it works best for, and at what point in a disease or treatment regimen it is best used. In the United States, the results of large clinical trials can lead to U.S. Food and Drug Administration (FDA) approval of a treatment.
The FDA approved 15 completely new cancer medicines in 2024 and 13 in 2023. Before a medicine or combination therapy is approved by the FDA, it is not available to patients outside of a clinical trial.
Trial results also provide evidence that can change the way doctors make decisions. For example, a trial might help investigators discover if the treatment works better when certain biomarkers, which are features of the cancer or the patient’s individual biology, are present.
What do clinical trials test?
Clinical trials for cancer typically test treatments for safety and for how well they work against the cancer. The focus of the study depends on the phase of the trial and the trial’s design.
For example, drugs make their debut in phase 1 clinical trials, which focus on safety. Phase 2 and phase 3 trials focus on both safety and efficacy, a measure of how well the treatment works against the cancer. In phase 2 and phase 3 trials, efficacy is often measured in comparison to current standard of care treatment. Phase 3 trials involve many participants who are followed over a longer period so that they can provide statistically significant evidence to the regulators at the FDA about the safety and efficacy of the therapy being studied.
Clinical trials may also aim to answer additional questions, such as whether there are features of a cancer that make a therapy more likely to work for a given patient.
If a drug appears unsafe or if there no signs that it is working against the cancer at least as well as current standard of care, a trial may be stopped early.
Who participates in clinical trials?
Clinical researchers aim to include a diverse set of people who share a common disease. It is essential that clinical trials include people of different races, ancestries, zip codes, and socioeconomic status to make sure that the new treatments being studied will work well for all people.
The percentage of cancer patients participating in trials in the U.S. is quite low, at just 3 percent. Enrolling in a clinical trial is a way for patients to potentially improve treatment for themselves and for many other patients in the future.
Every trial has eligibility criteria that specifies the group the trial is focused on, such as a person’s stage of cancer, type of cancer, or specific biomarkers. The criteria might also state that participants cannot have certain unrelated conditions due to safety concerns or that they cannot have taken specific drugs previously.
Some patients worry that they won’t qualify for a trial and that will rule out all clinical trials. That is not the case. Each trial’s eligibility requirements are unique, so being ineligible for one trial has no bearing on eligibility for another.
Learn more about common myths and misunderstandings about clinical trials.
Who funds clinical trials?
Clinical trials can be funded in multiple ways.
Some trials are paid for by the pharmaceutical company that manufactures the therapeutic being tested. In that case, the company typically provides full funding for the trial. The trial isn’t conducted by the pharmaceutical company. Rather, clinical providers who specialize in clinical research and patient care carry out the trial. There are strict regulations about how trials are conducted and how data is handled ensure that the trial results can be trusted.
Other trials, such as investigator-initiated trials, rely on the investigator to find funding. In these trials, investigators like those at Dana-Farber come up with an idea based on what they see every day in the clinic and in research labs. For instance, the investigator might want to combine two drugs that might work better together than either alone, or that might work together to stave off resistance to treatment. The investigator can seek funding from the companies that make the drugs they want to test and must fill in the remainder with grants and philanthropy.
Trials are also funded by the National Cancer Institute (NCI). Dana-Farber is part of the Experimental Therapeutics Clinical Trials Network and has a federal grant enabling the Institute to conduct studies sponsored by the NCI through the National Clinical Trials Network.
Where can I learn more about clinical trials at Dana-Farber?
Learn more about clinical trials at Dana-Farber Cancer Institute.
About the Medical Reviewer
Dr. Meyerhardt received his MD from Yale School of Medicine in 1997. He completed a residency in internal medicine at Beth Israel Deaconness Medical Center, in Boston, followed by a medical oncology fellowship at DFCI. He joined the Gastrointestinal Cancer Center at DFCI in 2002..