Answering Common Questions, Revealing Benefits of Cancer Clinical Trials

Many myths surround clinical trials and their potential risks and benefits. Below, I answer some common questions to combat myths with facts to help illustrate the importance and potential benefits for patients considering participating in cancer clinical trials.

What are clinical trials?

Clinical trials are research studies that involve humans who volunteer to evaluate new treatments, drugs, vaccines, medical devices, behavioral interventions, or screenings. They are the best approach to discovering whether certain drugs, treatments, or devices are safe and effective for patients. All medical therapies or screenings must undergo clinical trials before being approved by the Food and Drug Administration (FDA) for regular use in the United States. Clinical trials are also performed to compare newer treatments to the current standard of care or to improve on them.

What are the benefits of clinical trials?

The most important benefit of participating in a clinical trial for patients is having early access to potentially new and successful treatments or screenings before they are available to the general public. For example, many cancer patients who participated in a chemotherapy or immunotherapy study before FDA approval received revolutionary treatments before they became available to cancer patients who did not participate in the trial.

Secondly, patients participating in a clinical trial are treated under safety guidelines and regulations established by federal and state agencies and local institutional review boards (IRB), which originated in the 1974 National Research Act.

Third, even though no promise of benefit is guaranteed with a clinical trial, patients are closely followed by a highly trained team of clinicians, nurse practitioners, nurses, and other clinicians to advance research, offering patients an added layer of medical care and support.

Finally, information from clinical trials often leads to breakthrough treatments or improved screening or treatment guidelines, and participation benefits future patients.

Are clinical trials safe?

The short answer is yes. Before any clinical trial can be made available to patients, it must go through federal and state agencies and local IRB to ensure the risk-benefit ratio is acceptable. Patients who participate in clinical trials receive additional care and support. Their care team consists of highly trained specialists in advanced research who are available every step of the way. If a patient has side effects, this team of specialists acts immediately to mitigate these symptoms, even if the patient has health concerns unrelated to the clinical trial.

Any clinical trial testing a drug, device, vaccine, or screening method must be submitted to the FDA as a proposal for approval well before recruiting patients to participate. Once the FDA approves the experimental drug or device, animal studies (not involving human beings) can proceed in a preclinical setting. After a degree of safety has been determined in preclinical testing, the studies are approved to move to human testing through different phases of clinical trials.

What are clinical trial phases? How many are there, and what does each phase test?

There are four phases of clinical trials. Phase I cancer clinical trials have been responsible for great strides in improving cancer outcomes. This phase of the study evaluates the dosage and safety of a drug in a small number of patients. It helps us determine the safest dose to treat cancer and recognize potential side effects. Phase II clinical trials test the effectiveness of the drug or device in a larger group of patients with a specific cancer to further determine and confirm the side effects of the treatment. Phase III clinical trials are performed to verify the effectiveness of a drug or combination with standard therapy compared to patients treated with just the standard treatment alone. The participants in the phase III study are divided into a test group and a standard treatment group to ensure the results are not biased. This phase further ensures the safety profile of the new treatment. Finally, a phase IV clinical trial evaluates the long-term effects of a therapy after its FDA approval in clinical practice.

As you can see, patient safety is the top priority during all phases of clinical trials.

Will I be getting a placebo when enrolled in a clinical trial?

The misguided fear of being part of a control group and possibly receiving a placebo that does nothing to treat the disease keeps many patients from participating in clinical trials, which is a myth detrimental to cancer research. The truth is even control group participants receive the current standard care for their disease. This misinformation stalls progress on treatment advances and may cost some patients a chance at a longer life.

I use the "no placebos" message as a key part of my mission to improve clinical trial participation, especially among minority communities, who make up a significant portion of Karmanos' patient population.

Are clinical trials only for people with no other treatment options?

Because there are different phases of clinical trials, it is not true that clinical trials are only for people with no other treatment options. Clinical trials are available for different stages of cancer and various cancer types. For example, there are trials for newly diagnosed cancers where one drug or a combination of therapies is used as the first line of treatment. There are clinical trials for patients who have already been treated for cancer up to four times in the past with no cure, plus trials that treat recurrent cancer after successful initial treatment.

More than half the drugs used today in cancer treatment were initially tested at Karmanos Cancer Institute in Detroit, where I work, in phase I trials. The key to quickly developing new and more effective cancer therapies is breaking through misconceptions among the patient community and referring physicians, who are sometimes reluctant to recommend clinical trials due to similar myths. Since 1991, the cancer death rate has declined by 33%, mainly attributable to gains from cancer research, such as clinical trials. This fact alone is why continued participation is critical. Through a combination of outreach and personal involvement, my mission is to contribute to the growth of participation in phase I trials at Karmanos by 10% each year.

I encourage you to ask about participating in a clinical trial or discuss potential participation in a clinical trial with a family member or friend who has been diagnosed with cancer. Know that these promising new treatments are available to clinical trial patients before they become standard of care, and your participation helps move forward cancer care as we know it for your family, friends and neighbors who may be faced with cancer in the future.

Wasif Saif, M.D., MBBS, is a medical oncologist at the Barbara Ann Karmanos Cancer Institute. He is also the leader of Karmanos' Phase I Clinical Trials Multidisciplinary Team (MDT), Director of the Eisenberg Center for Translational Therapeutics, co-leader of the Gastrointestinal and Neuroendocrine Oncology MDT, and professor in the Department of Oncology at Wayne State University School of Medicine.

Originally published in the September 2025 News From SPOHNC Newsletter.