Clinical trials: 5 things you should know

Clinical trials are how progress is made in medicine. “They’re really essential to drive our ability to deliver better cancer care,” says Funda Meric-Bernstam, M.D.

Enrollment in a clinical trial is entirely voluntary, but cancer patients can benefit from joining one while also helping future patients. The insights we gain from clinical trials today helps us improve treatment options that will benefit other patients.

Clinical trials are complex, though, and there are a lot of misconceptions surrounding them. Here's what you should know before you join a clinical trial – or decide it’s not for you.

Clinical trials are safe

“Safety is the most important thing,” says Ecaterina Ileana Dumbrava, M.D. Because patients on clinical trials are the first ones to receive experimental medications, there are many safeguards in place to ensure they’re not harmed.

In addition to the routine assessments cancer patients receive for standard treatment, clinical trial participants typically need additional clinical visits, lab work, imaging scans and biopsies.

Also, you’re not just communicating with your oncologist and your cancer care team. Patients on clinical trials also have continuous contact with a research team to ensure they’re doing well on the new drug.

“We’re talking to patients, often weekly, to see if they’re experiencing any side effects and just checking in to see if they have any questions,” Meric-Bernstam says. Your clinical trial team also provides guidance on next steps if the patient does experience any side effects. “There's a lot of help built in with clinical trials to make sure that patients are getting optimal care,” Meric-Bernstam adds.

But the focus on safety doesn’t start there – it happens well before patients are enrolled. “The FDA doesn’t come in just at the end of the trial to approve a drug; they approve the drug before it can be even be administered to patients in the trial,” Dumbrava says.

At MD Anderson, clinical trials are designed by experts and go through several rounds of approval to ensure they’re safe for patients. A proposed clinical trial receives its final approval from the Institutional Review Board (IRB), a committee made up of physicians, nurses, researchers, patients and lawyers.

Clinical trials occur in phases

There are four phases of clinical trials, and each has its own goal. When a new drug or a new drug combination is developed, it's tested in Phase I clinical trials. These usually have a small number of patients, about 15 to 50. “These trials are designed to determine the safety of the drug or drug combination, and what dose to use in following trials,” Meric-Bernstam says. “We’re also looking efficacy, and identifying which patients are the most likely to benefit from the drug, and, therefore, be enrolled in future studies."

Phase II clinical trials may focus on a specific cancer type and examine how it responds to the experimental drug or procedure. These trials may enroll patients with specific diseases or based on certain test results called biomarkers.

Phase III clinical trials test whether a new treatment is better than what’s being used as the standard treatment. At this point, the drug or procedure may go to the FDA for approval, but studies of the drug aren’t done. A fourth phase (Phase IV) reviews the new treatment’s long-term benefits and side effects.

Clinical trials don’t just examine new drugs

Although many clinical trials study new medicines, they help improve all aspects of cancer care. They can study new ways of dispensing treatment, explore a new dosage, test a new drug combination or examine a drug’s success in treating different cancer types.

They also focus on ways to prevent cancer or a recurrence as well as ways to reduce treatment side effects. “We want to save more lives, but we also want to maintain patients’ quality of life,” Dumbrava says.

Clinical trials don’t limit your access to other care

“No matter what the scenario, often the best treatment choice is a clinical trial,” Meric-Bernstam says. She notes that if there’s already an effective treatment approach, clinical trials offer the opportunity to build on that to make it more effective. They usually require patients to receive known effective treatment options before starting something that’s more investigational. If there aren’t clear, effective treatments, clinical trials offer access to something new that may work.

“Unfortunately, we don’t know if an experimental treatment will be effective, but that’s true for some standard treatments, too,” Meric-Bernstam says.

If your disease isn’t controlled on a clinical trial, your doctor will stop your participation and may offer another treatment option. In some cases, you may enroll in another clinical trial.

“By joining a trial, you’ll usually be getting a treatment that we think may be better than our current options,” Meric-Bernstam says.

Dumbrava agrees, adding, “At minimum, you’ll be receiving the best treatment available.”

And even if you personally don’t see clinical benefit from the experimental treatment, your participation still helps advance cancer research.

Clinical trials are for patients at all stages of cancer

Although all clinical trials have criteria for the participants, clinical trials are available to patients at all stages of cancer. The eligibility criteria, which is meant to ensure patients’ safety, may include the patient’s age, gender, cancer type and stage, previous treatments and overall health.

Your doctor is the best resource for knowing your treatment options. But don’t hesitate to ask if there are clinical trials that are available to you.

“It's also an opportunity to pay it forward and truly contribute to better outcomes for future patients,” Meric-Bernstam says.

Request an appointment at MD Anderson online or by calling 1-877-632-6789.