8 Questions to Answer Before Going On a Clinical Trial

Publication
Article
CUREWinter 2020
Volume 19
Issue 1

Anyone who wants to enroll in a clinical trial should first weigh all the details. Patients should ask key questions like these before signing up.

Whether a patient has received an initial diagnosis of cancer or experienced a recurrence, the treatment possibilities may include clinical trials. Some patients seek out trials because they believe that an experimental regimen could result in better health outcomes. Others are interested because of the close monitoring provided by medical staff or because they want to help advance research to benefit patients in the future.

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Searching for trials can be a complex process, as can sorting through the eligibility requirements for those that seem like good prospects. But once a promising possibility turns up, the research shouldn’t stop. It’s vital to consider a number of factors before deciding a particular trial is the right fit.

Elizabeth A. Ness, a registered nurse and clinical trials expert, has experience in advising patients on how to weigh the pros and cons of studies. She is the director of the Office of Education and Compliance at the National Cancer Institute (NCI) Center for Cancer Research, where she educates research teams on writing protocols and conducting clinical trials. She also served as a coordinator for phase 1 trials at Georgetown Lombardi Comprehensive Cancer Center for six years.

Ness filled CURE ® in on what patients should consider as they read about studies and what questions they should ask their physicians or the researchers when evaluating an opportunity to join.

1) What are the goals of the trial, and do they match what I’m looking for?

Patients should get a clear picture of differences between the trial drug(s) and other available treatment choices. They should ask why study investigators think an experimental therapy could be better than standard-of-care treatment approved by the Food and Drug Administration and also why the trial drug might not perform as hoped.

In addition, Ness says, patients should understand the overall objective of a study, including the trial’s phase, and what that means. “For phase 1 trials, patients have to understand that the goal is to look for a safe dose in humans,” she says. “It’s OK to hope the treatment might help, especially if it’s a personalized, or targeted, therapy, but if patients really want something with higher effectiveness and a more known side-effect profile, then phase 1 may not be for them.”

In phase 2 or 3 trials, drugs are tested for effectiveness, sometimes with certain patients getting the experimental treatment and others, for the sake of comparison, receiving a different therapy. In some cases, the trial design prevents patients from knowing whether they’re getting the experimental drug, another aspect they should ask about in advance.

“If they’re going to be randomized to receive standard of care, or standard of care plus the experimental treatment, or even placebo,” Ness says, “those are things they’ll need to understand before anything else. They need to make sure they’re informed about the goal of the study and that they don’t have any false pretenses about what the study is going to do for them.”

2) How intensive is the regimen, and will it fit my schedule?

It’s a good idea to ask about the time commitment required and compare that against the intensiveness of other available regimens. A patient should ask what tests and procedures will be done, whether they will be painful and if they will require hospitalization. Patients should also know how frequently they’ll be expected to visit the trial site for medication, tests and checkups. This information can also be found in the study schedule, part of the informed consent document.

Is the study drug likely to cause short-term or long- term side effects? If so, what kind? Patients should ask these questions, Ness says, and if the answer to the first is yes, find out if they’ll need medications or procedures to ease the side effects. It’s important to know whether that treatment could be done by local doctors rather than at the trial site, which may be farther from home, she says.

Although most appointments for either standard or experimental care occur on weekdays during business hours, Ness says, trial schedules may be more restrictive; for instance, all appointments for participants may be held on a certain day each week. “One of the things patients need to think about is what happens if they’re not able to come by themselves to an appointment when on a clinical trial,” she notes. “What is their support system like? Will that person be able to take time off from work? If the patient will be traveling to and from appointments alone and is unable to drive, will that impact his or her ability to participate? Could the research site pay for a taxi?”

Finally, it’s wise to determine how long participation will last. “Ask what would result in you being taken off the treatment part of the study,” Ness says. “And once you’re off that treatment, how long will you stay on the study? Will, you still need to come in periodically, or will someone call you by phone to see how you’re doing, and for how long?”

3) Will I have to travel to participate?

Although a lot of research is conducted at academic medical centers, studies occur in community settings, too, Ness says. “Community oncologists may have opportunities for patients to participate in clinical trials, either through the NCI or an industry-sponsored study,” she says. “Patients should ask their oncologists if they participate in any trials, because then travel may not be necessary.”

Patients who do plan to travel for a trial should ask investigators how they plan to communicate with the medical professionals back home. “Ask: ‘Will you communicate with my care team so my oncologist knows what this study is about, in case I have a complication and can’t get here?’” Ness says. To make sure all doctors are informed, she encourages patients to carry a card that indicates that they’re a on a clinical trial and provides investigator contact information. Patients should also know who on the trial team they can call if they have questions after routine business hours.

4) What financial costs will I bear if I join?

Although the pharmaceutical company sponsoring a trial typically pays for the drug on behalf of participants and most health insurers pay for routine care, there may be costs that insurance doesn’t pick up, Ness says.

“Before the trial happens, there’s a coverage analysis that outlines for the research team what will be billed to a third party and what the company or sponsor would have to pay for,” Ness says. “Patients should ask what they will be expected to fund.”

One gray area involves who will pay for medication needed to quell side effects caused by an experimental drug, she says, or hospitalizations due to those effects. “There are other hidden costs,” she adds. “Patients often don’t go by themselves, so this can include lost work for them and their caregivers, as well as any travel expenses not paid by the trial site or charitable programs such as the American Cancer Society’s Hotel Keys of Hope.

5) What does the informed consent document say?

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“The biggest thing patients should do, even though it seems daunting, is read the informed consent document,” Ness says. “They should write questions on it, highlight the things they don’t understand, and come back and ask the researchers those questions. Too often I see patients ready to just sign.

"If they really read the document, it will answer a lot of their questions about the drug to be tested and the goals and particulars of the trial.”

6) Could I end up getting placebo instead of real medicine?

In cancer clinical trials, patients generally get placebo only when the standard of care would be to not give any medicine, according to Ness. “For ethical reasons, oncology tends to not use a lot of placebos, but every once in awhile, we might — for instance, when we give standard of care plus an experimental therapy or placebo,” she says. “Typically, the design then may be blinded so no one knows what treatment they’re getting, but if their disease progresses, they can cross over to the other study group and receive what they didn’t receive before. This should all be clearly stated in the title of the protocol and the consent but ask whether you’re going to be getting the study drug or placebo and whether you will be randomized or not.”

7) Is it risky for me to participate in a clinical trial?

Both standard and experimental treatments bring risks, but less is known about the potential side effects of investigational drugs. Deciding whether to take on the risk of a clinical trial “is going to be very personal based on a patient’s (medical options,) age and where they are in their life cycle,” Ness says.

“Somebody younger who is raising a family may decide that they’re willing to take something of greater risk because the benefit to them personally is greater: They’re trying to live to be able to see their children grow up. Somebody older may not have that same feeling or same need, so it’s extremely personal, and I don’t think there’s a right answer.”

8) How do I feel about participating?

“Patients need to make sure they take the time they need and not feel pressured by family or the research team to participate,” Ness says. “I’ve heard investigators say, ‘We only have one more slot left at this dose level, and then it will be four weeks until another one opens up.’ Patients feel pressured to participate, and that’s not what you want, because the patient may not be happy or able to complete the study because they didn’t have time to think and prepare.”

It’s crucial that patients understand that participation is voluntary, and they can withdraw at any time, for any reason, without jeopardizing their care, Ness adds. “Sometimes they don’t want to disappoint their family or doctor or may not understand that they have the right to withdraw. They may feel that, by dropping out, they are no longer contributing to science or that their family will look at them as giving up, but that’s not a reason for them to continue their participation, and we have to help them embrace and understand that.”

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