Considerations for Participating In Cancer Clinical Trials

Clinical trials are conducted for all pharmaceutical drugs, but when consumers go looking for them, it’s often in connection with cancer treatments. Patients taking an aggressive stance against their disease may believe clinical trials offer the best (sometimes only) hope of a cure. A clinical trial may be the only way you can get early access to a breakthrough medical treatment of the future — but it’s a gamble, not a sure thing. As such, it is important to know as much as possible about what you are getting into, so you can stack the odds in your favor.

According to Roy S. Herbst, MD, PhD, professor of medicine and professor of cancer biology at the University of Texas MD Anderson Cancer Center in Houston, it’s important to understand that the clinical trial is another aspect of cancer treatment, not a winning lottery ticket. If your current treatment isn’t working or isn’t hopeful, a clinical trial is a reasonable choice. But, “it’s not a substitute for good clinical care — rather, it should be considered in the context of your current standard of comprehensive management by your oncologist,” says Dr. Herbst.

FIRST, AN OVERVIEW

Clinical trials are research studies that explore a new drug or treatment in three distinct phases, each with its own parameters, prior to FDA approval. In a Phase I trial, often the first human trial, the safety and the dosing level of a new drug are tested in a small group of patients (usually between 20 and 80). The trial examines the pharmacologic action of the drug in humans and its side effects for different doses. In a Phase II trial, the drug is further tested for safety and for efficacy in a larger group of people (100 to 300). Once preliminary efficacy has been demonstrated, the drug is tested in a Phase III trial among several hundred to thousands of patients to further explore its safety and efficacy and whether or not it is better than standard treatment for a specific type of disease.

Phase III trials attract the most attention among cancer patients since they offer the opportunity for treatment with new drugs that already demonstrate some promise. But not everyone can be in a trial, nor are they guaranteed to get the drug being tested. To be in the trial, patients must first meet the eligibility criteria, which is often very specific and may therefore be limiting. Once they are accepted, patients may be randomly assigned to different treatment groups, depending on the phase of the trial. In a Phase III trial, the “experimental” or “test” group receives the new drug, while the “control” group receives either the standard treatment for that specific type of cancer or a placebo (an inactive pill, liquid or powder). In some trials, called a “single-arm” study, researchers are aware of who is taking what drug, while in others, called “double-blind,” neither the participants nor the research team knows what the participants are receiving. Note: It is very rare for a cancer patient participating in such research to receive nothing at all in a clinical trial, even if he or she is in the placebo group.

WHAT IF A CLINICAL TRIAL ENDS EARLY?

Sometimes trials are halted early, for a variety of reasons. One possibility is that one treatment is found to be vastly superior to the other, while another is exactly the opposite — a treatment is found to be dangerous. When trials end early because of positive results, the patients receiving the beneficial treatment will continue to get it, and typically, so will the rest of the patients. If the results are negative, the drug is immediately stopped.

IF YOU’RE CONSIDERING A CLINICAL TRIAL

The first step for anyone with a worrisome cancer prognosis is to get a second opinion, which may be very different from what you’ve been told. In fact, according to one study, the second opinion altered the course of treatment for 30% to 50% of breast cancer patients. This is especially important if you’ve been told your cancer is difficult to treat or incurable, said Dr. Herbst. “ Ask about the efficacy of standard therapy for your cancer. If you’re not happy about the risk versus reward of the standard therapy, then you may want to consider available clinical trials.” The decision-making process can be complicated, he acknowledged, suggesting it might be a good idea to get a third, even a fourth opinion — all options are worth exploring if it’s your life that’s hanging in the balance.

  • Go where the experts are. Make sure your team captain, so to speak, is an oncologist who knows what’s going on everywhere, not just in the local hospital. It may be worth traveling some distance to find the best expert on your specific illness. Similarly, he/she may encourage you to consider traveling to another state or across the country for access to specialized treatment or trials that look promising. (See the resources listed below to learn about the available tools for evaluating cancer centers.) Be aware that different cancer centers participate in different clinical studies, and you aren’t necessarily limited to what’s going on where your doctor practices.
  • Take charge, if you want to treat your cancer aggressively. “Read everything you can about the center and the trial, and meet everyone on the medical team and be sure you feel comfortable with all of them,” urges Dr. Herbst. Plan to ask questions and write down the answers, as you may not be able to remember all the details later. In fact, it’s a good idea to bring someone with you to the appointment. A friend or family member can help take notes and may think of things you haven’t considered. And, says Dr. Herbst, if a physician or researcher doesn’t answer all the questions you ask — don’t work with them.
  • Ask questions. Key questions to ask include:
    • Have there been other studies that evaluated this drug, and if so, what were the results?
    • How do the risks, side effects and benefits compare with my current treatment? Is it possible this new drug can harm me?
    • What are the chances that I will receive a placebo if I participate in the study?
    • Who are the health care professionals who will take care of me during the study? How will my own oncologist and other members of my cancer team be involved?
    • Will involvement require an extra time or travel commitment on my part?
    • How might the study affect my daily life?
    • Will I have to be admitted to the hospital for any parts of the study?
    • How will I be informed about new risks that may be identified during the trial?
    • If the treatment is working for me, can I choose to continue getting it even after the study ends?
    • Who pays for my care during the trial? Will my insurance cover the costs? If not, what will it cost me?
    • Are there other patients already taking part in the study whom I could speak to?
    • Will I be able to find out about the results of the study?
  • Take time to make your decision. “Often the way people cope with the news that they have cancer is to do everything they can, immediately — but you should fight that tendency,” said Dr. Herbst. “You don’t have to make a decision about a clinical trial the same day you hear about it. Take time to think about it.” Weigh the treatment potential in terms of risks and benefits, on its own, and relative to staying the course with your current treatment.

FINDING A CANCER CLINICAL TRIAL

You can’t assume that your doctor is aware of all clinical trials that relate to your disease. There are just too many of them. So, you’ll need to do some research on your own. Dr. Herbst recommends the following sites:

The National Cancer Institute (NCI) maintains a database of clinical trials. You can search the list by the type and stage of cancer, by the type of study (for example, treatment or prevention) or by geographic location. You can access the list at www.cancer.gov/clinicaltrials or by calling 1-800-4-CANCER.

The National Institutes of Health (NIH) maintains a database of clinical trials, including non-cancer ones, at www.clinicaltrials.gov.

EmergingMed provides a free and confidential matching and referral service to patients looking for clinical trials at www.emergingmed.com. Note: Clinical trial matching firms are businesses, built on revenues from receiving a fee for listing studies or a “finder’s fee” from those running the studies. The bottom line: Buyer beware!

CenterWatch is a publishing and information services company that maintains a list of both industry-sponsored and government-funded clinical trials for cancer and other diseases at www.centerwatch.com.

Major cancer centers usually offer lists of clinical trials on their Web sites. You can find the major cancer centers closest to you on the American Cancer Society Web site at www.cancer.org/docroot/FTC/ftc_0.asp.