Editor's note: This is the second of a two-part package shedding light on cancer clinical trials. Click here to read part one, which focuses on one woman's experience in a clinical study.
When Dr. Elihu Estey sees a patient at Seattle Cancer Care Alliance, he offers them two choices: standard treatment or a clinical trial.
About 70 percent of his patients choose to enroll in a clinical trial, far higher than the national average of 3 to 5 percent.
Estey, a member of Fred Hutchinson Cancer Research Center’s Clinical Research Division, is an expert in acute myeloid leukemia. His high trial participation rate is in part a reflection of the disease’s grim prognosis: Standard treatment is effective for only about a quarter of the people with that type of blood cancer. In addition, people who seek out care at a research center like Fred Hutch are often hoping to find an experimental therapy, either because earlier treatments failed or they were diagnosed with a particularly lethal cancer in the first place.
But another reason that so many of Estey’s patients go on trials is that he offers them the option.
Not all doctors do.
“We’ll hear from people, ‘My doctor never mentioned this,’ or ‘my doctor won’t help me with this,’” said Judy Petersen, an oncology nurse educator with the National Cancer Institute’s Cancer Information Service, which is based at Fred Hutch.
Cancer researchers are asking with increasing urgency why more adult cancer patients don’t participate in trials after a recent study found that a lack of volunteers—not treatment failure or toxicity—was the main reason 1 in 5 adult cancer trials were never completed.
“We have the treatments we have today because of clinical trials,” said Petersen. “Our standardized, recognized treatments all came from research. So it’s important to educate the public about what clinical trials are.”
That is exactly what Petersen and information specialist Laura Rankin do at the CIS, a free, bilingual cancer information service that responds to more than 10,000 phone calls, emails and live-help chat sessions a month from cancer patients and their families as well as from health professionals and the public.
Petersen and Rankin understand why not everyone is interested in clinical trials. Sometimes, the current standard treatment is the appropriate—and effective—choice.
“It’s very unusual for someone with an early stage, treatable cancer to be asking about a clinical trial,” said Rankin. “It’s often people who have exhausted other options. That’s what motivates them: They think a trial is their last hope.”
But even when the standard therapy is effective, there may be studies on giving an already approved drug at lower doses or on a different schedule, which could improve care.
And for cancers for which no effective therapy exists, clinical trials are the only way to move research forward.
“I wish more doctors would bring up with their patients as part of a balanced discussion,” Rankin said. “It would mean so much coming from the doctors. It would open this opportunity up for people.”
“If we could double that 4 or 5 percent to 10 percent, my goodness, how much faster we could learn,” she said.
Costs can be a barrier
People who call the CIS—much like people who seek out care at academic research centers—often have already overcome that first barrier. They may not know which trials are available, but they know at least to ask.
But callers do have other concerns. Chief among them is costs.
Some patients without health insurance seek out clinical trials because they believe that treatment will be free. But that is not usually the case, said Petersen and Rankin. Even when the study sponsor covers research costs and the cost of the treatment under study, patients—or their insurance companies—are responsible for routine health care costs associated with the study, including any other drugs, procedures or services needed during the trial period.
In addition, there can be hidden costs to participating in clinical trials that affect even people with insurance, particularly people who live far from a major research center.
“If the study requires you to have more frequent trips to the doctor and you’re 50 miles away, gasoline costs pile up, childcare costs add up,” said Petersen.
But not having insurance or other resources doesn’t automatically rule out participating in a trial. The CIS staff just tries to be realistic that care is not free and suggests that patients talk with their doctor and with the study staff about options.
On the flip side, some people with insurance don’t consider trials because they fear that their insurance company won’t cover the costs. Don’t assume—ask your insurance company, said Petersen and Rankin. Although research costs associated solely with data collection or analysis may not be covered, many insurance policies as well as Medicare do pay routine costs that would be covered if the patient were receiving standard treatment.
Under the Affordable Care Act, the health care reform bill passed in 2010, all insurance companies will be required to cover routine costs for patients participating in approved, federally funded clinical trials, unless a policy was grandfathered in and is not subject to the reforms. But other restrictions may apply, such as whether the doctor or medical center overseeing the trial is in the patient’s network. The new health care reform law does not apply to Medicaid; coverage of routine care varies by state.
Not everyone can be a 'guinea pig'
Fear of taking an experimental treatment, or “being a guinea pig,” is a barrier for some patients. But the majority of callers to the CIS have the opposite concern: They fear not getting the new therapy being tested.
Human drug trials are typically divided into three phases. Many of the clinical trials overseen by researchers at the Hutch are Phase 1 trials, which test a new experimental drug or intervention in a small number of people to determine a safe dosage and identify side effects, or Phase 2 trials, which determine whether a new treatment is effective, again in a relatively small number of people. If a drug makes it through these two phases, it moves on to Phase 3, which tests for both safety and efficacy in hundreds or thousands of patients by comparing the outcomes between those taking the new drug and those getting a placebo or standard care.
It’s rare for cancer treatment trials to use a placebo; usually the new treatment is tested against the current standard treatment. But because patients are randomly assigned to one treatment or the other, some patients don’t want to participate because they are only interested in getting the experimental treatment.
“People sometimes think that clinical trials must be better than what’s currently available,” said Rankin. “We have to explain that these trials might result in finding that that treatment isn’t better than standard of care.”
Such patients also have the option of seeking out an earlier phase trial in which only the new therapy is being tested, with the understanding that researchers are at an earlier stage of determining dosage, safety and effectiveness. Estey, for example, works on cutting-edge treatments in Phase 1 or 2 trials, leaving it to others to conduct the larger Phase 3 trials.
Know what to ask
Neither a doctor nor the CIS can tell a patient which trial is best—that is, after all, why a new therapy is under study. But they can discuss which trials are available and also lay out the risks, one of which is that the therapy under study may not work.
The best physicians, like Estey, discuss all options with patients and their families, including standard therapy, clinical trials and no treatment.
In addition, they remind patients that simply learning about a clinical trial doesn’t commit them to enrolling. And even after they enroll, they can drop out.
“Our role is to be neutral, and to provide credible, evidence-based information,” said the CIS’s Rankin. “Sometimes what we’re doing is coming up with questions to consider, questions to ask the doctor to have a deeper conversation about the pros and the cons. It’s important for people to understand and be educated, to know what their options are.”
To contact the CIS, call 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 8 a.m. to 8 p.m. You can also email, participate in online chats or find more information on the National Cancer Institute web site, www.cancer.gov.
Mary Engel is a former staff writer at Fred Hutchinson Cancer Research Center. Previously, she covered medicine and health policy for the Los Angeles Times, where she was part of a team that won a Pulitzer Prize for Public Service. She was also a fellow at the Knight Science Journalism Program at MIT. Follow her on Twitter @Engel140.