Trials & Tribulations: Pursuing a Clinical Trial Requires Determination and Education

After receiving a cancer diagnosis, some patients want to consider every option before deciding on treatment. Rather than waiting to enroll in a clinical trial as a last resort, they may be willing to explore the possibility upfront, seeking trials that include standard treatment in addition to the experimental approach. But identifying and ultimately participating in a study can be a challenge. Beyond learning the potential risks and benefits, patients should also understand the barriers around becoming involved.

Patients may be reluctant to participate in trials for a variety of reasons—they feel they don’t know enough, they might not meet strict eligibility requirements or they worry about how to cover the costs. Yet, as strange as it may seem, physicians are often a roadblock between patients and trials. According to a 2010 report by the Institute of Medicine, primary care doctors might not mention cancer trials as a possibility because, as generalists, they often are unfamiliar with them—understandable, considering the National Institutes of Health track more than 122,000 clinical trials in 179 countries.

Oncologists might not mention them either—or may even discourage participation—if they object to less traditional treatments or worry about the additional time, cost and paperwork burdens they would bear on behalf of patients who enroll, a legitimate concern in this age of financially squeezed healthcare. A survey published in 2011 in the Journal of Oncology Practice found that 44 percent of 213 trial-eligible cancer patients did not recall discussing trials with their doctor.

If a patient’s oncologist doesn’t mention trials or balks at the subject, the patient should get a second opinion, says Richard Schilsky, MD, section chief of hematology/oncology at The University of Chicago Medicine, noting that an oncologist once advised one of his own family members not to become a “guinea pig.”

“Second opinions are worthwhile. Patients deserve to hear different points of view and have as much information as possible,” Schilsky says. “We know that patients have strong emotional attachments to their physicians; sometimes it’s a doctor they’ve been with for a long time. But at the end of the day, patients must do what hopefully will be best for them.”

Even if the patient’s oncologist is cooperative, finding an appropriate trial—much less one within a reasonable geographic distance—can be daunting, given every patient’s unique medical history, disease variables, demographic characteristics, eligibility criteria and the vast array of trials. David Spigel, MD, a clinical oncologist and director for the Lung Cancer Research Program at the Sarah Cannon Research Institute in Nashville, Tenn., encourages his patients to explore clinical trials databases on their own first. Later, during his free time, he digs through and narrows their findings to one or more studies he believes are most applicable—in effect, working as a translator, navigator and advocate for the patient.

“It helps me find a way to help,” Spigel says. “That makes things easier for everybody.”

[Read "Recruiting for Clinical Trials Takes Center Stage"]

Although no centralized, searchable database with up-to-the-minute information exists, the most comprehensive one is ClinicalTrials.gov from the National Institutes of Health. Others include the National Cancer Institute’s (NCI) list; TrialCheck.org, developed by the Coalition of Cancer Cooperative Groups; proprietary databases at drug and biotechnology companies; and the International Federation of Pharmaceutical Manufacturers & Associations Clinical Trials Portal.

Databases can be difficult to navigate, even though most provide instructions. It helps if patients know all the details of their disease and any prior treatments before searching, so they can correctly refine the search parameters. The NCI also provides live help online and by phone (800-422-6237) if patients have search-related or other questions. Patients also can try a free clinical trials matching service, like those at the BreastCancerTrials.org, EmergingMed.com and ResearchMatch.org. However, it’s important to ask other such services if they charge a fee, where they get their clinical trials information, whether they keep personal information confidential or if they represent a drug brand or pharmaceutical company.

Jim Ferrell, 59, a retired police officer in Enumclaw, Wash., says he has enjoyed “an embarrassment of riches” since he received a diagnosis of stage 4 peripheral T-cell lymphoma, a rare disease, in 2008 and participated in a trial of the drug Istodax (romidepsin). To qualify for the trial, participants had to have undergone at least one prior therapy. Ferrell had received four cycles of a chemotherapy regimen, followed by four cycles of a different regimen. When an imaging test detected four cancerous nodes two months later, Ferrell and his doctor began exploring the clinical trial option.

I haven’t had a map, yet every turn I’ve taken has been the correct one, and every door I’ve reached has been unlocked.

Ferrell says Istodax has not only been highly effective against his disease, he also didn’t encounter any obstacles along the clinical trials path and remains lymphoma-free. Ferrell strongly encourages other cancer patients to take full advantage of databases and to enroll in a suitable trial, even if it means switching to a more supportive oncologist.

“I haven’t had a map,” he says, “yet every turn I’ve taken has been the correct one, and every door I’ve reached has been unlocked.”

Finding one or more trials for which a patient may be eligible is only half the battle. To determine whether a study is appropriate and the best choice, and to avoid any unwelcome surprises down the road, patients should gather as much information as possible by talking with their doctor and contacting the research team. Although studies have shown that cancer treatment expenses within and outside of clinical trials are about the same, insurance coverage varies: Patients could end up being billed for a study-related treatment, imaging test or doctor visit that falls outside the “routine care” definition. Physicians aren’t prepared to determine what a given insurer will or will not cover, says Howard Sandler, MD, chairman of radiation oncology at Cedars-Sinai Medical Center in Los Angeles. But oftentimes, he adds, patients can successfully appeal trial-related charges. The trial consent form should distinguish between standard and investigational procedures.

[Read "Understanding Clinical Trials"]

Beginning in 2014, a provision in the Patient Protection and Affordable Care Act (PPACA) requires individual and group health insurance plans implemented after 2013 to cover routine costs associated with approved clinical trials. Seventeen states and the District of Columbia have existing mandates that meet the PPACA provision: Alaska, Arizona, California, Colorado, Georgia, Indiana, Iowa, Kentucky, Maine, Massachusetts, Nevada, New Jersey, Ohio, Oregon, Tennessee, Texas and Vermont.

Of course, if a study is located out of town, there are additional expenses—for transportation, accommodations, meals and the like—that insurance won’t cover. The American Cancer Society lists many outside sources of financial aid for these and other costs. (Visit cancer.org.) Patients might also check the Marti Nelson Cancer Foundation for a list of cancer-specific patient-advocacy groups and foundations, to see if they offer assistance. (Visit canceractionnow.org and click “Cancer Resources.”) Some entities, including the Lazarex Cancer Foundation (lazarex.org), earmark funds specifically for clinical trial enrollees.

There’s always an element of chance when it comes to dealing with cancer. But overcoming the hurdles associated with identifying and participating in a clinical trial depends more on determination and information-gathering than luck.