Clinical trials are the only avenue that advances in medicine occur. Every approved drug or treatment must go through the clinical trial process. With the increasing pace of science and technology advances, there were 19 drugs with new approvals for cancer treatment in 2018 according to “CenterWatch” (n.d.). Despite the excitement of tapping into the latest science that may appear on the news, less than 5% of adults diagnosed with cancer participate in research studies (Unger, Cook, Tai, & Bleyer, 2016). Ironically, more than 50% of children under the age of fifteen diagnosed are treated on a clinical trial (Unger et al., 2016). So why the discrepancy? Numerous myths persist about clinical trials leaving cancer patients unsure and uninformed about their available treatment options.
Myth #1: Clinical Trials are only for patients who have exhausted all their treatment options.
This is one of the most common misconceptions people have about clinical trials. When I was working as a clinical research nurse, after introducing myself, this was the first thing I addressed.
“First, let me reassure you that the reason we’re talking to you today about clinical trials is NOT because your cancer is so bad we don’t know how to treat it. It’s NOT because you don’t have any other treatment options.”
I could literally see the relief come over the patient and their family member’s face. “It’s not?” was the typical response stated by the patient in disbelief.
“No, it’s not. In fact, you have more treatment options which is why your doctor asked me to explain this one and help answer questions you may have,” I replied.
FACT: Clinical trials are available for every stage of cancer, including prevention trials for people who are high-risk for developing a specific cancer. While clinical trials can provide treatment options for patients who have not benefited from standard approved drugs, it is more often used to explore additional options earlier in the course of treatment. Patients are not giving up standard treatment by participating in a research study, as this is often part of the trial. As science advances and makes new discoveries about how cancer cells behave and respond to treatment, researchers use that information to develop new medicines believed to work better than the current treatment available. By participating in clinical trials patients get access to these state-of-the-art drugs that aren’t available yet. Many clinical trials take the standard treatment and compare it to the standard treatment with the new “study drug” added to it. Being offered a clinical trial does not mean there are not options for treatment, as a matter of fact, it gives patients more options.
Myth #2: If I participate in a clinical trial, I’ll get a placebo or “sugar” pill that won’t help me.
FACT: Placebos are rarely given in clinical trials. Most studies compare the current standard treatment with a new study drug or treatment that is believed to be better than what is currently available to patients. This comparison is often accomplished by half of the patients in the trial getting the standard treatment (exactly what they would receive if not on a study) and the other half receiving the standard treatment plus the new study drug or treatment. The hope is that adding the study drug or treatment will result in greater benefit for patients.
In general, the only time that placebos are used are when there is no standard treatment. For example, imagine an early stage cancer is typically treated with surgery alone with no further medication given afterwards. However, we still see patients who have a recurrence later. A clinical trial might investigate if giving patients a study drug after surgery would help decrease the number of patients who have their cancer recur. In this scenario, there is no standard treatment after surgery. A trial may be designed to have half of the patients receive a study drug after surgery and the other half get a placebo (or no treatment, which is standard) after surgery. If a clinical trial is going to use a placebo, it will be clearly stated in the consent form discussing the study.
Myth #3: I’ll have lots of extra costs that my insurance won’t cover.
FACT: In the United States, federal law requires insurance companies to cover routine costs of therapeutic trials, things you would need regardless of whether you participated in the trial, such as regular blood work or x-rays and scans to evaluate treatment effectiveness. In fact, Medicare has been covering clinical trial costs for nearly a two decades, since 2000. If there are any extra testing or costs not covered under the federal law, the study sponsor typically covers these. The research nurse and your doctor will review any potential costs with you prior to making your decision about participating. The social worker or your oncologist’s office can also check with your insurance company to verify your treatment will be covered.
Myth #4: I have to go to a big university or large cancer center to be on a clinical trial.
FACT: Cancer clinical trials are offered in a variety of locations, including large academic or university centers, community cancer centers, oncologist’s offices, and even through VA (Veterans Affairs) hospitals. While large university or academic centers may have more clinical trials available (particularly for rare tumor types), community cancer centers often participate in clinical trials so their patients can receive the same state-of-the-art care at home where they have their support network.
When you see your cancer doctor, be sure to ask if they participate in clinical trials and if there are any clinical trials available that may give you additional treatment options. If a trial that you want to participate in is available at another cancer center, your doctor can contact their office and send your medical records to see if you may be eligible for the study.
Myth #5: I’ll be treated like a “guinea pig” if I participate in a clinical trial.
FACT: Many clinical trial participants feel they receive extra attention and have more people on their care team looking out for them, such as research nurses or study coordinators. Whether you receive treatment on a clinical trial or receive standard approved treatments, there is no way to guarantee how an individual will respond or react. The body is complex and while one person may respond well to an approved drug, the next person may not respond at all. A doctor can tell patients about potential side effects of a drug, including what is common down to what is rare, but it does not mean that every patient will experience the same ones. There is no difference on a clinical trial. There is no guarantee that it will be better than the standard treatment alone, but there is reasonable belief that it will be based on the latest science and data. Doctors and the research nurse can explain the potential side effects from common, through those that are rare, but patients may respond differently just like with standard treatment.
Through surveys, many clinical trial participants have reported extremely positive patient satisfaction regarding respect for patients, adequate time spent with patients, explanation of their role in the study, answering questions fully and staff friendliness (Pflugeisen, Rebar, Reedy, Pierce, & Amoroso, 2016). They reported contributing to science and helping others with similar conditions as well as the hope that their own condition would be improved as the primary motivation for participating in a clinical trial (Pflugeisen et al., 2016). In a colon cancer trial, participants surveyed reported that trial participation was worth it (95%), they would do it again (90%), and (95%) would recommend it to other people (Sloan et al., 2011).
Once patients enroll in a clinical trial, they remain in control of their care. They have the right to decline participation or “quit” the trial at any time. Doctors would then switch to the standard approved treatment for their condition. While this is always an option, if a patient withdraws from a study, their slot does not open back up for someone else. Because trials have a specific number of patients allowed to enroll, once a slot has been taken, whether they complete the treatment or not, it is no longer available. This can mean that someone else will not have the opportunity to enroll. Patients are encouraged to be mindful of this prior to deciding to participate and enrollment in a trial. In addition, patients are updated throughout the trial with any new information as it becomes available, as well as informed of the results of the study after the trial is completed.
The Bottom Line
Clinical trials give patients additional treatment options and access to the latest drugs and procedures. It is the only way that we advance medicine, including cancer treatment. So why are less than five percent of adults diagnosed with cancer participating in research studies (Unger et al., 2016)? A significant gap remains in the general public’s knowledge and understanding of What Clinical Trials Are and many Myths about Clinical Trials persist. It is important to increase our awareness about clinical trials. As a patient, be sure to ask your oncologist if there are any clinical trials available for your condition so you understand all of your treatment options. Discussing the differences between the standard approved treatment and the study treatment allows patients and caregivers to understand their options prior to making an informed decision about what treatment is best for them.
Centerwatch. (n.d.) FDA Approved Drugs for Oncology. Retrieved from https://www.centerwatch.com/drug-information/fda-approved-drugs/therapeutic-area/12/oncology
Pflugeisen, B., Rebar, S., Reedy, A., Pierce, R., & Amoroso, P. (2016). Assessment of clinical trial participant patient satisfaction: A call to action. Trials, 17. doi:10.1186/s13063–016–1616–6.
Sloan, J. A., Mahoney, M. R., Sargent, D. J., Hubbard, J. M., Liu, H., Basch, E. M., Shields, A. F. Chan, E., Goldberg, R. M., Gill, S., Kahlenberg, M. S., and Alberts, S. R. (2011). Was it worth it (WIWI)? Patient satisfaction with clinical trial participation: Results from North Central Cancer Treatment Group (NCCTG) phase III trial N0147. Journal of Clinical Oncology, 29:15_suppl, 6122–6122.
Unger, J. M., Cook, E., Tai, E., & Bleyer, A. (2016). The Role of Clinical Trial Participation in Cancer Research: Barriers, Evidence, and Strategies. American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting, 35, 185–198. doi:10.14694/EDBK_156686