New cancer treatments depend on clinical trials to test the safety and efficacy of treating specific types of cancer, but enrollment in clinical trials remains incredibly low, especially among patients from racial and ethnic minority groups.
One of the main concerns that keeps patients from electing to enroll in a trial is that their quality of life will suffer more than if they opted for standard treatment. Patients’ quality of life encompasses many aspects, including their physical, social, and emotional health; their perception of the quality of care they receive; and the financial repercussions of their care.
Many researchers, concerned with understanding why enrollment in clinical trials is so low, are attempting to identify specific barriers to enrollment. Few studies have specifically considered whether patient concerns about clinical trials are legitimate. By directly comparing whether patients enrolled in trials face barriers that patients receiving standard treatment do not, they can demonstrate to patients, clinicians, and other stakeholders if concerns about a trial causing worsening patient-reported outcomes are valid.
Surbhi Sidana, MD, of Stanford University School of Medicine, Division of Blood and Marrow Transplantation and Cellular Therapy, led a study to see if patients with multiple myeloma or lymphoma who enrolled in clinical trials reported similar patient-reported outcome measures as patients not in clinical trials. Specific patient-reported metrics considered included quality of life, quality of care, and financial burden. The study results, reported in JCO Oncology Practice, demonstrated that patients enrolled in clinical trials reported similar outcomes as patients who were not enrolled in clinical trials over a 1-year period.
This study recruited 123 patients at the Mayo Clinic in Rochester, Minnesota, who were newly diagnosed or had experienced relapsed multiple myeloma and were also ready to start a new line of treatment. Participants were put into 2 groups based on whether the new treatment they were starting was a clinical trial (n = 35) or standard of care (n = 88).
From August 2017 to October 2019, the researchers collected patient-reported outcome measures over the course a year. Participants left the study if they had disease progression, changes in treatment, died, or decided to withdraw.
Quality of life was assessed using the Functional Assessment of Cancer Treatment – General (FACT-G) questionnaire, which includes 4 domains asking about participants’ physical, social, emotional, and functional well-being.
Quality of care was assessed using 6 questions adapted from the Consumer Assessment of Healthcare Providers and Systems cancer care survey and focused on communication with clinicians, access to care, and overall satisfaction.
Financial burden was assessed using 10 questions the researchers adapted from the Medical Expenditure Panel Survey, a widely used survey set. The questions asked if participants:
- needed to borrow money or file for bankruptcy related to paying for treatment
- worried about paying medical bills, and
- were unable to pay for their medical bills.
The researchers looked at participants’ changes in these 3 patient-reported outcome measures at 6 time points. Participants completed monthly surveys for 3 months (after recruitment, Month 1, Month 2, and Month 3) and then completed surveys at Month 6 and Month 12. The researchers considered change scores across timepoints and looked at clinically significant changes for the FACT-G, percentages of patients ranking top options for the quality-of-care survey, and comparisons of composite scores for the financial burden survey. They also looked at participants’ views of clinical trials.
Participants in the clinical trial and standard of care groups had similar social and health characteristics as well as initial patient-reported outcome measures. Most patients (77%) were starting their first line of treatment. Participants with multiple myeloma tended to be in the clinical trial group (77%) compared with the standard of care group (28%). Of the participants in the standard of care group, only 34% were offered a clinical trial.
Quality of life did not differ significantly between participants in the clinical trials throughout the 12 months. Across groups, participants reported drops in quality of life for several months followed by gradual increases throughout the remainder of the year, returning to above baseline quality-of-life scores. This trend was true across the physical and functional well-being domains. Participants’ social and emotional domains were more stable over time and did not differ between groups.
Participants perceived their quality of care as “high,” and this perception did not change. There were no differences between the groups’ communication with clinicians, access to care, and overall satisfaction.
Financial burden increased throughout the 12 months for both groups. Between 16% and 19% of participants in the clinical trial and standard of care groups, respectively, reported financial burden. The percentages increased at each timepoint to a maximum of 33% and 49%, respectively. The increases in financial burden did not significantly differ between groups, though the absolute increase in financial burden was higher in the standard of care group.
The researchers concluded that the lack of difference in self-reported quality of life, quality of care, and financial burden between patients enrolled in clinical trials and those receiving standard of care demonstrates this common barrier to clinical trial enrollment is not based on evidence. The data from this study can be used to counter patient concerns about what will happen to them if they enroll in a clinical trial.
Noted study limitations include the small clinical trial group size, a 50% drop-out rate at 12 months, and recruitment at an academic cancer center that may not be generalizable to a community setting.
Oncology nurses can discuss patients’ concerns about clinical trials to understand the reasons that may prohibit them from choosing to enroll. If patients voice concerns about the type of care they will receive, symptoms they may experience, or financial repercussions of trial enrollment, the nurse can counter with evidence that these concerns are comparable, whether the patients enroll in the trial or receive standard of care.