Background: Clinical trial participation in the United States remains low, hampering development of and access to novel therapies. To assist patients and their oncologists with identifying relevant and available blood cancer clinical trials and reducing enrollment barriers, The Leukemia & Lymphoma Society created its Clinical Trial Support Center (CTSC), a free, telephone-based service led by nurse navigators.
Objectives: To understand patient- and caregiver-reported perceptions and experiences with this navigation service.
Methods: Eligible participants were English-speaking, adult blood cancer patients or caregivers who resided in the United States or Canada and had a telephone interaction with the CTSC between May 2018 and April 2019. A 53-item survey assessed demographics, clinical characteristics, experiences, satisfaction, and enrollment outcomes. Interviews were conducted with a subset of survey respondents to capture individual narratives.
Results: Of 474 eligible patients/caregivers, 131 completed the survey (28% response rate). Most patients/caregivers reported contacting the CTSC to identify a trial they might be eligible for (81.7%), for general information about clinical trials (78.6%), about a trial recommended by a healthcare provider (21.4%), or one found through another source (19.1%). In addition to seeking help with identifying trials and learning about trials, patients/caregivers reported discussing with the nurse navigator insurance/coverage issues (25%) and how to talk with their healthcare provider about trial participation (24%). More than 93% of respondents reported that the nurse navigator gave them the information and support needed to make an informed decision about trial participation. After speaking to a nurse navigator, nearly 29.6% reported enrolling in a trial; the most common reasons for nonenrollment were that their doctor did not recommend it (33.7%), not eligible for any existing trial (30.3%), no trial available at a convenient location (24.7%), and chose a different type or plan of treatment than offered by trial(s) (23.6%). Interview respondents (n=10) described how the navigation enhanced their sense of control over the treatment decision-making process.
Conclusion: Amid an overwhelming treatment landscape and continued low clinical trial enrollment rates, this clinical trial navigation service effectively provides blood cancer patients and caregivers support with identifying relevant trial options and facilitating enrollment if the patient so chooses in collaboration with their healthcare team. Further inquiry is needed to understand how to integrate independent navigation programs such as this directly into clinical workflows so that more patients and oncologists can be assisted, especially among patients from groups underrepresented in clinical trials.
Cancer clinical trials are critical for advancing medicine toward more effective and less harmful therapies and to enhance patient access to novel treatments. Yet, the clinical trial participation rates among cancer patients remain low, averaging 5% to 8% nationally; the rates are lowest within community centers at 6% on average, and highest within academic centers at about 15% on average.1-4 Common barriers to clinical trial access include provider and infrastructure capacity, insufficient patient–provider communication, restrictive trial eligibility, financial burden, travel, and attitudes and preferences toward specific treatment decisions among both providers and patient.4-11 When making decisions about treatment with their healthcare team, patients may face challenges with information overload, recalling information, health literacy, and feeling that they do not have enough time to ask questions.12,13 Even among eligible patients, clinical trial options are often overlooked in treatment planning discussions.1,6,14
When making decisions about treatment with their healthcare team, patients may face challenges with information overload, recalling information, health literacy, and feeling that they do not have enough time to ask questions.
Patient navigation is one tool that has been used in oncology settings for over 30 years to facilitate patient-centered care.15 Patient navigation can expand access to clinical trials for patients by enhancing their knowledge about trial options and reducing barriers to enrollment by providing social and instrumental support.16,17 To help blood cancer patients and oncologists identify relevant and available clinical trials and address barriers to enrollment, The Leukemia & Lymphoma Society (LLS) created a navigation service in 2017, the Clinical Trial Support Center (CTSC).18 This free service is provided via telephone by nurse navigators, who are master’s-level oncology nurses with extensive knowledge of hematologic malignancies, treatment methods, stem cell transplantation, and genomics. The service aims to increase opportunities for participation in clinical trials, facilitate informed choices about participation, and mitigate barriers to enrollment.
The CTSC’s approach is grounded in the idea that providing high-quality, timely information to cancer patients and their family members is an essential component of patient-centered cancer care,19,20 which facilitates patient involvement in care and informed decisions. The CTSC nurse navigator begins by providing the patient/caregiver with information about what clinical trials are and completes a comprehensive nursing assessment of the patient’s diagnosis, genetic profile, medical history, financial situation, insurance coverage, support network, and ability to travel. The nurse navigator then curates an individualized list of potential trials that takes into consideration these unique patient characteristics. The clinical trial search is done utilizing a proprietary database that includes all trials available on clinicaltrials.gov but also includes up-to-date information gleaned from interactions with principal investigators, study sponsors, and site contacts. Patients and caregivers are encouraged to share this list with their healthcare team, emphasizing the importance of shared decision-making. Nurse navigators may help with enrollment by reaching out to site research nurses and trial investigators and assisting with logistics to address enrollment barriers. Navigators can also provide support to patients while on a trial.
In a previous publication, we described the CTSC’s navigation approach in detail and reported factors associated with clinical trial enrollment among new cases opened between October 2017 and 2019.18 To further understand patient- and caregiver-reported experiences with this navigation service, in 2019, LLS commissioned RTI International to conduct a mixed-methods evaluation. This paper describes the results of that evaluation, the goal of which was to better understand patients’ and caregivers’ needs when exploring, considering, or seeking clinical trial options and to inform ways to improve this service.
We developed a 53-item survey instrument for patients and caregivers that assessed self-reported demographic and clinical characteristics, how they first learned about the CTSC, reasons they decided to contact the CTSC, topics discussed with the nurse navigator, the frequency and type of interactions, perceived helpfulness and satisfaction with the service, potential areas for improvement, and enrollment outcome. New items developed for the survey were tested and revised based on cognitive interviews (n=3), which assessed participant understanding of the questions, response options, and flow of the instrument.
The survey instrument was programmed using Voxco Survey software and was administered online to all blood cancer patients and caregivers who had a telephone interaction with the CTSC between May 1, 2018, and April 30, 2019, who were English speaking, aged 18 or older, and resided in the United States or Canada. Caregivers included family members and friends. We sent a survey invitation to all eligible participants with an email address on record with LLS and 3 reminder emails 1 week apart from July 2, 2019, until August 5, 2019. No incentives were provided.
We conducted telephone interviews with a subset of survey respondents (n=10) to capture individual patient and caregiver narratives. We invited respondents who represented a diverse group with regard to blood cancer subtype, age, patient versus caregiver, and years since diagnosis. Interviews collected in-depth information about their needs, whether and how the nurse navigator met their needs, their level of comfort with the nurse navigator, any barriers experienced enrolling in a clinical trial, the most valuable elements of their interactions, whether and how the nurse navigator affected their ability to make informed decisions, and recommendations for improving CTSC services.
This study was deemed not to be human subject research by the RTI International Institutional Review Board because it was considered a program evaluation based on the US Code of Federal Regulations (45 CFR 46.102) (IRB ID: STUDY00020549).
We conducted univariate and bivariate analyses of survey data using Stata. We compared responses to selected questions across subgroups of respondents, including patients and caregivers, year of diagnosis, diagnosis, and sociodemographic characteristics using Mantel-Haenszel chi-square tests.
Qualitative interview data were organized by theme to enhance the understanding gained through the survey. We developed primary themes inductively to facilitate alignment with survey data and developed subthemes deductively using an analytic matrix.
A total of 474 patients and caregivers were eligible for this study; 131 patients and caregivers completed the survey (28% response rate). Sixty-four percent (n=84) identified as a patient with blood cancer, and 36% (n=47) identified as a caregiver of someone diagnosed with blood cancer. Most respondents identified as female (52%, n=66), White (92%, n=114), and had at least a 4-year college degree (71%, n=89). Four percent identified as Hispanic or Latino (n=5). Forty-seven percent were 65 and older (n=60). Table 1 provides respondents’ demographic characteristics.
Diagnoses reported reflected a variety of subtypes; the most common diagnoses were in the categories of leukemia (47%, n=62) and lymphoma (31%, n=40). Five percent (n=7) were diagnosed in 2019, 27% in 2018 (n=35), and 67% in 2017 or earlier (n=86).
Ten survey respondents agreed to participate in a 30- to 45-minute interview about their experience with the CTSC. Seven interviewees were patients aged 40 to 80 years. Three were caregivers aged 33 to 64 years.
We conducted a nonresponse analysis to compare respondents to survey nonrespondents. Those who submitted partial responses were coded as respondents. The 2 groups were compared by recency of interaction with a nurse navigator, primary disease category, and respondent type (patient or caregiver). There were no significant differences by disease category or status as a patient or caregiver. However, we found significant differences in response rate based on recency of interaction: respondents with more recent interactions with the CTSC were more likely to respond to the survey (P<.0001, degrees of freedom 1 [df=1]; Table 2).
Respondents were asked to think about the reasons why they first decided to contact the CTSC (Table 3). The majority were seeking help to identify a trial for which they or their loved one might be eligible (n=107, 81.7%) or information about clinical trials (n=103, 78.6%); some were seeking information about a clinical trial recommended by a healthcare provider (n=28, 21.4%) or a trial found through another source (n=25, 19.1%). Ten percent reported that a CTSC nurse navigator made first contact with them (n=13). Most respondents reported first learning about CTSC services through the LLS Information Resource Center (26.0%, n=34), the LLS website (24.4%, n=32), an online search (12.2%, n=16), a healthcare provider (10.7%, n=14), or family and friends (n=12, 9.2%).
Interview data indicated that respondents contacted the CTSC for help understanding and navigating the clinical trial process, which was described as a tedious and often fruitless endeavor for patients and caregivers to attempt alone. Interview respondents reported difficulties in being able to identify trials through their oncologist or web-based resources, which they reported as hard to navigate, difficult to understand, and often outdated. Many of those interviewed also reported contacting the CTSC for education about the steps involved in selecting and enrolling in a clinical trial and what to expect after enrolling (eg, side effects).
Many respondents communicated extensively with their nurse navigator beyond their initial communication. About one-third reported having 4 to 9 calls with the nurse navigator (37.2%, n=48), and about 1 in 10 (9.3%, n=12) reported having 10 or more calls. Over half (51.6%, n=64) received 4 or more emails from the nurse navigator. One in 10 reported 10 or more emails (10.5%, n=13).
In addition to talking with their nurse navigator to identify trials and ask questions about trials, some patients discussed insurance or insurance coverage issues (n=31, 25%), how to talk with a doctor about participating (n=30, 24%), and compassionate use/expanded access (n=22, 17%); see Table 4 for all topics discussed.
More than two-thirds of respondents who received a list of clinical trials from the nurse navigator (68.9%, n=73) discussed or shared this list with family or friends, and 50.9% (n=54) discussed or shared the list with a doctor. Some (13.2%, n=14) reported that they did not discuss or share the information with anyone. Respondents who did not share the information included caregivers, patients in a “watch and wait” phase who were not ready to enroll, and patients who used the information from the CTSC to validate or clarify what their doctor already had shared.
Interview data revealed that after an initial assessment, the nurse navigator often provided the patient/caregiver with education about clinical trials and/or with a list of clinical trials to consider. Nurse navigators explained the open trials as well as more general information about trial stages and procedures, and the different types of therapies used in certain trials.
Those interviewed also reported learning about how to reach out to a clinical trial site to identify required eligibility screening. Patients and caregivers further noted learning about expected side effects of treatments and what procedures a clinical trial would utilize. Nurse navigators sometimes helped clarify clinical trial options, strategize backup options based on important considerations (eg, drivable distance from home), and identify ways to overcome barriers to enrollment (eg, lodging or travel assistance, navigating insurance issues like whether the trial is covered or in-network, and insurance appeals). One patient described the nurse navigator’s perseverance—her ability to “keep going when I was too exhausted or too overwhelmed” to continue searching for potential trials. Another patient emphasized the value of the many interactions in complement with physician input, stating:
And then I made a huge list of questions, and I called her back. And we talked through what the different phases of the trials meant. And…which ones were open, and the different types of drugs….And then I talked to my doctor. And I guess there was a lot of back and forth, which is why it was so amazing and supportive. Because any time I had a question, she was willing to get on the phone with me and chat about it.
Nearly one-third (29.6%; n=37/128) of respondents reported that they or a loved one enrolled in a clinical trial after speaking with a CTSC nurse navigator (see Table 5 for a comparison of respondent characteristics that enrolled or did not enroll in a clinical trial). Among these, 51.4% (n=19) stated that the nurse navigator helped them find the trial. Respondents who spoke with a navigator reported that the navigator was “very helpful” or “extremely helpful” in obtaining an appointment at the trial site (73.5%, n=36/49), finding financial support for transportation/travel (82.1%, n=32/39), and insurance issues (78.8%, n=26/33).
More than 93% of respondents reported that the nurse navigator gave them the information and support they needed to make an informed decision about clinical trial participation (n=121, 93.7%), and nearly as many (n=116, 89.8%) reported that they felt more knowledgeable about clinical trials after the call. Moreover, many respondents stayed in contact with the nurse navigator after they enrolled in a clinical trial (67.6%, n=25). When asked why they continued to stay in contact, most indicated that it was to “let them know how I/or my loved one is doing” (n=17, 68.0%); others also stayed in contact for “emotional support” (n=10, 20.4%).
A number of interview respondents described how navigation enhanced their sense of control over the decision-making process. As one patient stated:
Just having that ownership and feeling that I made this [treatment] decision really helped me have the resilience to get through all of the stuff I had to get through.
Another patient emphasized the value of having ownership while experiencing the difficulties of treatment:
…A bone marrow transplant is terrible. All of it is really hard. And…having that ownership and feeling that, ‘I made this decision,’ really helped me have the resilience to get through all of the stuff I had to get through.
The most common reasons for nonenrollment were that the doctor did not recommend the patient to enroll in a clinical trial or recommended a different treatment plan (n=30, 33.7%), not eligible for any existing clinical trial (n=27, 30.3%), no trial available at a convenient location (n=22, 24.7%), and the patient/caregiver chose a different type or plan of treatment than offered by trial(s) (n=21, 23.6%). See Table 6 for the reasons for nonenrollment.
One patient described the impact of navigation despite the not being eligible for any trials at the time:
Oh, she made me feel a lot more secure about it. She gave me the information that I needed. She was willing to go to bat for me….If there was one, then she would help me find the right one. So she gave me confidence in her, and I will definitely go when I’m eligible and a trial comes up....
When asked about their experience regarding the conversation with the nurse navigator, the majority reported it as extremely helpful (84.3%, n=107/127), extremely comfortable (95.3%, n=122/128), and extremely clear (90.5%, n=115/127). A total of 98.4% of respondents reported that the nurse navigator’s ability to answer questions was great or excellent (n=124/126), and 100% reported being treated with courtesy and respect (n=128/128). Nearly all reported that the CTSC nurse navigator spent enough time with them (99.2%, n=127/128). See Table 7 for patient perceptions of the nurse navigator.
Nurse navigators provided support equally across key demographic groups. Satisfaction did not vary significantly by age, gender, race, ethnicity, education, or whether the respondent lived in an urban or rural area. Similarly, no differences in satisfaction by clinical characteristics were reported, such as diagnosis or time since diagnosis. There were no significant differences in satisfaction with the service among those who did versus did not enroll in a clinical trial after speaking with a nurse navigator.
Those interviewed described the positive impact that the nurse navigator had on them; the 2 quotes below exemplify what was expressed by many:
She always had time….Whenever I called her, either she wouldn’t pick up and then she’d call me back, or if she picked up, I knew that she had at least 15 or 20 minutes to spend with me. She was never trying to get me off the phone. She always wanted to be sure that I got what I came for. And really, it felt like having somebody in my corner. And that support was wonderful. It’s something that I would wish for anybody going through this. – Caregiver
You just really need someone you feel like you can lean on. Someone who knows what’s going on that you can trust. That’s what I got, and it’s priceless. – Patient
The blood cancer treatment landscape is complex, advances rapidly, and available treatments can be influenced by specific disease biomarkers, prior therapies, insurance, cost, and where someone lives. Understanding and making decisions about treatment can be overwhelming.12,13 For many, the opportunity to be part of a clinical trial is not part of this process. Even when they do learn about clinical trials—or a specific clinical trial—as a treatment option, patients often lack the information, resources, and support needed during the decision-making process. This study shed light on why patients and caregivers reach out to this navigation service provided outside the site of care, what they discussed with the nurse navigator, reasons for trial nonenrollment, experiences working with the navigators, and how they helped.
Findings demonstrate that some patients and caregivers reached out to the CTSC with a trial that had been recommended by their healthcare provider. These people often shared how the service helped them navigate the process of selecting a trial or eliminating barriers to enrollment. They used the CTSC to better understand and weigh the pros and cons of treatment options and explore solutions for managing the cost of care, suggesting that this navigation service can supplement information and support provided at the site of care.
However, most patients sought assistance to identify a trial that they were eligible for or to inquire about a trial they had found through a source other than their healthcare provider. Unfortunately, the topic of clinical trial participation is often overlooked during treatment planning discussions, even among eligible patients.1,21 Our findings showed that this navigation service can play a role in discussing with patients how to talk with their doctor about clinical trials.
About one-third of respondents ultimately enrolled in a trial, and similar to other research findings,5 salient reasons for nonenrollment included provider recommendation, ineligibility, location, and choosing a different treatment plan. Regardless of whether a patient was enrolled, patients and caregivers expressed considerable satisfaction with the service. The goal of the CTSC is not to enroll every patient with blood cancer into a clinical trial but rather to help them uncover all treatment options so they can assess, in collaboration with their healthcare team, whether a clinical trial is right for them. Study respondents indicated that the nurse navigator provided a safe space for patients and caregivers to ask numerous questions to develop an understanding of their or their loved one’s clinical trial options, to better understand clinical trials, and feel more confident in their ability to make an informed decision about clinical trial participation.
About one-third of respondents enrolled in a trial, and similar to other research findings, salient reasons for nonenrollment included provider recommendation, ineligibility, location, and choosing a different treatment plan.
Barriers to clinical trial participation are numerous, and clinical trial nurse navigation can support patients and oncologists in overcoming them. Navigation services such as the CTSC can provide support to institutions and oncologists by identifying relevant trial options, reducing logistical barriers, and facilitating enrollment. Further inquiry is needed to understand how to integrate support from independent navigation programs directly into physician workflows so that more patients and oncologists can be assisted, especially patients from groups underrepresented in clinical trials.
The study sample represented a group of primarily White, English-speaking patients with higher levels of education. Results may not be generalizable to underrepresented populations. The composition of this sample demonstrates that continued attention is needed to better engage underrepresented populations with navigation programs like this to enhance opportunities for all.
Amid an overwhelming treatment landscape in which most patients do not have access to and enroll in clinical trials, this free, national nurse-led clinical trial navigation service for patients with blood cancer can help patients and their caregivers identify and understand relevant trial options and facilitate enrollment, if the patient so chooses, in collaboration with their healthcare team. Nurse navigators assisted with making connections with the trial site, finding financial support for transportation/travel, and addressing insurance issues (eg, answering questions about coverage and appeals). Patients and caregivers reported that navigation gave them the information and support they needed to make an informed decision about clinical trial participation. About one-third did ultimately enroll in a clinical trial; the most salient reasons for not enrolling included provider recommendation, ineligibility, location, and patient choosing a different treatment plan. Both patients who enrolled and those who did not reported benefiting from the nurse navigator’s help. Future efforts should focus on expanding access to this service to a broader population of blood cancer patients and healthcare providers and replicating this type of free service for other cancer types. Further inquiry is needed to understand how to integrate independent navigation programs such as the CTSC directly into clinical workflows so that more patients and oncologists are assisted, especially among patients from groups underrepresented in clinical trials.
Funding: This study was funded by The Leukemia & Lymphoma Society.
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