Patient advocates' role in clinical trials
Perspectives from Cancer and Leukemia Group B investigators and advocates
Patient advocates are increasingly involved in cooperative group trials, single-institution cancer programs, and peer-review of research applications. The purpose of this study was to evaluate the role and value of patient advocates from the perspective of Cancer and Leukemia Group B (CALGB) advocates and investigators.
An online survey was sent to current and past (within 5 years) patient advocates and investigators.
Response rates were 72.7% (16 of 22) for advocates and 56.4% (102 of 181) for investigators. Patient advocates were more likely than investigators to report the following: the clinical trial process benefited from advocate involvement on committees (100% of advocates vs 72.1% of investigators; P < .05), advocates contribute to protocol development (92.8% vs 33.8%, respectively; P < .001), the cultural appropriateness of protocols (21.4% vs 10.4%, respectively; P < .05), advocates assist with patient accrual (78.6% vs 23.4%, respectively; P < .001), and advocates add value to concept development and protocol review (100% vs 63.2%, respectively; P < .001). Over half of advocates and investigators reported gaps in patient advocate knowledge and suggested that additional clinical trials training was needed. To improve clinical trials, advocates suggested their earlier involvement in protocol development and increased support from investigators. CALGB investigators recommended improving patient advocate selection and communication skills training:
The majority of patient advocates and investigators perceived benefits from advocate involvement in the clinical trials process; patient advocates placed more value on their role than investigators. The current results indicated that strategies to improve advocacy training and advocate-investigator communication may further enhance the role of patient advocates, and future studies that clarify the role of advocates in the prioritization and development of protocol, consent, and education materials, and on patient accrual, are warranted. Cancer 2012. © 2012 American Cancer Society.
Patient advocates are involved increasingly in cancer clinical research through participation in cancer cooperative groups, single-institution cancer programs, and peer-review of research applications to foundations and government. Currently, there are hundreds of patient advocates assisting with various aspects of cancer research in the United States.1 Patient advocates are included in the Cancer and Leukemia Group B (CALGB) to incorporate the perspectives of patients and their families in the development and delivery of cancer clinical trials. Patient advocates who have participated in CALGB are cancer survivors or family members of cancer patients, and only a few CALGB patient advocates had experience participating in a clinical trial. Patient advocates are recruited to participate in CALGB after being nominated by a CALGB investigator or CALGB patient advocate, or occasionally they have contacted the CALGB office to volunteer. Patient advocates receive a CALGB orientation guidebook and undergo training about basic concepts associated with clinical trials research that was developed by CALGB patient advocates. CALGB patient advocates are assigned to a specific disease committee (eg, breast cancer committee) usually based on their own disease or that of their loved one. Patient advocates do not communicate about specific clinical trials to individual patients; however, CALGB patient advocates have discussed the benefits of clinical trials participation to the general public.
CALGB patient advocates may assist the clinical trial process by: 1) ensuring that information about trials and consent forms are written at a literacy level that is understandable; 2) promoting patient accrual by assisting with trial design, eligibility criteria, cultural appropriateness, study procedures, and the development of educational materials; 3) identifying and addressing potential ethical issues in clinical trials; 4) promoting patient safety and confidentiality throughout the process of clinical research; and 5) translating clinical trial findings for the general public. However, sometimes, the roles of patient advocates may be in potential conflict, just like researchers, and both patient advocates and investigators must be aware of “therapeutic misconception.”2
In an effort to improve the success of cancer trials, the Institute of Medicine recently called for greater involvement of patient advocates in the design of clinical trials and in patient recruitment efforts to encourage patient acceptance and participation in clinical trials.3 However, despite the presence of patient advocates in cooperative groups and research institutions, there is limited information on the role and impact of patient advocates. We sought to evaluate the role and value of patient advocates in the cancer clinical trials process from the perspective of CALGB patient advocates and cooperative group investigators (committee chairs, co-chairs, study chairs, and protocol coordinators). We believe that this evaluation is an important first step toward developing strategies to improve the role of patient advocates in the cooperative group cancer clinical trials process.
MATERIALS AND METHODS
The research study team, which included a patient advocate (D.E.C.), developed a Web-based survey to assess the perceived role and value of patient advocates as well as the experiences of the advocates and investigators with patient advocates in the CALGB clinical trial process. The short survey that was developed for this study was reviewed for content by experts; however, the survey was not tested for reliability or validity. Survey items included demographic characteristics and addressed patient advocates' knowledge, training, clinical trial experience, communication with investigators, and activities within the CALGB process. Most items were based either on a 4-point Likert scale, from 1 (not at all) to 4 (highly) or from 1 (none) to 4 (high), or on a 5-point Likert scale, from 1 (not important) to 5 (very important) or from 1 (never) to 5 (always). However, several items were open-ended, allowing participants to insert comments. Surveys were developed for patient advocates and investigators with the same questions stated from a slightly different perspective.
The surveys were posted online at SurveyMonkey, and a custom link to the survey was sent in an email request from the CALGB Group Chair (R.L.S.) in the Fall of 2009. E-mail requests were sent to current and past (within 5 years) patient advocates (n = 22) and investigators (n = 181). A reminder e-mail was sent 1 week after the first e-mail request to improve participation rates. The brief survey took approximately 10 minutes to complete. The study was approved by the Institutional Review Board of the Dana Farber Cancer Institute.
Summary statistics (means, standard deviations, and frequencies) were calculated to describe the survey respondents. Patient advocate responses were compared with investigator responses using Fisher exact tests and t tests and were considered significant at P < .05. Statistical analyses were performed by CALGB statisticians using SAS statistical software (version 9.1.3; SAS Institute, Inc., Cary, NC). All written comments by patient advocates and investigators were coded by a trained member of the research team (M.L.K.) and were grouped into 3 major categories based on the common themes that emerged from the review.
The response rate was 72.7% for patient advocates (16 of 22) and 56.4% for investigators (102 of 181) (Table 1). Participants predominantly were men (69 of 118 participants; 58.5%), they ranged in age from 30 to 69 years (86 of 118 participants; 72.9%), and they self-reported as non-Hispanic whites (90 of 118 participants; 76.3%) who were currently serving (84 of 118 participants; 71.2%) as CALGB patient advocates or investigators (committee chairs, co-chairs, study chairs, and protocol coordinators). Patient advocates were older than the investigators (P < .05) but had been with CALBG for fewer years (range, 1-12 years; mean, 4.8 years) than investigators (range, 1 to ≥30 years; mean, 13.6 years; P < .01).
Table 1. Characteristics of Cancer and Leukemia Group B Patient Advocates and Investigatorsa
|Sex|| || |
| Males|| 8 (50)||61 (59.8)|
|Age, yb|| || |
| <30||0|| 2 (2)|
| 30-49|| 2 (12.5)||31 (30.4)|
| 50-69|| 9 (56.3)||44 (43.1)|
| ≥70|| 3 (18.8)|| 1 (1)|
|Race|| || |
| White||14 (87.5)||76 (74.5)|
| African American||0|| 1 (1)|
| Asian||0|| 2 (2)|
| Eurasian American||0|| 1 (1)|
| Eastern European|| 1 (6.3)||0|
|Ethnicity|| || |
| Non-Hispanic||15 (93.8)||79 (77.5)|
|Length of CALGB association|| || |
| Mean [range], yc||4.8 [1-12]||13.6 [<1 to ≥30]|
Cancer and Leukemia Group B Patient Advocates' and Investigators' Perspectives
There were significant differences in the perceptions of CALGB patient advocates and investigators about the value of patient advocates in the clinical trial process. Patient advocates were more likely to report benefit to the trial process by having patient advocates assist with: 1) serving on disease and modality committees (100% vs 72.1%; P < .05), 2) protocol development (92.8% vs 33.8%; P < .001), 3) making trials culturally appropriate (21.4% vs 10.4%; P < .05), and 4) patient accrual to trials (78.6% vs 23.4%; P < .001). In addition, patient advocates were more likely than investigators to report that, in general, patient advocates provided value to the clinical trials process (100% vs 63.2%; P < .001).
Investigators were frequently unaware of patient advocates' roles in CALGB. In particular, they reported being unaware of advocates' role in concept or protocol review during trial development (32.5%), input on strategies to increase patient accrual for ongoing trials with poor accrual (28.6%), concept or protocol review for cultural appropriateness (52%), and assistance on specific requests (eg, help with identifying patient concerns about a specific trial) during the clinical trials process (49.4%).
There appeared to be broad agreement, without statistical differences between patient advocates and clinical investigators, that patient advocates have: 1) the ability to represent cancer patients (100% vs 88.5%, respectively), 2) knowledge about cancer and its treatments (100% vs 89.6%, respectively), clinical trial design and conduct (100% vs 90.6%, respectively), and clinical trial ethics training (100% vs 93.7%, respectively), and 3) first-hand experience as a cancer patient (93.3% vs 82.3%, respectively) or as a family member of a cancer patient (80% vs 68.1%, respectively).
Patient advocates reported mixed degrees of active contribution and involvement in cooperative group processes. A minority of patient advocates and investigators (20% and 21%, respectively) reported that questions are directed to the patient advocates during committee meetings, and only 33.3% of patient advocates and 31.5% of investigators reported that patient advocates volunteer comments during the meetings. However, when advocates participate, their input is valued. Almost two-thirds of patient advocates (60%) and investigators (65.2%) reported that comments by the patient advocates are considered during discussions about clinical trials or that patient advocates' comments are respected by committee members (patient advocates vs investigators, 60% vs 68.5%). In addition, a majority of patient advocates (53.3%) and investigators (60.4%) reported major gaps in patient advocates' knowledge and training about cancer treatment, clinical trial design, and clinical trial ethics.
Three categories emerged from patient advocates' and investigators' written comments. Categories were general observations, important roles of the patient advocates, and recommendations to improve the role of patient advocates in the clinical trial process. Examples of comments from patient advocates and investigators illustrating the 3 categories are listed in Table 2.
Table 2. General Observations, Important Roles, and Recommendations to Improve the Role of Patient Advocates in the Clinical Trials Process by Cancer and Leukemia Group B Patient Advocates and Investigators
| General observations|
| • Dedicated and take their role in CALGB seriously|
| • Participation in process is often limited because of a specific individual (committee chair) or disease-specific committee|
| Important roles|
| • Provide perspective from cancer patients about barriers to trial participation|
| • Share clinical trial findings and ongoing trial information with cancer patients|
| • Earlier involvement in the clinical trials process|
| • Support from investigators|
| • Better communication between patient advocates and investigators|
| • Continuing education and training is necessary|
| General observations|
| • Level of knowledge and sophistication varies considerably|
| • Comments based on own experiences rather than the majority of cancer patients|
| Important roles|
| • Disseminate clinical trial findings to community|
| • Raise awareness of practical and ethical issues from the cancer patient perspective vs scientific contribution|
| • Earlier involvement in the clinical trial process|
| • Input by developing a more formal feedback process|
| • Improve patient advocate selection process|
| • Continuing education and communication skills training (proactive voice) is necessary|
Amid calls for greater patient advocate input and participation in clinical trials, we sought to evaluate the perceptions and roles of advocates both among themselves and among investigators in the CALGB. We identified opportunities for improving knowledge among investigators and for enhancing the roles of advocates.
The observation that patient advocates perceived their input and the value of their role in the clinical trials process as more beneficial than investigators was not a surprising study finding. By definition, patient advocates have chosen to represent the voice of the patient in the clinical research process,1, 4-7 and the respondents to this survey had voluntarily chosen to work with CALGB to contribute to cancer research. In addition, patient advocates would be aware of the multiple aspects of clinical research in which they participated; whereas, as demonstrated in our survey, only a minority of investigators was aware of some patient advocate activities. Still, this discrepancy, with up to one-third of investigators suggesting that the role of patient advocate does not add value, suggests at minimum a need to clarify the role and potential value of patient advocates to all investigators and a need to further explore why the potential value of patient advocates is not appreciated or realized in some settings. It is particularly concerning that a substantial minority of investigators was not aware of the roles that patient advocates may play (or do play) in the CALGB clinical trials process. Clearly, improved communication between advocates and investigators within cooperative groups is warranted to actualize the full potential of advocacy involvement.
An important observation from this study is also the lack of diversity among the patient advocates and investigators. This mirrors the lack of diversity among patients who are recruited to clinical trials and is also a reflection of the absence of diversity among scientific investigators.8, 9 Therefore, we believe it would be advantageous for clinical trials groups to make a special effort to engage patient advocates from minority and underserved populations. Currently, as the National Cancer Institute consolidates the 9 cooperative groups into 4 entities and makes adjustments to the review process,10 patient accrual from minority and underserved populations and limited representation of minority investigators remains a critical issue in the clinical trials process.11 By increasing the diversity of patient advocates, there is potential to increase trial accrual from these population groups by having specific issues addressed in the design of a clinical trial and by having supportive voices that encourage trials in various communities.
An unanticipated study finding is that only approximately 20% of patient advocates and investigators report that questions are directed to patient advocates, and only one-third of patient advocates and investigators report that patient advocates volunteer comments during committee meetings. This finding also may be related to several factors, including the concerning finding that >50% of patient advocates and investigators report major gaps in the patient advocates' knowledge and training (this varies significantly by advocate), perceived power differences between individuals during committee meetings, or suboptimal communication skills among patient advocates and investigators. This information, coupled with the finding that most patient advocates and investigators report that comments by patient advocates are considered and respected by committee members, suggests that including communication skills training in the education of patient advocates and investigators may provide patient advocates with a stronger “voice” in committee meetings. One investigator indicated in a written comment that patient advocates appeared to provide a reality check, making sure that the concepts seem reasonable to both investigators and patients and that patients will be willing to participate in a proposed trial.
In response to our findings, the Alliance for Clinical Trials in Oncology, a cooperative group consortium of the CALGB, the North Central Cancer Treatment Group, and the American College of Surgeons Oncology Group, has developed and is starting to implement an improvement plan for the patient advocates' role in the clinical trials process. The CALGB Executive Committee (EC) has begun to explore the following proposals: 1) movement of a formal patient advocate feedback process for concept reviews so that it may occur before CALGB EC approval and patient advocates' names will be listed on protocols; 2) assignment of a patient advocate to provide input to every study result summary; 3) identification of additional training topics and regular educational sessions will be conducted at meetings; 4) conducting regular meetings with patient advocates and protocol coordinators; 5) development of proactive communication between committee chairs and patient advocates, including providing updates about patient advocate activities to committees; 6) developing criteria to evaluate potential patient advocates; and 7) planning communication skills training for all CALGB members.
This study should be interpreted in the context of its limitations. Because it was a cross-sectional survey, changes in perceptions of the patient advocates' role in the clinical trials process over time could not be documented. Although we attempted to maximize the study participation rate by sending 2 e-mail requests to complete the survey from the CALGB Group Chair at the time of the survey, differential response rates of participation for the patient advocates and investigators may bias the findings. However, response rates were quite high among both the patient advocates and investigators. There is no information available for the evaluation of nonresponders compared with the study responders. Survey items were designed for this study and were not tested for reliability or validity. Finally, the survey was completed among CALGB patient advocates and investigators, and the findings may not be generalizable to the role of patient advocates and investigators in other settings.
In conclusion, this study fills a gap in the available literature regarding the role of patient advocates in the cancer clinical trials process by describing the perception of their role from the perspective of patient advocates and investigators. The majority of patient advocates and investigators perceive benefits from patient advocate involvement in the cancer clinical trials process; however, they have identified areas that may improve the role of the patient advocates' role in the trials process. Strategies to improve patient advocate selection, training, and advocate-investigator communication may further enhance the role of patient advocates, which ultimately will improve the cancer clinical trials process. Future studies are warranted that clarify the role of patient advocates in the prioritization and development of protocols, consent, educational materials, and patient accrual. Ultimately, efforts to optimize the role of patient advocates in the cancer clinical trials process will improve the process and help solidify the general publics' trust of the clinical trials process.
No specific funding was disclosed.
CONFLICT OF INTEREST DISCLOSURES
The authors made no disclosures.