Patient Engagement in Neurological Clinical Trials Design: A Conference Summary

Abstract

Objectives

The conference objectives included educating patients and advocates about clinical trials, educating the clinical research community about patient perspectives on participating in clinical trial design, and identifying strategies to increase participation in clinical trial design for neurological disorders.

Design

Observations were noted during a 1‐day conference attended by patients, patient advocates, clinical trial staff, and investigators. The conference offered didactic sessions, small, and large group discussions.

Participants

Conference participants were patients, patient advocates, clinical trial staff, students, and investigators interested in engaging patients in clinical trial design for neurological disorders.

Measures

Conference participants were asked to consider lessons learned that could increase patient engagement in clinical trial design.

Results

We found that there is growing interest in including patients in the design of clinical trials for neurological disorders. Several themes emerged on how to move forward: networking; the multifaceted roles of advocates in research; training and education; creating patient–researcher partnerships; and clinical trials regulation issues.

Conclusions

The conference provided a forum for dialogue regarding stakeholder engagement in the design of clinical trials for neurological disorders. This experience provides a template for replication and dissemination of this conference and informs next steps to accelerate the pathway from dialogue to action.

Background

Many clinical trials in the United States end early due to lack of patient participation and poor enrollment.1 To address poor recruitment and retention in ongoing clinical trials, researchers have often turned to patient engagement strategies and patient advocate groups to increase participation. Increasingly, the biomedical community, led by the cancer research community, has shifted to include patients and advocates as partners in research at earlier stages in clinical trial design.2 As a result, breast cancer and HIV/AIDS research advocates have shown that patients and advocates can play a role in the entire process of clinical trial design, from question development to dissemination of results (ibid). The response in the research community has been a growing willingness to include patients as investigators on grants, engage in community‐based participatory research methods, and find ways of including patients in clinical trial design, recruitment, and participation.3

Patients are the ultimate stakeholders in NIH‐sponsored clinical trials. Designing clinical trials that are pragmatic and informed by clinicians and clinician–scientist stakeholders as well as by patients and patient advocates, is proposed to be a pathway to improve trial design, completion, and results implementation (ibid). In fact, many grant funders are now requiring that patients and advocates sit on advisory committees or serve as coinvestigators in clinical trials. For example, the Patient‐Centered Outcomes Research Institute (PCORI) requires “meaningful and authentic” patient engagement in their funded research and has trained patient advisor members on funding application review boards to judge how well applicants have achieved this goal.4 Additionally, the Clinical and Translational Science Award (CTSA) Programs support investigators and institutions that engage communities in research through partnerships with community‐based groups, public health professionals, and healthcare providers.5

The Neurological Emergencies Treatment Trials (NETT) network Clinical Coordinating Center at the University of Michigan was created by NINDS to conduct confirmatory phase trials in emergent neurological conditions, such as traumatic brain injury, status epilepticus, and stroke. The NETT has been interested in engaging patients as partners in the design of its clinical trials. Although patient participation in clinical trial design is not required for NETT studies, NETT investigators identified patient and patient advocate involvement in the clinical trial development process as a means to improve clinical trial applicability and relevance to the study populations involved. We wanted to learn more about patient perspectives on participation in clinical trials design and assess the level of existing knowledge of patients, patient advocates and clinical investigators in the process. Principal investigators and research staff at the University of Michigan held a free 1‐day local conference to discuss the best ways to educate and engage patients and investigators in clinical trials design for neurological disorders. This is a summary of the conference titled LISTEN Clinical Trials—Learning about Investigator‐Stakeholder Team Engagement in Neurological Clinical Trials: A Conference about Patient Engagement in Clinical Trial Design for Neurological Disorders.

Methods

The primary goal of the conference was to bring together patients, patient advocates, clinicians, and clinical trial design experts for a local 1‐day shared learning workshop to discuss improving patient and advocate engagement in clinical trial design for acute neurological disorders. Topics included challenges in health outcomes, clinical trials design, recruitment through engagement, dissemination, implementation, and translation of trials to health outcomes.

Planning

The conference objectives were (1) to educate patients and advocates about clinical trials; (2) to educate the clinical research community about patient perspectives on participating in clinical trial design; and (3) to identify strategies to increase public participation in designing clinical trials focusing on neurological disorders within the NETT and affiliated institutions.

The conference planning committee members included NETT principal investigators, research staff, and patient advocates. The committee helped to identify individuals, hospitals, departments, support groups, and community‐based organizations to notify about the conference. Several methods were used to invite participants including phone calls, emails, flyers, social media, Website posts, and announcements on University of Michigan, Wayne State, Sparrow Health System, Henry Ford Health System, and community‐based organization membership list serves. Specifically, conference planners engaged federal partners to help disseminate news of the conference nationally. Locally, emails and calls were made to invite investigators and other study team members, patients, advocates, caregivers, community‐based organizations, and support groups in stroke, Parkinson's disease, spinal cord injury, traumatic brain injury, epilepsy, sudden cardiac arrest, and motor neuron diseases. Conference planning committee contacts in nonneurologic fields were also invited.

Expert speakers were invited to give didactic presentations on the importance of engaging patients and advocates as partners at every step of the research process, the basics of understanding the need for and components of clinical trials, and regulation of clinical trials.

Conference

During the conference, small group sessions were held with five to seven participants led by trained discussion facilitators. Each participant was assigned to rotate through two session topics. The first small group was a case study of a neurological clinical trial protocol. The case study was prepared to elicit feedback from participants on the design of the clinical trial protocol. Conference participants were asked to consider the overall design of the trial, the consent process, risks and benefits of participation, and the appropriateness of the trial's outcome measures. The second small group session was aimed at eliciting lessons learned from the conference. Conference participants were asked to consider ways that researchers could recruit patients and advocates to participate in clinical trial design, challenges and benefits of designing patient‐centered clinical trials, and lessons learned about the regulation of clinical trials.

A panel discussion was held during which two experienced patient advocates shared their perspectives and expertise on participating in research advocacy and clinical trial design. There was also time for networking among conference participants and for sharing insights within the larger group of conference participants. Participants were encouraged to ask questions during and after each session to foster discussion. Detailed notes were taken by a designated note‐taker and by trained discussion facilitators. Notes were analyzed for common themes.

Results

A total of 82 people registered for the conference and 59 people (72% of registrants) attended. Of those who attended, 29% identified as advocates, 20% as patients, 17% as investigators, and 17% as other (attendees could choose more than one affiliation).

At the end of the conference, participants returned 24 evaluation forms that detailed best sessions, conference lessons learned, and suggestions for future conference topics. Just under half (46%) of these evaluations were submitted by participants who identified as patient advocates.

Our overall observation was that there is a growing interest in patient and patient advocate involvement in the design of clinical trials for neurological disorders. Although this is a biased sample of patients and advocates who chose to attend the conference, it still suggests that there will be an adequate number of interested laypersons available to meaningfully participate in designing future NETT trials. The level of sophistication among the participants was variable, suggesting that some baseline education and/or mentoring would be needed for both lay person participants as well as clinical investigators. Although we expected that laypersons would want and need some formal education regarding the regulatory environment, conference participants did not find this information useful to them. It was clear that patients and patient advocates are interested in being involved in every aspect of the clinical trial enterprise, and not only in trial design.

Several themes emerged in the small group, large group, and question and answer sessions.

Networking

Conference participants repeatedly voiced their appreciation for networking opportunities between advocates, and between investigators and advocates while at the conference. Many participants also expressed the desire to have more opportunities to discuss and share ideas amongst different stakeholders outside of the conference. Similarly, mentorship between experienced and inexperienced advocates was noted to be a key facet of networking. Some experienced patient advocates highlighted the crucial role that nonprofit community‐based organizations—such as patient advocacy groups—play in linking experienced advocates with inexperienced advocates and researchers with advocates.

The Roles of Advocates in Research

Some participants stated that prior to the conference, they were unaware of the existence of patient advocates in the research arena. Others noted that the role of advocates in research is broader than they had imagined. One participant stated that the most beneficial aspect of the conference was “learning about all the models of patient engagement in research. I did not realize there are so many of us doing this.” In one of the small group sessions, participants suggested that patient advocates could help investigators by reviewing the consent processes for clarity. Others suggested that patients could help to tailor the presentation of the option to participate in the study as a balance between a physician's recommendation and an objective review of the risks and benefits. One participant suggested that the pitfalls of using patient advocates in research should also be discussed.

Training Opportunities

Conference participants felt that there should be more training opportunities for patients and investigators to learn how to engage in discussions around designing clinical trials together. Many felt that the NETT provides a good structural model for such training. Additionally, participants identified a need for actions directed toward the lay public to learn about research through radio, TV, and other types of media that help transform biomedical research from an uncommon and poorly understood event to a common and expected means to improve health and healthcare of our communities.

Partnership

Several participants preferred to use the idea of partnership when discussing how investigators can engage patients and advocates. They felt that the goal should be to strive for a model that emphasizes a team approach between scientists, medical professionals, patient advocates, and community members. One participant who identified as an education coordinator noted, “I loved hearing from patient advocates.” Participants emphasized that the partnership should begin early in the process of developing a clinical trial so that patients and advocates can be contributors to all aspects of design. Another participant emphasized the importance of stakeholder meetings: “[We must]… continue the joint discussion so that science doesn't forget the patient.”

Clinical Trials Regulation

The majority of conference participants had mixed views about how clinical trials are regulated. Generally all participants felt that regulations were important, but some stated that the regulations were complicated, difficult to follow, and may focus too much on protecting investigators or institutions rather than sharing information and confirming patient intent to participate. Participants recognized that there is a challenging balance to strike between regulations that best serve the needs of patients and those that protect institutions and investigators.

Conclusions

Patients are the ultimate stakeholders of NIH‐sponsored clinical trials. The NETT leadership was interested in assessing the level of interest of patient and patient advocacy groups for participation in clinical trials design for the NETT. Further, we were interested in learning more about the optimal approach to educating patients, patient advocates, and trial investigators about layperson participation in clinical trials design. We learned that there is intense interest among patients and patient advocacy groups to participate in every aspect of the clinical trials enterprise, including trials design. Although additional research is needed to identify optimal means to prepare a multistakeholder clinical trials leadership workforce, research partnerships that include diverse perspectives and expertise from patients, advocates and researchers will translate into better trials for better health. Based on lessons learned from this conference, we will refine future conference plans with the goal of increasing the pool of patient advocates with an interest in participation in clinical trial planning and design for neurological disorders. We feel that future conferences should put more emphasis on focus groups and less on didactic presentations. It is also critical that experienced patient advocate mentors are intimately involved in planning and conference presentations.