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. Clin Trans Sci 2015; Volume 8: 776-778

Optimizing Enrollment of Patients into Nephrology Research Studies

“…Partnership with a geographic or patient community can be used to share the rationale and methods of biomedical research, develop research agendas, and design feasible trials (3). Patient advocacy groups, such as the American Kidney Fund, National Kidney Foundation, NephCure Kidney International, and Polycystic Kidney Disease Foundation, may serve as powerful partners in convening or participating in community engagement activities.…”

“…Patient advocacy groups, such as the American Kidney Fund, National Kidney Foundation, NephCure Kidney International, and Polycystic Kidney Disease Foundation, may serve as powerful partners in convening or participating in community engagement activities. Research sponsors, networks, and health systems conduct forums for open dialogue, health fairs, and other educational events, where community and research team members have the opportunity to collaborate to improve community awareness and participation in biomedical research (3,4). The Rare Disease Clinical Research Network website provides opportunities for patients to enroll in disease-specific patient contact registries.…”

“…Patients and their caregivers are often willing to describe symptoms, signs, and adverse reactions to therapies and rank health challenges to inform research priorities (3,35,36). Tremendous examples exist of patients as members of study development and design teams, study steering committees, and oversight committees, such as data safety and monitoring boards and IRBs (37)(38)(39)(40)(41).…”

The ACTTION Guide to Clinical Trials of Pain Treatments, part II: mitigating bias, maximizing value

“…Patient engagement in clinical trials includes participation in identifying unmet research needs; selecting and prioritizing outcome measures; collaborating on study design, execution, analysis, and interpretation; and translating, disseminating, and implementing clinical trial results in the community. 43 , 53 Such patient engagement has been recommended and implemented across multiple therapeutic areas, including cardiology, 13 infectious disease, 6 neurology, 4 and oncology. 2 , 7 Studies of the impact of patient engagement in clinical trials have reported that the participation of patients can provide important contributions to study feasibility, rigor, and relevance 19 and can also have financial value by contributing to the avoidance of protocol amendments and the improvement of enrollment, adherence, and retention.…”

Behavioral and Social Sciences at the National Institutes of Health: adoption of research findings in health research and practice as a scientific priority

“…Research on medication adherence can be applied more systematically to ensure adequate medication adherence in these trials [33]. Another challenge of health research is the engagement of participants in longitudinal trials and observational cohort studies [34]. Principles of behavioral and social sciences such as altruism, persuasion, and motivation can be applied to improve the sustained engagement of participants in research.…”