Enhancing Diversity and Inclusion in Clinical Trials.

Women and people from most racial and ethnic groups in the United States have historically been under-represented in clinical trials of investigational medical products. Inadequate representation of these groups may lead to an incomplete understanding of the safety and efficacy of new drugs, devices, biologics, and vaccines, and limit the generalizability of trial findings. As a result, new medical products may not be beneficial to all people who need them, and existing inequities in outcomes among various population groups may remain unchanged or worsen, or new disparities may arise. Although much work has focused on study-level strategies, research organizations must make systemic changes to how clinical trials are envisioned and implemented to achieve sustainable support for diversity and inclusion in clinical trials. The Clinical Trials Transformation Initiative (CTTI) conducted interviews with leaders at institutions that conduct clinical trials to explore perspectives on organizational-level practices that promote diversity and inclusion in clinical trials. Leaders described motivations, such as an ethical and moral imperative; organizational practices, such as staff investment and resource allocation; perceived return on investments, such as better science; and deterrents, such as cost and time. The CTTI also convened an expert meeting to discuss the interview findings and provide guidance. We present the interview findings and expert guidance in a framework that describes four key areas-commitment, partnerships, accountability, and resources-on sustaining organizational-level approaches for improving diversity and inclusion in clinical trials, with the ultimate goal of advancing health equity. Institutions who conduct and support clinical trials should implement organizational-level approaches to improve equitable access and diverse patient participation in clinical trials.

Putting participants and study partners FIRST when clinical trials end early.

Between 2018 and 2019, multiple clinical trials ended earlier than planned, resulting in calls to improve communication with and support for participants and their study partners ("dyads"). The multidisciplinary Participant Follow-Up Improvement in Research Studies and Trials (Participant FIRST) Work Group met throughout 2021. Its goals were to identify best practices for communicating with and supporting dyads affected by early trial stoppage. The Participant FIRST Work Group identified 17 key recommendations spanning the pre-trial, mid-trial, and post-trial periods. These focus on prospectively allocating sufficient resources for orderly closeout; developing dyad-centered communication plans; helping dyads build and maintain support networks; and, if a trial stops, informing dyads rapidly. Participants and study partners invest time, effort, and hope in their research participation. The research community should take intentional steps toward better communicating with and supporting participants when clinical trials end early. The Participant FIRST recommendations are a practical guide for embarking on that journey.

Designing the Fostering Inclusivity in Research Engagement for Underrepresented Populations in Parkinson's Disease study.

BACKGROUND: Population reflective research enrollment improves study generalizability and disease knowledge. Nevertheless, the proportion of underrepresented groups (URGs) in Parkinson's disease (PD) research remains low. Hence, the current manuscript describes the process of designing a study to analyze the effectiveness of strategies to overcome barriers to URG recruitment in PD research. METHODS: The Fostering Inclusivity in Research Engagement for Underrepresented Populations in Parkinson's Disease (FIRE-UP PD) study asked participating sites to identify a URG or geographical region to target to assess knowledge and attitudes toward PD research as well as increase Fox Insight (an online study with The Michael J. Fox Foundation) participation across eight months. URGs were defined as racial and ethnic minorities, women, rural populations, and low socioeconomic status groups. Participating sites were paired based on their proposed interventions and were randomly assigned to either the intervention or control condition. RESULTS: The FIRE-UP PD study was divided into pre-intervention, intervention, and post-intervention periods to measure changes in awareness and trust in PD research along with engagement and interest in PD protocols through the use of several surveys. Interventions included developing educational tools to engage local communities, building partnerships within local PD communities, and recruiting stakeholders to reimagine medical and research information for the community. CONCLUSION: Improving representation in research is a crucial step toward improving access to PD diagnoses and treatments. This is one of the first multi-site PD research studies to include community engagement to address barriers to research participation and improve research recruitment of URGs.

Use of Figurative Language by People With Parkinson Disease to Describe "Off" Periods: Clear as Mud.

OBJECTIVE: "Off" periods are characterized by the reemergence of motor and nonmotor symptoms in individuals with Parkinson disease (PD) and often negatively affect daily functioning. Individuals' experiences are diverse and may be difficult to articulate; figurative language is often used by patients to describe such experiences. Our objective was to understand how individuals with PD use figurative language to explain off periods and how experts interpret such expressions. METHODS: Individuals with self-reported PD participating in the online Fox Insight study were invited to participate in a survey about off periods. Those endorsing off periods were asked to describe their experiences with open-ended free-text responses. Instances where any type of figurative language was used were identified and classified into themes. Three movement disorder neurologists reviewed each phrase and specified what symptoms they felt were likely represented. RESULTS: A total of 109 instances of figurative language phrases were identified across descriptions from 86 patients. Allusions to viscous materials (e.g., mud and cement) and effects of chemicals (e.g., drunkenness) were common (18.35% and 17.43% of phrases, respectively). Most phrases were interpreted by the neurologists as representing motor symptoms, but neurologists agreed on what specific symptom was being referred to for only 42 (38.5%) phrases. CONCLUSIONS: To describe off periods, individuals with PD use various forms of figurative language, but this language is not uniformly interpreted and understood by specialists. Given the subjective interpretation of figurative language, exploring what patients are trying to convey when they use such language is important and could improve patient-physician communication.

Hispanic Perspectives on Parkinson's Disease Care and Research Participation.

BACKGROUND: Hispanics are under-represented in Parkinson's disease (PD) research despite the importance of diversity for results to apply to a wide range of patients. OBJECTIVE: To investigate the perspective of Hispanic persons with Parkinson disease (PWP) regarding awareness, interest, and barriers to participation in research. METHODS: We developed and administered a survey and qualitative interview in English and Spanish. For the survey, 62 Hispanic and 38 non-Hispanic PWP linked to a tertiary center were recruited in Arizona. For interviews, 20 Hispanic PWP, 20 caregivers, and six physicians providing service to Hispanic PWP in the community were recruited in California. Survey responses of Hispanic and non-Hispanic PWP were compared. Major survey themes were identified by applying grounded theory and open coding. RESULTS: The survey found roughly half (Q1 54%, Q2 55%) of Hispanic PWP linked to a tertiary center knew about research; there was unawareness among community Hispanic PWP. Most preferred having physician recommendations for research participation and were willing to participate. Hispanics preferred teams who speak their native language and include family. Research engagement, PD knowledge, role of family, living with PD, PD care, pre-diagnosis/diagnosis emerged as themes from the interview. CONCLUSION: Barriers exist for participation of Hispanic PWP in research, primarily lack of awareness of PD research opportunities. Educating physicians of the need to encourage research participation of Hispanic PWP can address this. Physicians need to be aware of ongoing research and should not assume PWP disinterest. Including family members and providing research opportunities in their native language can increase research recruitment.

A Call to Action: Promoting Diversity, Equity, and Inclusion in Parkinson's Research and Care.

The current base of knowledge around Parkinson's disease has been assembled in partnership with a cohort of participants that does not resemble the diversity of people with the disease. This poor representation in research results in an incomplete picture of the disease and disparities in care. The Michael J. Fox Foundation has defined four major areas of action: 1) identifying barriers and solutions to research participation; 2) funding inclusive research with greater participant diversity; 3) building a clinician/researcher workforce committed to health equity; and 4) supporting a more holistic understanding of PD. While factors driving disparities, including broader societal challenges, are complex, it is imperative that the PD research, care, and patient communities move in a decisive and coordinated fashion to identify and implement strategies that advance treatments for everyone with PD and eliminate care inequities.

Video research visits for atypical parkinsonian syndromes among Fox Trial Finder participants.

BACKGROUND: Use of video research visits in neurologic conditions is rising, but their utility has not been assessed in atypical parkinsonian syndromes. We sought to evaluate the diagnostic concordance between video-based vs self-reported diagnoses of multiple system atrophy, progressive supranuclear palsy, dementia with Lewy bodies, and corticobasal syndrome. We also assessed patient satisfaction with video-based visits. METHODS: We conducted a study of video-based research visits in individuals with an atypical parkinsonian syndrome enrolled in The Michael J. Fox Foundation's Fox Trial Finder. Participants completed a recorded real-time video visit with a remote evaluator who was blinded to the participant's self-reported diagnosis. The investigator conducted a structured interview and performed standard assessments of motor function. Following the visit, the investigator selected the most likely diagnosis. The recorded visit was reviewed by a second blinded investigator who also selected the most likely diagnosis. We evaluated diagnostic concordance between the 2 independent investigators and assessed concordance between investigator consensus diagnosis and self-reported diagnosis using Cohen's kappa. We assessed participant satisfaction with a survey. RESULTS: We enrolled 45 individuals with atypical parkinsonian syndromes, and 44 completed the investigator-performed video assessment. We demonstrated excellent concordance in diagnosis between the investigators (κ = 0.83) and good reliability of self-reported diagnosis (κ = 0.73). More than 90% of participants were satisfied or very satisfied with the convenience, comfort, and overall visit. CONCLUSIONS: Video research visits are feasible and reliable in those with an atypical parkinsonian syndrome. These visits represent a promising option for reducing burden and extending the reach of clinical research to individuals with these rare and disabling conditions.

Transportation innovation to aid Parkinson disease trial recruitment.

Among the barriers to participation in clinical trials, transportation to and from study sites may be a prominent issue. Patients with Parkinson's disease have unique circumstances that add to the barriers including dementia, loss of driving ability, timing of medications, impact of reduced mobility, and bowel and bladder concerns. We sought to alleviate some of the burden of transportation by setting up pre-arranged rides through a third-party ride sharing service. This pilot project was established to assess feasibility and to explore the possibility that reducing the transportation burden may enhance participation in studies. One out of three academic sites was successful in setting up this service, and surveyed participants on the impact of this service. In general, study participants who opted into the ride-sharing service felt it made the process easier and less stressful. Most participants agreed that they are more likely to participate in another study if transportation was provided. This short-term pilot intervention suggests that participants were satisfied with a ride sharing service to help with their medical transportation needs, but larger studies that include data collection about retention are needed.