Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished clinical outcomes. However, there is a growing sense of hope: by working together across various sectors, we can make meaningful strides in improving healthcare & clinical trial access and support for families, no matter their background.

Unified Collaboration for Change

Recently, PPMD hosted a pivotal discussion at its Duchenne Drug Development Roundtable (DDDR), bringing together industry partners, clinicians, ethicists, and other experts in a pre-competitive space. The primary goal of the roundtable was to discuss how to make clinical trials more accessible to all individuals living with Duchenne and Becker, and to identify the unique challenges faced by underserved communities and to better understand the experiences of industry partners and healthcare providers working with diverse geographic, socioeconomic, racial and ethnic populations with Duchenne and Becker.

Engaging Communities and Overcoming Barriers

A central theme of the discussion was the need for health initiatives to go beyond just clinical trials and therapies. A multi-system approach is needed to actively engage with communities to improve health outcomes, which means understanding the cultural distinctions in how health challenges are perceived and acknowledged within different communities. At times, this includes engaging with trusted community and family leaders, such as clergy members or grandparents, who can help support families through their decision-making process. The group discussed how clinicians and industry partners must make room for and respect these perspectives in order to foster trust and collaboration.

Language barriers were another significant topic of conversation. It was noted that language challenges are not solely about translation, but also tied to culture, tradition, and health literacy. Families may fear complex medical terminology or feel overwhelmed by unfamiliar concepts. Ensuring that health information, including industry and institution-created materials, is accessible, understandable, available in a patient’s native language, and using clear and simple language, is essential to overcoming these barriers. As clinical trials are developed, these considerations must be kept front and center to ensure that families fully understand their participation options and can make informed decisions. With numerous barriers to trial participation, if language barriers can be minimized, it allows greater time to focus on the other, more difficult burdens faced by families and mitigate unnecessary delays throughout the trial process.

Making Clinical Trials More Inclusive

Diversity in clinical trials is critical to ensuring that all members of the community have an opportunity to participate in research if they so choose and that therapies are safe and effective for everyone. A major step toward inclusion involves creating Diversity Action Plans (DAPs) to deliberately include more diverse populations in clinical research. The FDA requires industry sponsors of Phase 3 or pivotal clinical studies to submit a DAP to provide details about how they will increase enrollment of underrepresented populations in their trial. Additionally, DAPs will increase the accessibility of clinical trials to a broader portion of the Duchenne community, accommodating specific community needs, regulatory guidance, cultural competence, and health literacy best practices. This includes offering personal assistance services, compensating families for travel, assisting with childcare, providing educational materials for all educational levels, ensuring materials are accessible in a family’s native language, and fostering a supportive environment for patients and their families early on. 

The Role of Industry and Healthcare Provider Collaboration

Industry partners and healthcare providers are in a unique position to help minimize barriers for families with Duchenne and Becker to make access to clinical trials more inclusive. Attendees addressed strategies to help minimize barriers, including transportation difficulties, taking time away from work, or language. Simple yet impactful actions—like simplifying educational materials, screening patients for social determinants of health, or employing “teach-back” methods where patients repeat information to confirm understanding—can help ensure that families fully comprehend their diagnosis and therapy options. 

Furthermore, the healthcare community cannot solve these challenges alone. Pharmaceutical companies, healthcare providers, advocacy groups, and community leaders must work together to recognize and support families to overcome these barriers. As one attendee aptly stated, “We won’t solve all the problems, but if we each take something from today and do our part, we can move in the right direction.”

A Path to Equity in Duchenne and Becker Clinical Trials

By ensuring that every family has access to resources, care, and support, we can create a more equitable healthcare and clinical trial landscape. The collaboration of all stakeholders is essential to improving the lives of individuals and families affected by Duchenne and Becker.

As part of PPMD’s Drug Development Roundtable, industry sponsors working to develop therapies for Duchenne will continue to work throughout the coming year to evaluate issues and put forward solutions to make clinical trial participation accessible to a broader portion of the Duchenne and Becker community. Furthermore, a group of ten companies has committed to working together to address specific issues related to diversity, equity, and inclusion in clinical trials.

PPMD is fighting for EVERY future and will not stop until all families have access to the therapies, care, and research opportunities they deserve.