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PP05-43: Understanding and Addressing the Holistic Rare Disease Patient Experience Through Community Advisor Program

Poster Presenter

      Jeffrey Smith

      • Senior Director, Head, Patient Advocacy
      • Wave Life Sciences
        United States


The purpose of this report is to share an effective framework for engaging patients and families, gaining a deep understanding of their needs and perspectives, and integrating learnings into a biopharmaceutical company.


Participants were identified in partnership with a patient advocacy organization. Pre-meeting interviews were conducted to obtain background information, set expectations and begin the trust-building process. Pre- and post-meeting surveys were conducted, and a follow-up engagement plan was prepared.


Three Community Advisor Meetings were coordinated with a total of 30 parents of children living with Duchenne muscular dystrophy. Participants came from diverse backgrounds and experiences and were representative of the broader Duchenne community. Thirteen US states were represented, and children of the parents were aged 4 to 22 years old and had been diagnosed at ages from pregnancy to 10 years old. 75% of participants stated this was their first advisor meeting. The agenda of each meeting focused on understanding the lived experience of Duchenne, discussing the challenges and impactful moments in the Duchenne journey, and identifying potential programs and solutions that can help Duchenne families The program also included an opportunity to educate families on the limitations of drug development and increase trust and understanding. Discussions during the meetings were often emotional, as participants coped with feelings and perspectives they had seldom shared. Insights were gained related to the importance of community and an effective support system, the impact of inconsistencies in Duchenne care, the increased challenges and disparities faced by underserved communities, and the importance of learning to live fully and support resiliency by pursuing experiences that were enjoyable to families. The meetings themselves were viewed by participants as a unique opportunity to make an impact on others by sharing their voice with a biopharmaceutical company, as well as an important opportunity to meet other families living with similar challenges. Based on a post-meeting survey, 90% of respondents rated the overall experience as very good or excellent and 95% stated that they would participate in future meetings.


Understanding the holistic experience of individuals and families living with a rare disease is vital to biopharmaceuticals seeking to address their needs. We created a unique forum that enabled us to establish trust and connect more deeply with participants. In this environment, we were able to gain deeper insights and help participants understand and accept the limitations industry has with certain engagements. This enabled participants to have their voices and experiences contribute in ways that had a clear impact on other families and on our own programs. To maintain the trust developed, it is critical to identify avenues to share learnings back with each individual participant and co-create initiatives that build on their contributions.