Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease


Meaningful engagement between biopharmaceutical companies and patient communities has increasingly become an important part of the therapeutic-development process, as such engagement improves the understanding of the multifaceted challenges and unmet needs that communities experience and provides an opportunity to inform the approach to the development of new therapies and services. Presented here are learnings from a community-advisor program designed to engage families of patients with Duchenne muscular dystrophy (DMD) in a manner that enabled caregivers to make valuable contributions to the therapeutic-development process and to the DMD community. Parents of children with DMD, representing the broader DMD community, were identified in partnership with patient-advocacy organizations and invited to participate in a community-advisor meeting with members of Wave Life Sciences. The community-advisor meeting was designed to provide participants with an opportunity to share their personal experiences with DMD, to help to inform the therapeutic-development process, and to identify potential solutions for addressing unmet needs. Three community-advisor meetings were held with a total of 30 parents, representing 36 children with DMD. Key themes that emerged from the advisors’ discussion included the importance of the community’s emotional and mental support, the inconsistencies in DMD care, the increased challenges and disparities faced by underserved communities, and the need for more comprehensive, holistic approaches to the treatment and management of DMD. The advisors viewed the meetings as an opportunity to share their voices with a biopharmaceutical company, coupled with the advantage of meeting other families living with similar challenges. Most of the advisors stated that this was their first advisor meeting. This community-focused approach empowered participants to voice their needs and perspectives, to brainstorm potential solutions for addressing those needs, and to initiate and foster connections in ways that had a considerable impact on one another and on therapeutic-development programs at Wave Life Sciences.

Key words


Patients and their families living with rare diseases, such as Duchenne muscular dystrophy (DMD), have increasingly become important, influential voices for informing the development of therapeutics. An understanding of the holistic experience of these patients and their families is vital to the development of new therapies, programs, and services that meet their needs. Biopharmaceutical companies, through patient and community engagement, have the obligation to gain a deeper understanding of how diseases impact the overall quality of life of patients and their families. Furthermore, the integration of such engagement as a part of a patient-centric development process has the potential to facilitate the delivery of optimal care and treatment while meeting patients’ and families’ broader needs.

  • Khodyakov D.
  • Kinnett K.
  • Grant S.
  • et al.
Engaging patients and caregivers managing rare diseases to improve the methods of clinical guideline development: a research protocol.