Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease
Abstract
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
Introduction
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Stakeholders (eg, health care providers, biopharmaceutical companies, payers, regulatory bodies) have recognized the value of the perspective of patients and caregivers and of the patient-centric process.
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Particularly in rare diseases, characterized by a low prevalence and a lack of widespread physician expertise, patients and caregivers are often forced to become experts on their own conditions, and are thus able to provide a nuanced, deep understanding that other stakeholders cannot.
The shift to a more patient-centered paradigm is supported by the establishment of the Patient-Focused Drug Development initiative by the US Food and Drug Administration (FDA), which aims to integrate the patient’s perspective into the early stages of the therapeutic-development process and to inform important decisions related to clinical-trial design and regulatory benefit–risk assessments.
The value of patients’ and caregivers’ perspective has been further demonstrated by the FDA’s invitation to Parent Project Muscular Dystrophy, an advocacy organization focused on DMD, to write the first patient advocacy–initiated draft guidance on the development and review of DMD therapies.
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Patients’ engagement in rare diseases may be hindered by a lack of participant availability, high cost, scheduling challenges, geographic dispersion, and a perceived risk of engagement.
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While community engagement has been focused on assessing patients’ and/or caregivers’ understanding and perceptions of clinical research and on developing patient-reported outcomes measures, the potential exists for more meaningful integration of patients’ insights into the therapeutic-development process at its earliest stages.
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Here we describe a community-advisor program designed to facilitate collaborative engagement with the families of patients with DMD in a manner that enables meaningful contributions that can be integrated into the therapeutic-development process.
Community-advisor Meeting Design
A series of 3 community-advisor meetings between the families of patients with DMD and members of various functions of Wave Life Sciences, a biopharmaceutical company located in Cambridge, Massachusetts, was planned. Parents or caregivers, representing the diverse experiences of families affected by DMD in the United States, were identified in partnership with members of the DMD community and patient-advocacy organizations. Participation in the community-advisor meetings was limited to individuals living in the United States who were self-identified parents or other caregivers of a person with DMD, who were willing to share their experiences of and insights on living with DMD, and who had the ability to participate in a full-day meeting (including travel if necessary). A representative of either Wave Life Sciences or a DMD patient-advocacy organization contacted potential participants to gauge their interest in serving as an advisor at one of the community-advisor meetings. All potential participants who had expressed interest were contacted by Wave Life Sciences via e-mail. In advance of the meeting, a Wave Life Sciences representative (J.S.) conducted introductory phone calls or premeeting interviews to confirm eligibility, to gather background information (eg, relationship, age of the person with DMD), to establish expectations about participating in the meeting, and to initiate the trust-building process.
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Advisors were grouped into teams and tasked with co-creating “Living with Duchenne” storyboards, in which imagery, phrases, and symbols were chosen to capture their experiences, feelings, and challenges. Then, as a large group, the advisors and members of Wave Life Sciences worked together to co-create potential opportunities for addressing prioritized Moments that Matter. Through storyboards, advisors were able to creatively share and rephrase poignant moments in a safe, comforting space, to find similarities to and common threads with their fellow advisors while retaining their uniqueness, and to identify unmet needs. Advisors were also asked to share their perspectives on the current standard of care in DMD, their expectations for the future, as well as their preferred sources of information and/or channels for sharing information with the community.
Following the community-advisor meeting, advisors were asked to complete postmeeting surveys for providing feedback on the meeting’s design and outcomes. A cross-functional team at Wave Life Sciences separately convened to prioritize community insights and to ideate ways to organically integrate learnings from the community-advisor meetings into the company’s work while addressing the needs of the DMD community. Insights and key learnings from the meetings were subsequently shared with the advisors via phone calls and/or e-mail.
Results
Figure 1Advisor characteristics. DMD = Duchenne muscular dystrophy.
Key Insights
Figure 2Key themes that resonated across the 3 community-advisor meetings. DMD = Duchenne muscular dystrophy; FDA = US Food and Drug Administration; HCP = health care provider; OT/PT = occupational therapy/physical therapy; PAO = patient-advocacy organization.
Advisors’ Feedback on the Community-advisor Meetings
Advisors viewed the meetings as a unique opportunity to share their voices with a biopharmaceutical company and to have a meaningful impact on the DMD community. The meetings were also considered to be a valuable occasion to meet other families living with similar challenges.
Figure 3Advisor feedback on meeting design and outcome.
Postmeeting Ideas and Implementation
Participants from Wave Life Sciences reported that the engagement with the advisors was emotionally impactful and provided an opportunity to understand the experience of living with DMD and to integrate these insights into the work of Wave Life Sciences. Following the community-advisor meetings, a cross-functional team at Wave Life Sciences developed an initial framework for addressing areas of unmet need emphasized by advisors that could be incorporated into the company’s activities. Key recommendations included community education on the therapeutic-development and clinical-trial processes; increased data sharing with the community; use of multilingual communication and virtual technology for improving outreach; and the formation of a coalition for facilitating research collaboration and exploration, as well as community education and engagement.
Discussion
Understanding and applying the perspectives of patients and caregivers can inform a biopharmaceutical company’s development of holistic solutions that address unmet needs beyond clinical symptoms. In this vein, the Wave Life Sciences community-advisor meetings described here were purposefully structured to foster an open, caring, and empowering environment so that advisors could share their experiences and perspectives candidly. The key insights and unmet needs identified through this collaborative approach are intended to inform the development of comprehensive, meaningful therapies and programs for patients with DMD and their caregivers.
Unlike conventional approaches focused on medical education, this format was designed to create a highly interactive and comfortable environment for advisors to communicate on themes, messages, and experiences they considered to be most important. A Wave facilitator led an interactive exercise that allowed advisors to channel memories of experiences, emotional responses, and the resulting impact into a more positive outcome: a visual storyboard of shared reality. Advisors noted feeling heard and understood as a result of the exercise. This patient-centric approach provided advisors the opportunity to educate and communicate directly with members of Wave Life Sciences. Furthermore, the supportive environment established in this type of setting may extend beyond the meeting sessions, providing renewed hope and enabling caregivers to broaden and deepen their connections within the community.
According to the advisors, conventional engagement between biopharmaceutical companies and patients/caregivers can often be limited or one-sided. Many advisors, in fact, explained this was their first interaction of this kind with a biopharmaceutical company. A crucial discussion point remains: How can biopharmaceutical companies, particularly in rare disease, apply this methodology to partner with patients and families to obtain a deeper understanding, identify insights, prioritize needs, and co-create programs to address them?
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Engaging patients and caregivers early in the therapeutic-development process can also help to identify crucial unmet needs, which can aid in the development of a patient-centric target–therapeutic profile or support strategic decision making.
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Furthermore, community engagement during clinical development can enable a patient-centric study design (eg, end points of relevance to patients) as well as improve enrollment, retention, treatment adherence, and the overall patient experience.
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Patients and caregivers can also help to effectively disseminate key data or information in a manner that is relevant and easy for the broader community to understand.
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Many of these benefits were also observed during the community-advisor meetings on DMD. Emotional storyboarding and the Moments that Matter exercises provided advisors with an opportunity not only to reflect on their journey but also to identify and characterize their evolving unmet needs. Advisors also exchanged tangible strategies for improving their daily lives and for navigating obstacles (eg, insurance coverage and reimbursements) that cannot be readily addressed by health care providers. Although clinical-trial design was not the primary focus of these meetings, the advisors’ experiences with clinical trials helped members of Wave Life Sciences to better understand potential challenges or barriers to consider and/or address when designing new clinical trials.
thereby potentially skewing engagement toward high-income families or those with access to greater resources. Patients and caregivers may be less inclined to participate due to poor impressions from past engagements and/or of biopharmaceutical companies, including a false appearance of inclusiveness.
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The heterogeneity of patients’ experiences with rare disease and the inherent bias that patients and caregivers may have toward advocating for their own needs may lead to findings that are not necessarily reflective of the broader community.
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For the community-advisor meetings, members of Wave Life Sciences established a relationship with the advisors through premeeting discussions, which helped to set clear expectations about the engagement. Three separate community-advisor meetings were held across the United States to facilitate participation and to enhance geographic and demographic diversity. The meetings were also carefully designed to ensure that the advisors had an opportunity to share their experiences and insights through small group activities, such as the emotional storyboarding, and to collectively reflect on these shared experiences to prioritize the key unmet needs that emerged. An effort was also made to continue engagement and relationship building through postmeeting updates.
Conclusions
The needs of families with DMD or other rare diseases are often complex and underserved; biopharmaceutical companies must consider care that extends beyond treating the clinical symptoms of the disease. Insights gained through community engagement may help biopharmaceutical companies working in DMD to better understand the patient journey and the unmet needs of patients’ families, and to facilitate the development of more impactful treatments, programs, and services. Lessons from this unique approach for successful and meaningful community engagement may be applied to similar engagement initiatives in DMD or other rare diseases.
Author Contributions
All of the authors were involved in meeting design, participated in the community-advisor meetings and postmeeting ideation, and contributed equally to the analysis and interpretation of the data and in the preparation of the manuscript.
Declaration of Competing Interest
This study was funded by Wave Life Sciences Ltd . J. Smith and K. Damm are employees of Wave Life Sciences. G. Hover and J. Chien are employees of Imbue Partners LLC, which was contracted by Wave Life Sciences to support the research described in this article. The authors have indicated that they have no other conflicts of interest with regard to the content of this article.
Acknowledgments
We thank all participants and their families who attended the advisory meetings and shared their valuable insights for supporting this work. We thank the DMD patient-advocacy community for support in identifying participants. In particular, we thank Parent Project Muscular Dystrophy (PPMD) for conducting outreach to potential participants through its outreach network, PPMD Connect. We thank Disha Patel, PhD (Chameleon Communications International, with funding from Wave Life Sciences), for editorial assistance in the preparation of this report.
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Article Info
Publication History
Published online: December 26, 2020
Accepted:
December 6,
2020
Publication stage
In Press Corrected Proof
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