Document Type


Date of Degree

Spring 2024

Primary Advisor

Sabrina Figueiredo, PhD, MSc, BSc (PT)


Clinical Trial Design; Duchenne Muscular Dystrophy Clinical Research Partnership


To provide actionable information about the safety and efficacy of new medications, clinical trials should be designed to test outcomes that patients find meaningful. This imperative is increasingly acknowledged by the scientific community and regulatory authorities and in recent years has been enshrined in regulatory guidance documents for industry; however, clinical trials often fail to adequately address patient priorities. Evidence suggests that partnerships between researchers and patient communities may result in clinical trials that better reflect the patient experience, but there are few successful models of such partnerships. Within the clinical research landscape, the Duchenne Muscular Dystrophy (DMD) community has emerged as a positive outlier in this regard, having engaged in a research partnership over multiple years and resulting in the approval of several disease modifying therapies where previously there were none. After seeking feedback from key stakeholders from the muscular dystrophy community and incorporating it into the research design, we undertook a qualitative descriptive case study to examine the factors that have enabled the DMD community to effectively create and sustain this partnership, and to develop a model that may assist other communities in forging similar partnerships. Data was collected from interviews with 18 individual interview participants and four participants in a group interview. Participants were patients, parents, and researchers from academic and industry settings. Observations were also conducted at large meetings of the patient and research communities, which were used to contextualize and aid in the interpretation of interview data. Interview data were analyzed using initial open episodic coding followed by axial and selective coding. Five themes emerged from the qualitative analysis: specific nature of the condition; utilize legislative advocacy to influence funding and regulatory climate; create a cohesive community and leverage its resources; foster a culture of collaboration; and fight for future generations. These themes were interpreted through the lens of the Behaviour Change Wheel, the guiding framework for the project, to create an academic model of patient community readiness for research partnership. This model was further translated into a stakeholder-facing application model, the FOCUS Model, which describes the key factors identified in this study and how they interact to impact community readiness for research partnership. The FOCUS Model is presented along with a discussion guide that communities may use in a meeting or workshop setting to evaluate and improve their research partnership readiness.


©2023 by Jennifer Catena Davis. All rights reserved.

Open Access


Available for download on Saturday, March 29, 2025

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