Abstract
Purpose
Clinical trials in Duchenne muscular dystrophy (DMD) are increasing due to technical
advances in genetics, muscle biology and muscle imaging, and translational science.
Yet the ability to achieve and measure progress in clinical trials in DMD is severely
constrained by recruitment difficulties and low levels of patient and family participation.
Clinical trials that do not have full inclusion of patients may affect how well new
therapies perform in clinical practice.
Methods
This study qualitatively investigated family-centered and clinician-based knowledge,
attitudes, and perceptions of engagement in clinical research in DMD. Thirteen focus-group
sessions (8 parent based and 5 clinician based) were held at 5 demographically and
geographically diverse sites (Houston, Texas; Minneapolis, Minnesota; Pittsburgh,
Pennsylvania; Sacramento, California; and Washington, DC). Thematic analysis was used
for identifying patterns of meaning across the dataset.
Findings
Totals of 28 parents and 33 clinicians participated in innovative and thoughtful discussions
regarding clinical research in DMD and approaches to eliciting family engagement.
Five overarching themes emerged from our qualitative data. The theme of Information
discussed the lack of accessible and coherent information, as well as the overabundance
of fragmented information. The theme of Conversation demonstrated the importance of
having open and in-depth dialogue about research with families in eliciting trust
and obligation toward the research process. The Barriers and Incentives themes presented
the parents’ and clinicians’ views of the life-altering sacrifices that families make
to participate in research and ways to reduce these burdens. Under the Solutions theme,
parents and clinicians also suggested innovative ways to incentivize families and
clinics and thoughtful solutions to increase family engagement in research.
Implications
Effective recruitment for clinical studies in rare diseases requires a truly committed
and engaged study team, as well as the necessary resources to overcome the multitude
of barriers that families face. A clearly delineated recruitment plan, developed together
with families, should be the standard operating procedure during clinical trial development.
Protocols utilizing direct family-centered strategies for providing information and
for recruiting research participants in studies in rare diseases are essential.
Key words
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Article info
Publication history
Published online: April 29, 2016
Accepted:
April 4,
2016
Identification
Copyright
© 2016 Elsevier Inc. All rights reserved.
