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Research Platform
Issue Date: August 2016
Published Online: August 01, 2016
Updated: January 01, 2021
Strategies for Engaging the Duchenne Muscular Dystrophy Community in Research
Author Affiliations
  • University of Pittsburgh
  • Children's National Health System
Article Information
Evidence-Based Practice / Musculoskeletal Impairments / Assessment/Measurement
Research Platform   |   August 01, 2016
Strategies for Engaging the Duchenne Muscular Dystrophy Community in Research
American Journal of Occupational Therapy, August 2016, Vol. 70, 7011500004. https://doi.org/10.5014/ajot.2016.70S1-RP203B
American Journal of Occupational Therapy, August 2016, Vol. 70, 7011500004. https://doi.org/10.5014/ajot.2016.70S1-RP203B
Abstract

Date Presented 4/8/2016

Through parent-based and clinician-based focus groups, we qualitatively explored barriers to engaging families of children with rare diseases in clinical trials. The insight gained may improve the future design of clinical research protocols and improve participation in future clinical trials.

Primary Author and Speaker: Roxanna Bendixen

Contributing Author: Lauren Hache Morgenroth

PURPOSE: Duchenne muscular dystrophy (DMD) is an X-linked rare degenerative neuromuscular disorder that causes severe progressive muscle loss and premature death. Research in DMD is critical to provide advancements in treatment and care to improve the quality of life and ultimately find a cure for individuals with DMD. Yet our ability to achieve and measure progress in clinical research in DMD is constrained by recruitment challenges and low levels of participation. Successful completion of clinical trials fully depends on successful engagement with the intended beneficiaries. This gap, resulting from clinical trials that do not have full inclusion of patients, affects how well new therapies perform in the real world.
We seek to understand the barriers to engaging the DMD community in clinical trials, as well as strategies that build healthy partnerships and inform and assist with our recruitment efforts. Increasing enrollment and participation in clinical trials in DMD is our primary goal.
DESIGN: In collaboration with the Cooperative International Neuromuscular Research Groups (CINRG) and associated Muscular Dystrophy Association (MDA) clinics, our qualitative study included parent-centered and researcher–clinician-centered focus groups that were conducted at five sites: Pittsburgh, PA; Washington, DC; Minneapolis, MN; Houston, TX; and Sacramento, CA. Parents of boys with DMD and researchers–clinicians involved in DMD research were recruited through CINRG and MDA sites.
METHOD: Guided focus group sessions were held in private locations that were convenient for parents and researcher–clinicians. Parent participants received a $25.00 gift card. Meals were provided for parent and researcher–clinician participants. All sessions were audio recorded and transcribed nonstrict verbatim (methods of recruitment).
ANALYSIS: Qualitative thematic analysis of focus group transcripts was conducted to identify themes from both parent and researcher–clinician focus groups. A deductive thematic analysis approach was used that involved a six-phase process: (1) familiarization with the data; (2) coding; (3) searching for themes; (4) reviewing themes; (5) defining and naming themes; and (6) writing up.
RESULTS: A total of 13 focus-group sessions were conducted between the five sites with 33 researcher–clinician participants and 28 parent participants of boys with DMD. Major themes were identified, including (1) incentives and barriers to research participation, (2) the importance of open and honest communication and information to help families make informed decisions, (3) elements to developing an ideal recruitment plan, and (4) novel solutions and strategies for reaching out and engaging families in research. Researcher–clinicians and parents shared similar ideas on research engagement; disconnect between the two groups existed regarding perceived barriers and incentives to research participation.
DISCUSSION: Identifying barriers to research participation and recruitment provided insight into development of strategies to improve recruitment efforts that partner with and support both clinicians involved in recruitment and families participating in research in DMD.
IMPACT: The insight gained through qualitatively investigating family-centered and clinician-based attitudes and perceptions may improve the future design of clinical research protocols and improve participation in future clinical trials