Reshaping Clinicians' Perceptions of Disease Burden in Becker Muscular Dystrophy: The Impacts of Incorporating the Patient Perspectives in CME
Carole Drexel1, Eve Wilson1, Aravindhan Veerapandiyan2, Luca Bello3, Katie Kowalski4
1PlatformQ LLC, 2Arkansas Childrens Hospital/UAMS, 3University of Padova, Department of Neuroscience NPSRR, 4National Organization for Rare Disorders
Objective:
To study the impacts of continuing medical education (CME) that incorporates patient perspectives to address misperceptions regarding the burden of Becker muscular dystrophy (BMD).
Background:
Clinicians (HCPs) often perceive BMD as milder compared to Duchenne muscular dystrophy (DMD). However, emerging therapeutics and an improved understanding of BMD’s variable disease trajectory have emphasized the need to recognize its true burden. We hypothesized that incorporating patient perspectives into CME activities presents an opportunity to address misperceptions.
Design/Methods:
A 60-min panel discussion was launched online in August 2023 (available for one year). Portions of a recorded interview with a person with BMD provided insights into quality of life and experiences with HCPs. Short video segments of the program were sent on social media to NPI-verified neurologists to extend reach. Impacts were assessed through pre- and post-tests, polling, and self-report of intended practice changes. Chi-square tests compared responses (P<0.05; pre/post).
Results:
A total of 4,532 HCPs (neurologists 83%) participated. About half reported seeing 1 patient with BMD per week. At baseline, HCPs demonstrated low awareness of contraction-induced injury mechanisms (29%) and emerging agents for BMD (23%). Knowledge increased post-activity but remained average (41% and 61% correct, respectively; P<0.01). Prior to education, 76% described BMD as “much” or “somewhat milder” than DMD. Post-test responses showed gains in identifying the common cause of death (+20%; P<0.01) and grading disease severity (+21%; P<0.01). Half of HCPs reported an intention to change their practice, most commonly by adjusting treatment/management approaches (35%) and referral patterns (26%), possibly reflecting increased awareness of disease burden.
Conclusions:
Integrating patient perspectives into education improved HCPs’ understanding of BMD pathophysiology and emerging therapies. The findings also suggest it helped reshape perceptions of disease burden. Overall, the data highlight the value of patient-centered CME in preparing HCPs to deliver more patient-focused care for individuals with BMD.
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