Becker muscular dystrophy (Becker) is a serious, rare, neuromuscular disorder with no approved treatments. There is a need to understand the lived experience of Becker individuals to ensure that treatments in development address disease aspects that are relevant and meaningful. As regulators, payers, and other key stakeholders place growing emphasis on integrating the patient voice into drug development, qualitative interviews serve as an invaluable data source that provides deep insights into the patient experience across every phase of the drug development lifecycle.
In-trial interviews were conducted with 33 participants in 2 trials (open label [ARCH] and randomized, placebo controlled [CANYON]) who consented to participate; for the placebo-controlled study, both participants and interviewers were blinded to treatment. Interview discussion guides were designed to elucidate the signs, symptoms, and impacts reported in Becker individuals, participants’ trial experiences, and what would constitute a meaningful treatment benefit. Questions about the relevance of the North Star Ambulatory Assessment (NSAA) were asked in ARCH.
Participants described a range of signs, symptoms, and impacts as part of their lived experience with Becker. The most common symptoms were general muscle weakness (mainly in the lower body) (n=31), limited endurance (n=29), and pain, soreness, and/or cramping (n=28), which impacted participants’ physical and socio-emotional functioning. Although participants indicated that improvements in functioning or reversing the effects of Becker would be ideal, among those asked (n = 26), all noted that functional stability or slowing of disease progression would be an important and meaningful treatment outcome.
Exit interviews facilitate a deeper understanding of patient experiences and are used as a method to contextualize the meaningfulness of treatment outcomes. Improvements in symptoms/impacts and disease stabilization or slowing of progression are acknowledged as meaningful treatment benefits. These interviews provide support for the relevance of concepts assessed in the NSAA to Becker patients.