Pulmonary benefits of treating Duchenne muscular dystrophy A regulatory benefit-risk analysis [meeting abstract psy64]
This study demonstrates a strong preference for therapies with a pulmonary benefit as illustrated by a willingness to trade-off risks and burden. Specifically, in exchange for maintaining cough strength for 10 years, respondents are willing to tolerate high probabilities of diarrhea and the burden of additional blood draws. Although not powered to detect statistically significant differences, we found no qualitative differences in preferences between caregivers and patients.
Hollin, IL., Peay, H., & Bridges, JF. (2016). Pulmonary benefits of treating Duchenne muscular dystrophy: A regulatory benefit-risk analysis [meeting abstract psy64]. Value in Health, 19(3), A252. https://doi.org/10.1016/j.jval.2016.03.1004