Article ID Journal Published Year Pages File Type
5825504 Clinical Therapeutics 2014 14 Pages PDF
Abstract
We present a model process for advocacy organizations aiming to promote patient-centered drug development. The community-engaged approach was successfully used to develop and implement a survey to measure caregiver preferences. Caregivers were willing to accept a serious risk when balanced with a noncurative treatment, even absent improvement in life span. These preferences should inform the Food and Drug Administration's benefit-risk assessment of emerging DMD therapies. This study highlights the synergistic integration of traditional advocacy methods and scientific approach to quantify benefit-risk preferences.
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