EE291 Ethical Implications of Quality-Adjusted Life Year Assessments for Patients with Disabilities: A Duchenne Muscular Dystrophy Case Study

Abstract

Quality-adjusted life years (QALYs) are traditionally used to assess the value of treatments, but have been questioned for individuals with disabilities, particularly when there is an inability to achieve high utilities compared with healthier individuals. Duchenne muscular dystrophy (DMD) is a progressive genetic neuromuscular disease, resulting in loss of ambulation (LOA) by early teenage years and premature mortality. LOA results in diminished health utility; however, early non-ambulatory phase maintenance of upper body function allows individuals to retain a high degree of independence and participate in daily activities. Current DMD therapies are approved regardless of ambulatory status and aim to slow disease progression. This study assesses the impact of a QALY assessment focusing on the maximum treatment cost of a hypothetical treatment for non-ambulatory patients with DMD.