OBJECTIVES:

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.

METHODS:

The Institute for Clinical and Economic Review’s 2019 DMD model was replicated and adapted for early non-ambulatory patients. The base case assessed a 13-year-old early non-ambulatory patient. Assumptions regarding utilities (0.21 at baseline [early non-ambulatory] progressing to 0.18 [late non-ambulatory]), health-state transitions, costs, and treatment benefits (10-, 20-, and 40-year pause in disease progression) were replicated per the published report. Assessments included maximum cost-effective treatment cost at willingness-to-pay thresholds of $50,000/QALY, $100,000/QALY, and $150,000/QALY.

RESULTS:

At $100,000/QALY or less, net non-treatment costs exceeded treatment benefits, implying no cost-effectiveness of any treatment, irrespective of the amount of benefit, even at a $0 treatment cost. At $150,000/QALY, annual maximum treatment costs ranged from $260–$430, which is less than the annual maximum treatment cost of generic prednisone ($550).

CONCLUSIONS:

These results support previously described concerns regarding QALY assessments for individuals with disabilities. Low baseline health-state utility values for early non-ambulatory patients, who still maintain upper body function and independence, have a substantial and concerning impact on QALY value assessments of DMD treatments.