FTI Center for Healthcare Economics and Policy’s Jason Shafrin, PhD, and Nadine Zawadzki, PhD, and Moises Marin, recently published an article quantifying caregiver risk preferences to inform the “value of hope” for Duchenne Muscular Dystrophy (DMD) therapies affecting time to loss of ambulation (LoA). The article was published in Current Medical Research and Opinion and is titled “Caregiver Risk Preferences for Delaying Loss of Ambulation in Duchenne Muscular Dystrophy in the United States.” The article abstract is below.
ABSTRACT
Objective
Quantify caregiver risk preferences to inform the “value of hope” for Duchenne muscular dystrophy (DMD) therapies affecting time to loss of ambulation (LoA).
Methodology
Caregivers (medical decision-makers) of patients with DMD were surveyed to evaluate their preferences across 2 therapies with identical expected (average) time to LoA: 1 with variable (i.e. possibly longer or shorter than average) time to LoA and 1 with fixed (i.e. certain) time to LoA. Time to LoA with the fixed therapy was altered to determine the caregiver’s indifference point. Study endpoints were (i) the share of caregivers who preferred the variable (vs fixed) time to LoA therapy; and (ii) the length of fixed time to LoA that would result in caregiver indifference between the variable and fixed therapies, calculated using parameter estimation by sequential testing. The base case examined therapy choice for a hypothetical ambulatory DMD patient aged 9 years; sensitivity analyses explored preferences for younger (aged 5) and older (aged 13) patients.
Results
Among 103 caregivers surveyed, 72 (69.9%) preferred the variable time to LoA therapy for a hypothetical 9-year-old patient with DMD (p < 0.001). Caregivers were willing to give up 11.5 months (p < 0.001) of certain time to LoA for a chance of longer time to LoA. Caregivers’ preference for the variable therapy decreased with hypothetical patient age at treatment initiation, from 72.8% (75/103) for age 5 (p < 0.001) to 60.2% (62/103) for age 13 (p = 0.048).
Conclusion
Caregivers of patients with DMD demonstrated risk tolerance (positive value of hope) for therapies that could delay LoA.
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