Abstract
Health-related quality of life (HRQoL) is difficult to measure in rare diseases, especially in pediatric populations, yet capturing HRQoL is critical for evaluating treatment, including the cost-effectiveness of treatments. Given the ultra-rare nature of AADC deficiency (AADC-d) indirect elicitation of HRQoL data through proxy caregiver/parent ratings is not feasible. In these circumstances HRQoL data may be derived through vignette studies using the general public. The study aim was to generate health utilities for AADC-d. The study was completed online by panel participants from a French representative sample. Five health state vignettes, reflecting key milestones in the eladocagene exuparvovec clinical trials and economic model were presented to the participants: bedridden, head control, sitting unsupported, standing with assistance and walking with assistance. Participants have then valued the vignettes through a revealing preferences method (standard gamble and time trade-off). The vignettes had been previously developed with input from parents of AADC-d patients and expert opinions. A total of 1001 participants (51% females; mean age 46 years) completed the study. Utilities increased linearly as the health state improved for both the time trade-off (TTO), and standard gamble (SG). A significant minority had incongruent responses (high utilities for the bedridden compared to walking health states): 27% for TTO and 34% for SG. When these were removed the mean TTO health utilities (n=729) were: 0.3891 (bedridden state), 0.4839 (head control), 0.5271 (sitting unsupported), 0.5293 (standing with assistance) and 0.5577 (walking with assistance). The SG utilities (n=664) ranged from 0.5534 for bedridden to 0.7093 for walking with assistance. Health utilities derived for AADC-d through vignettes are suitable for use as a cost-effectiveness model of an AADC-d treatment.
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