Open AccessEliciting Health State Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Vignette Study in France
Author(s) -
Adam B. Smith,
Andria Hanbury,
Igor Beitia Ortiz de Zárate,
F. Hammès,
Gérard de Pouvourville,
K. Buesch
Publication year - 2021
Publication title -
patient related outcome measures
Language(s) - English
Resource type - Journals
ISSN - 1179-271X
DOI - 10.2147/prom.s306228
Subject(s) - vignette , medicine , quality of life (healthcare) , time trade off , health utilities index , eq 5d , population , gerontology , pediatrics , health related quality of life , environmental health , disease , pathology , nursing , social psychology , psychology
Health-related quality of life (HRQoL) is difficult to measure in rare diseases, especially in paediatric populations, yet capturing HRQoL is critical to evaluating treatment, including the cost-effectiveness of treatments. Given the ultra-rare nature of AADC deficiency 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 population. The aim of the study was to generate health utility values specific for France for AADC deficiency using vignettes.