Purpose This study aimed to develop a utility index (the
ABC-UI) from the Aberrant Behavior Checklist-Community
(ABC-C), for use in quantifying the benefit of
emerging treatments for fragile X syndrome (FXS).
Methods The ABC-C is a proxy-completed assessment of
behaviour and is a widely used measure in FXS. A subset
of ABC-C items across seven dimensions was identified to
include in health state descriptions. This item reduction
process was based on item performance, factor analysis and
Rasch analysis performed on an observational study dataset,
and consultation with five clinical experts and a
methodological expert. Dimensions were combined into
health states using an orthogonal design and valued using
time trade-off (TTO), with lead-time TTO methods used
where TTO indicated a state valued as worse than dead.
Preference weights were estimated using mean, individual
level, ordinary least squares and random-effects maximum
likelihood estimation [RE (MLE)] regression models.
Results A representative sample of the UK general public
(n = 349; mean age 35.8 years, 58.2 % female) each valued
12 health states. Mean observed values ranged from
0.92 to 0.16 for best to worst health states. The RE (MLE)
model performed best based on number of significant
coefficients and mean absolute error of 0.018. Mean utilities
predicted by the model covered a similar range to that
observed.
Conclusions The ABC-UI estimates a wide range of
utilities from patient-level FXS ABC-C data, allowing
estimation of FXS health-related quality of life impact for
economic evaluation from an established FXS clinical trial
instrument.
CORE (COnnecting REpositories)
CORE (COnnecting REpositories)