Health utilities and parental quality of life effects for three rare conditions tested in newborns.

ABSTRACT:

Background

Measurement of health utilities is required for economic evaluations. Few studies have evaluated health utilities for rare conditions; even fewer have incorporated disutility that may be experienced by caregivers. This study aimed to (1) estimate health utilities for three rare conditions currently recommended for newborn screening at the state or federal level, and (2) estimate the disutility, or spillover, experienced by parents of patients diagnosed with a rare, heritable disorder.

Methods

A stated-preference survey using a time trade-off approach elicited health utilities for Krabbe disease, phenylketonuria, and Pompe disease at varying stages (mild, moderate, severe) and onset of disease symptoms (infancy, childhood, and adulthood). We recruited respondents f

SUBMITTER: Simon NJ 

PROVIDER: S-EPMC6342747 | biostudies-literature | 2019 Jan

REPOSITORIES: biostudies-literature