Abstract:reviewThe number of conditions included in newborn screening panels has increased rapidly in the United States during the past decade, and many more conditions are under consideration for addition to state panels. The rare nature of candidate conditions for newborn screening makes their evaluation challenging. The scarcity of data on the costs of screening, follow-up, treatment, and long-term disability must be addressed to improve the evaluation process for nominated conditions. Decision analyses and economic… Show more
“…The major advantage of this approach is to retain a relatively high PPV and minimize screening costs. Cost-effectiveness is a major barrier to international uptake of newborn screening for CAH, and analyses are complicated by the uncertain public health impact of missed or delayed diagnoses (18). While a few cases of simple virilising disease were also detected by our screening program, we would expect the doi: 10.1210/jc.2014-3168 jcem.endojournals.org 5 majority to be diagnosed later in childhood.…”
Screening alone accounted for nearly 50% cases of CAH detected in the newborn period, including a fifth of affected females, indicating that clinical diagnosis is unreliable in both genders. Symptoms were mild at diagnosis and there were no adrenal crises. This study confirms the benefits of newborn CAH screening.
“…The major advantage of this approach is to retain a relatively high PPV and minimize screening costs. Cost-effectiveness is a major barrier to international uptake of newborn screening for CAH, and analyses are complicated by the uncertain public health impact of missed or delayed diagnoses (18). While a few cases of simple virilising disease were also detected by our screening program, we would expect the doi: 10.1210/jc.2014-3168 jcem.endojournals.org 5 majority to be diagnosed later in childhood.…”
Screening alone accounted for nearly 50% cases of CAH detected in the newborn period, including a fifth of affected females, indicating that clinical diagnosis is unreliable in both genders. Symptoms were mild at diagnosis and there were no adrenal crises. This study confirms the benefits of newborn CAH screening.
“…The generic instruments for measuring health utilities are applicable for a wide range of health conditions and treatments, although there are special issues in applications involving children (Ungar 2010; Prosser et al 2007; Grosse et al 2010; Prosser et al 2012; Payakachat et al 2012; Ungar 2007; Ungar 2011). In particular, evaluation of health utilities in young children (<5) is especially difficult as instruments are not designed for this age group and typically requires out-of-sample prediction.…”
Scientific Abstract
Comparative effectiveness of interventions for children with ASDs that incorporates costs is lacking due to the scarcity of information on health utility scores or preference-weighted outcomes typically used for calculating quality-adjusted life years (QALYs). This study created algorithms for mapping clinical and behavioral measures for children with ASDs to health utility scores. The algorithms could be useful for estimating the value of different interventions and treatments used in the care of children with ASDs. Participants were recruited from two Autism Treatment Network sites. Health utility data based on the HUI3 for the child was obtained from the primary caregiver (proxy-reported) through a survey (N=224). During the initial clinic visit, proxy-reported measures of the Child Behavior Checklist, Vineland II Adaptive Behavior Scales, and the PedsQL 4.0 (start measures) were obtained and then merged with the survey data. Nine mapping algorithms were developed using the HUI3 scores as dependent variables in ordinary least squares regressions along with the start measures, the Autism Diagnostic Observation Schedule to measure severity, child age, and cognitive ability as independent predictors. In-sample cross-validation was conducted to evaluate predictive accuracy. Multiple imputation techniques were used for missing data. The average age for children with ASDs in this study was 8.4 (SD=3.5) years. Almost half of the children (47%) had cognitive impairment (IQ<=70). Total scores for all of the outcome measures were significantly associated with the HUI3 score. The algorithms can be applied to clinical studies containing start measures of children with ASDs to predict QALYs gained from interventions.
“…This model will illustrate the estimated upper and lower ranges of predicted net benefit and harm to the population associated with newborn screening as compared with usual care with clinical identification. 6 After review of the evidence report, the Advisory Committee will now assign one of five ratings to nominated conditions (Table 1) on the basis of consensus regarding the magnitude and certainty of population net benefit. The most difficult decision anticipated for the Advisory Committee will be in determining an A versus B rating.…”
Section: Assigning the Magnitude And Certainty Of Net Benefitmentioning
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