Decision analysis, economic evaluation, and newborn screening: challenges and opportunities

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 evaluations can help inform policy decisions for newborn screening programs by providing a systematic approach to synthesizing available evidence and providing projected estimates of long-term clinical and economic outcomes when long-term data are not available. In this review, we outline the types of data required for the development of decision analysis and cost-effectiveness models for newborn screening programs and discuss the challenges faced when applying these methods in the arena of newborn screening to help inform policy decisions. 2012:14(8):703-712 Genet Med

Newborn Screening for Congenital Adrenal Hyperplasia in New Zealand, 1994–2013

“…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.…”

Predicting Health Utilities for Children With Autism Spectrum Disorders

“…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.…”

Decision-making process for conditions nominated to the Recommended Uniform Screening Panel: statement of the US Department of Health and Human Services Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children

“…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.…”