Newborn screening for severe combined immunodeficiency cost effective in the Netherlands?

Abstract

A cost-utility analysis based on real-world data has found that newborn screening for severe combined immunodeficiency (SCID) may be cost effective in the Netherlands, primarily for screening strategies that result in fewer referrals, say researchers who published in the International Journal of Newborn Screening. The analysis used data from the prospective SONNET trial, in which all parents of newborns born in three of the twelve provinces of the Netherlands were asked to participate in a trial of different newborn screening strategies using dried blood spots for SCID. The strategies tested were: no screening; referral for TREC* results recommended by the test manufacturer (≤6 copies per 3.2mm punch); referral for TREC results of ≤10 copies/3.2mm; and, a new strategy that distinguished between urgent referrals (TREC levels ≤2 copies/3.2mm) and those requiring a second heel prick after 7 days (TREC levels 2-10 copies/3.2mm). Costs** were assessed using a microcosting technique. Cost of screening consisted of costs of the TREC assay, laboratory equipment, material and personnel. An existing decision analysis model was adapted to compare the costs and utilities of each strategy over a lifetime, from a healthcare payer perspective. The costs of testing on first heel prick was determined at €6.36 per card, with repeat first heel pricks and second heel pricks estimated at €79.03. The cost to refer a child was €145. Compared with no screening, the incremental cost-effectiveness ratio (ICER) of the strategy to refer at TREC ≤6 copies/3.2mm was €41 300 per quality-adjusted life year (QALY) and the ICER for the new strategy was €41 600 per QALY; for the strategy with TREC ≤10 copies/3.2mm, the ICER was €44 100 per QALY. The researchers note that these ICERs are higher than their previous estimate of €33 400 per QALY based on literature data and expert opinions. However, these higher ICERs obtained with real-life data are still in the range of the willingness to pay values of €20 000 to €80 000 per QALY that are considered acceptable in the Netherlands .