Cost-Effectiveness/Cost-Benefit Analysis of Newborn Screening for Severe Combined Immune Deficiency in Washington State

Abstract

Objective To evaluate the expected cost-effectiveness and net benefit of the recent implementation of newborn screening (NBS) for severe combined immunodeficiency (SCID) in Washington State. Study design We constructed a decision analysis model to estimate the costs and benefits of NBS in an annual birth cohort of 86 600 infants based on projections of avoided infant deaths. Point estimates and ranges for input variables, including the birth prevalence of SCID, proportion detected asymptomatically without screening through family history, screening test characteristics, survival rates, and costs of screening, diagnosis, and treatment were derived from published estimates, expert opinion, and the Washington NBS program. We estimated treatment costs stratified by age of identification and SCID type (with or without adenosine deaminase deficiency). Economic benefit was estimated using values of $4.2 and $9.0 million per death averted. We performed sensitivity analyses to evaluate the influence of key variables on the incremental cost-effectiveness ratio (ICER) of net direct cost per life-year saved. Results Our model predicts an additional 1.19 newborn infants with SCID detected preclinically through screening, in addition to those who would have been detected early through family history, and 0.40 deaths averted annually. Our base-case model suggests an ICER of $35 311 per life-year saved, and a benefit-cost ratio of either 5.31 or 2.71. Sensitivity analyses found ICER values <$100 000 and positive net benefit for plausible assumptions on all variables. Conclusions Our model suggests that NBS for SCID in Washington is likely to be cost-effective and to show positive net economic benefit.

Introducing Newborn Screening for Severe Combined Immunodeficiency (SCID) in the Dutch Neonatal Screening Program

“…The long-term treatment costs would be lower as well, as early transplantation results in more favourable health outcomes. The results for the Netherlands are comparable with cost-effectiveness studies in the United States [14,15] and indicate that SCID screening might be cost-effective, but the range of possible cost-effectiveness ratios is broad due to many parameter-associated uncertainties such as the incidence of SCID, costs of screening tests and costs of late transplantation. In conclusion, SCID screening in the Netherlands could be cost-effective but, due to many uncertainties, an extensive pilot study should be performed to help actualise the results of this CEA.…”

“…Cost-effectiveness studies for newborn screening for SCID have already been performed in the United States and New Zealand [14][15][16]. However, as costs and benefits of screening and treatment are likely to differ between countries and especially between continents, a cost-effectiveness analysis (CEA) for SCID was carried out by the Netherlands Organisation for applied scientific research (TNO) in collaboration with Leiden University Medical Centre (LUMC).…”

Newborn Screening for Severe Combined Immunodeficiency-A History of the TREC Assay

“…Optimization of the assay and development of screening algorithms have made NBS for SCID life-saving, while cost-benefit analysis has repeatedly illustrated the economic advantage of early diagnosis [1,[33][34][35][36]. As a result, as of January 2017, the TREC assay is being used to screen for T cell lymphopenias and SCID in all but one state in the US, the District of Columbia, and Puerto Rico (Figure 3).…”

“…Early identification allows for treatment prior to the onset of often fatal infections and improves outcomes. HSCT prior to age 3.5 months results in survival rates of 94%, compared to 66-68% when transplanted after 3.5 months [33].…”

“…The ability to detect infants with SCID before the acquisition of SCID defining illnesses is critical to reduce morbidity and mortality in these infants. The cumulative survival rate for infants identified early by NBS is approximately 87% [2], compared to 54% for late identified infants [33]. Early identification allows for treatment prior to the onset of often fatal infections and improves outcomes.…”