A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK.

Alice Bessey,James Chilcott,Carmen De La Cruz,Ruth Wong,Joanna Leaviss

doi:10.3390/ijns5030028

Abstract

Severe combined immunodeficiency (SCID) can be detected through newborn bloodspot screening. In the UK, the National Screening Committee (NSC) requires screening programmes to be cost-effective at standard UK thresholds. To assess the cost-effectiveness of SCID screening for the NSC, a decision-tree model with lifetable estimates of outcomes was built. Model structure and parameterisation were informed by systematic review and expert clinical judgment. A public service perspective was used and lifetime costs and quality-adjusted life years (QALYs) were discounted at 3.5%. Probabilistic, one-way sensitivity analyses and an exploratory disbenefit analysis for the identification of non-SCID patients were conducted. Screening for SCID was estimated to result in an incremental cost-effectiveness ratio (ICER) of £18,222 with a reduction in SCID mortality from 8.1 (5–12) to 1.7 (0.6–4.0) cases per year of screening. Results were sensitive to a number of parameters, including the cost of the screening test, the incidence of SCID and the disbenefit to the healthy at birth and false-positive cases. Screening for SCID is likely to be cost-effective at £20,000 per QALY, key uncertainties relate to the impact on false positives and the impact on the identification of children with non-SCID T Cell lymphopenia.

Highlights

Screening newborns for severe combined immunodeficiency (SCID) using T-cell receptor excision circles (TRECs) on the blood spot was added to the core US Recommended Uniform Screening Panel in 2010 [1]
SCID; T Cell lymphopenia (TCL)—T-cell imlomweurninothdeesficrceeineendcayrm; ;HhoSwCevTe—r, thheelomnga-tteormpofoilelotwic-usptaenmd mcaenlalgetmraenntscposltas narte;hQighAerLtYhasn—in Quality-adjusted life years; incremental cost-effectiveness ratio (ICER)—Incremetshanemtneaol-oscrcorleosewtneearrffmien. cWtthihevileescntrheeeesnpseedrrpaaarttmiieo,n.tthceostisncarreealsoewienr, and the survival rates of complications are either the increases the number of patients experiencing both complications and the general follow-up costs leading to overall higher costs
TREC—T-cell receptor excision circles; quality-adjusted life years (QALYs)—Quality-adjusted life years, CI—Confidence interval. This CEA of SCID screening is the first to include QALY estimates based on SCID patients, UK-specific data from the two UK SCID treatment centres, and an expected value of perfect information (EVPI) analysis and is the first to attempt to quantify the impact on non-SCID patients

Summary

Introduction

Screening newborns for severe combined immunodeficiency (SCID) using T-cell receptor excision circles (TRECs) on the blood spot was added to the core US Recommended Uniform Screening Panel in 2010 [1]. Within the UK, the National Screening Committee (NSC) advises government and the National Health Service (NHS) concerning what newborn screening tests should be offered. This advice is based on independent assessments of the evidence of the potential benefits, harms and cost-effectiveness of each screening test [4]. There is evidence that earlier diagnosis substantially reduces the cost of treating SCID through reduced infection rates and subsequent hospitalisations [10,11]. As there are differences between countries in terms of treatment costs and benefits and how health interventions are assessed, a UK-specific cost-effectiveness model was developed

Methods

Results

Conclusion