Abstract
BackgroundThe use of neonatal screening for cystic fibrosis is widely debated in the United Kingdom and elsewhere, but the evidence available to inform policy is limited. This paper explores the cost-effectiveness of adding screening for cystic fibrosis to an existing routine neonatal screening programme for congenital hypothyroidism and phenylketonuria, under alternative scenarios and assumptions.MethodsThe study is based on a decision model comparing screening to no screening in terms of a number of outcome measures, including diagnosis of cystic fibrosis, life-time treatment costs, life years and QALYs gained. The setting is a hypothetical UK health region without an existing neonatal screening programme for cystic fibrosis.ResultsUnder initial assumptions, neonatal screening (using an immunoreactive trypsin/DNA two stage screening protocol) costs £5,387 per infant diagnosed, or £1.83 per infant screened (1998 costs). Neonatal screening for cystic fibrosis produces an incremental cost-effectiveness of £6,864 per QALY gained, in our base case scenario (an assumed benefit of a 6 month delay in the emergence of symptoms). A difference of 11 months or more in the emergence of symptoms (and mean survival) means neonatal screening is both less costly and produces better outcomes than no screening.ConclusionNeonatal screening is expensive as a method of diagnosis. Neonatal screening may be a cost-effective intervention if the hypothesised delays in the onset of symptoms are confirmed. Implementing both antenatal and neonatal screening would undermine potential economic benefits, since a reduction in the birth incidence of cystic fibrosis would reduce the cost-effectiveness of neonatal screening.
Highlights
The use of neonatal screening for cystic fibrosis is widely debated in the United Kingdom and elsewhere, but the evidence available to inform policy is limited
Cystic fibrosis is an inherited disorder associated with considerable morbidity and reduced life expectancy
Given the short-comings of these studies, and the lack of full economic evaluations based on UK service and survival data, we aimed to compare the lifetime cost-effectiveness of neonatal screening with no screening, under different possible scenarios for survival with cystic fibrosis
Summary
The use of neonatal screening for cystic fibrosis is widely debated in the United Kingdom and elsewhere, but the evidence available to inform policy is limited. A recent report from the UK NHS Health Technology Assessment Programme recommended that antenatal screening for cystic fibrosis should be offered routinely, and that "Health Authorities could consider introducing neonatal screening" [3]. Antenatal screening aims to prevent affected births and neonatal screening aims to improve prognosis by early intervention. Studies of the effectiveness of neonatal screening have measured short-term outcomes or are subject to statistical biases, including selection and lead-time bias, and the use of historical controls [4,5,6,7,8]. The ability of neonatal screening to alter long-term prognosis is not proven, there is limited circumstantial evidence favouring a benefit [3]
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