Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data

Ploeg, C.P.B. van der; Marle, M. Elske van den Akker-van; Langen, A.M.M. Vernooij-van; Elvers, L.H.; Gille, Johan J. P.; Verkerk, P.H.; Dankert-Roelse, J.E.

doi:10.1016/j.jcf.2014.08.007

Cited by 30 publications

(46 citation statements)

References 26 publications

(37 reference statements)

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“…Two subsequent CEAs from the Netherlands both assumed a 1.5 percentage point reduction in mortality to age 5 years, similar to the WDOH base model assumption of a 2 percentage point reduction to age 10 years and consistent with published findings . An individual‐level analysis of data from the Cystic Fibrosis Foundation CF Patient Registry found a similar survival advantage for infants whose diagnosis was reported during the first month of life, and a state‐level analysis found a 1.7 percentage point difference in mortality through age 9 years in states with and without CF NBS implemented prior to 1996 …”

Section: Washington State Nbs Policy Process and Economic Evaluationssupporting

confidence: 76%

“…However, the Wisconsin randomized trial found no difference in hospitalization costs with NBS . A partial economic analysis of CF screening in Wisconsin assumed no difference in hospitalization costs, as did the two Dutch CEAs . At the other end of the spectrum, a recent Canadian CEA of screening for CF assumed 85% lower hospitalization costs with NBS .…”

Section: Washington State Nbs Policy Process and Economic Evaluationsmentioning

confidence: 99%

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The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience

Grosse

Thompson

Ding

et al. 2016

The Milbank Quarterly

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Section: Washington State Nbs Policy Process and Economic Evaluationssupporting

confidence: 76%

Section: Washington State Nbs Policy Process and Economic Evaluationsmentioning

confidence: 99%

The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience

Grosse

Thompson

Ding

et al. 2016

The Milbank Quarterly

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“…The California findings are consistent with those modeled in a cost-effectiveness study of 4 different CF NBS algorithms. 30 However, 2 recent studies 31,32 suggest that IRT/pancreatitis-associated protein algorithms may be even more cost-effective than California's 3-step algorithm.…”

Section: Resultsmentioning

confidence: 99%

Newborn Screening for Cystic Fibrosis in California

et al. 2015

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, on behalf of the California Cystic Fibrosis Newborn Screening Consortium abstract OBJECTIVES: This article describes the methods used and the program performance results for the first 5 years of newborn screening for cystic fibrosis (CF) in California.METHODS: From July 16, 2007, to June 30, 2012, a total of 2 573 293 newborns were screened for CF by using a 3-step model: (1) measuring immunoreactive trypsinogen in all dried blood spot specimens; (2) testing 28 to 40 selected cystic fibrosis transmembrane conductance regulator (CFTR) mutations in specimens with immunoreactive trypsinogen values $62 ng/mL (top 1.6%); and (3) performing DNA sequencing on specimens found to have only 1 mutation in step 2. Infants with $2 mutations/variants were referred to CF care centers for diagnostic evaluation and follow-up. Infants with 1 mutation were considered carriers and their parents offered telephone genetic counseling.RESULTS: Overall, 345 CF cases, 533 CFTR-related metabolic syndrome cases, and 1617 carriers were detected; 28 cases of CF were missed. Of the 345 CF cases, 20 (5.8%) infants were initially assessed as having CFTR-related metabolic syndrome, and their CF diagnosis occurred after age 6 months (median follow-up: 4.5 years). Program sensitivity was 92%, and the positive predictive value was 34%. CF prevalence was 1 in 6899 births. A total of 303 CFTR mutations were identified, including 78 novel variants. The median age at referral to a CF care center was 34 days (18 and 37 days for step 2 and 3 screening test-positive infants, respectively). CONCLUSIONS:The 3-step model had high detection and low false-positive levels in this diverse population. WHAT'S KNOWN ON THIS SUBJECT:Several newborn screening models for cystic fibrosis (CF) exist, including DNA-based models that use mutation panels. There is limited experience with models (such as used in California) that include comprehensive DNA sequence testing methods as part of newborn screening.WHAT THIS STUDY ADDS: California' s 3-step newborn screening model for CF showed high efficiency, sensitivity, and positive predictive value. More than 300 mutations were found, reflecting the state' s diverse population. Some CF transmembrane conductance regulator-related metabolic syndrome cases converted to CF over time.

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“…While the PPV was lower than the goal of 0.3, the test protocol avoided detection of carriers and equivocal diagnoses. A cost analysis found that the IRT/PAP method had greater economic benefit …”

Section: Diagnosismentioning

confidence: 99%

Pediatric Pulmonologyyear in review 2015: Part 4

Savant

McColley

2016

Pediatr Pulmonol.

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Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data

Abstract: NBSCF is as an economically justifiable public health initiative. Of the four strategies tested IRT-PAP is the most economic and this finding should be included in any decision making model, when considering implementation of newborn screening for CF.

Cited by 30 publications

References 26 publications

The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience

The Use of Economic Evaluation to Inform Newborn Screening Policy Decisions: The Washington State Experience

Newborn Screening for Cystic Fibrosis in California

Pediatric Pulmonologyyear in review 2015: Part 4

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