Literature DB >> 26876279

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

Yao Ding1, John D Thompson2, Lisa Kobrynski3, Jelili Ojodu1, Guisou Zarbalian1, Scott D Grosse4.   

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. Published by Elsevier Inc.

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Year:  2016        PMID: 26876279      PMCID: PMC4846488          DOI: 10.1016/j.jpeds.2016.01.029

Source DB:  PubMed          Journal:  J Pediatr        ISSN: 0022-3476            Impact factor:   4.406


  46 in total

1.  Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

Authors:  Belinda Chan; Diane Wara; John Bastian; Michael S Hershfield; John Bohnsack; Colleen G Azen; Robertson Parkman; Kenneth Weinberg; Donald B Kohn
Journal:  Clin Immunol       Date:  2005-08-22       Impact factor: 3.969

2.  Newborn screening for severe combined immunodeficiency; the Wisconsin experience (2008-2011).

Authors:  James W Verbsky; Mei W Baker; William J Grossman; Mary Hintermeyer; Trivikram Dasu; Benedetta Bonacci; Sreelatha Reddy; David Margolis; James Casper; Miranda Gries; Ken Desantes; Gary L Hoffman; Charles D Brokopp; Christine M Seroogy; John M Routes
Journal:  J Clin Immunol       Date:  2011-11-10       Impact factor: 8.317

Review 3.  International perspectives on the cost-effectiveness of tandem mass spectrometry for rare metabolic conditions.

Authors:  Richard Norman; Marion Haas; Bridget Wilcken
Journal:  Health Policy       Date:  2008-09-26       Impact factor: 2.980

4.  Projected costs, risks, and benefits of expanded newborn screening for MCADD.

Authors:  Lisa A Prosser; Chung Yin Kong; Donna Rusinak; Susan L Waisbren
Journal:  Pediatrics       Date:  2010-02       Impact factor: 7.124

5.  Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data.

Authors:  Marie Caroline Clément; Nizar Mahlaoui; Cécile Mignot; Christine Le Bihan; Hasina Rabetrano; Ly Hoang; Bénédicte Neven; Despina Moshous; Marina Cavazzana; Stéphane Blanche; Alain Fischer; Marie Audrain; Isabelle Durand-Zaleski
Journal:  J Allergy Clin Immunol       Date:  2015-04-01       Impact factor: 10.793

6.  Fiscal implications of newborn screening in the diagnosis of severe combined immunodeficiency.

Authors:  Catherine Kubiak; Soma Jyonouchi; Caroline Kuo; Maria Garcia-Lloret; Morna J Dorsey; John Sleasman; Arthur S Zbrozek; Elena E Perez
Journal:  J Allergy Clin Immunol Pract       Date:  2014-08-28

7.  Comprehensive cost-utility analysis of newborn screening strategies.

Authors:  Aaron E Carroll; Stephen M Downs
Journal:  Pediatrics       Date:  2006-05       Impact factor: 7.124

8.  Budget impact analysis-principles of good practice: report of the ISPOR 2012 Budget Impact Analysis Good Practice II Task Force.

Authors:  Sean D Sullivan; Josephine A Mauskopf; Federico Augustovski; J Jaime Caro; Karen M Lee; Mark Minchin; Ewa Orlewska; Pete Penna; Jose-Manuel Rodriguez Barrios; Wen-Yi Shau
Journal:  Value Health       Date:  2013-12-13       Impact factor: 5.725

9.  Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years.

Authors:  Antonia Kwan; Joseph A Church; Morton J Cowan; Rajni Agarwal; Neena Kapoor; Donald B Kohn; David B Lewis; Sean A McGhee; Theodore B Moore; E Richard Stiehm; Matthew Porteus; Constantino P Aznar; Robert Currier; Fred Lorey; Jennifer M Puck
Journal:  J Allergy Clin Immunol       Date:  2013-07       Impact factor: 10.793

10.  Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID).

Authors:  Claire Booth; H Bobby Gaspar
Journal:  Biologics       Date:  2009-07-13
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  9 in total

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

Authors:  Scott D Grosse; John D Thompson; Yao Ding; Michael Glass
Journal:  Milbank Q       Date:  2016-06       Impact factor: 4.911

Review 2.  Newborn Screening for Severe Combined Immunodeficiency.

Authors:  John Routes; James Verbsky
Journal:  Curr Allergy Asthma Rep       Date:  2018-05-10       Impact factor: 4.806

3.  Using Decision Analysis to Support Newborn Screening Policy Decisions: A Case Study for Pompe Disease.

Authors:  Lisa A Prosser; K K Lam; Scott D Grosse; Mia Casale; Alex R Kemper
Journal:  MDM Policy Pract       Date:  2018-04-18

4.  First Universal Newborn Screening Program for Severe Combined Immunodeficiency in Europe. Two-Years' Experience in Catalonia (Spain).

Authors:  Ana Argudo-Ramírez; Andrea Martín-Nalda; Jose L Marín-Soria; Rosa M López-Galera; Sonia Pajares-García; Jose M González de Aledo-Castillo; Mónica Martínez-Gallo; Marina García-Prat; Roger Colobran; Jacques G Riviere; Yania Quintero; Tatiana Collado; Judit García-Villoria; Antonia Ribes; Pere Soler-Palacín
Journal:  Front Immunol       Date:  2019-10-22       Impact factor: 7.561

5.  Expanding TREC and KREC Utility in Primary Immunodeficiency Diseases Diagnosis.

Authors:  Ilya Korsunskiy; Oleg Blyuss; Maria Gordukova; Natalia Davydova; Alexey Zaikin; Natalia Zinovieva; Sergey Zimin; Robert Molchanov; Aminat Salpagarova; Alina Eremeeva; Maxim Filipenko; Andrey Prodeus; Anatoliy Korsunskiy; Peter Hsu; Daniel Munblit
Journal:  Front Immunol       Date:  2020-03-03       Impact factor: 7.561

6.  Economic Evaluation of Newborn Screening for Severe Combined Immunodeficiency.

Authors:  Sophy T F Shih; Elena Keller; Veronica Wiley; Melanie Wong; Michelle A Farrar; Georgina M Chambers
Journal:  Int J Neonatal Screen       Date:  2022-07-20

7.  Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency.

Authors:  Sophy T F Shih; Elena Keller; Veronica Wiley; Michelle A Farrar; Melanie Wong; Georgina M Chambers
Journal:  Int J Neonatal Screen       Date:  2022-07-20

Review 8.  Population-based screening of newborns: Findings from the newborn screening expansion study (part two).

Authors:  Kee Chan; Amy Brower; Marc S Williams
Journal:  Front Genet       Date:  2022-09-01       Impact factor: 4.772

9.  Cost and Cost-Effectiveness Assessments of Newborn Screening for Critical Congenital Heart Disease Using Pulse Oximetry: A Review.

Authors:  Scott D Grosse; Cora Peterson; Rahi Abouk; Jill Glidewell; Matthew E Oster
Journal:  Int J Neonatal Screen       Date:  2017-12-14
  9 in total

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