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

被引:16
|
作者
Bessey, Alice [1 ]
Chilcott, James [1 ]
Leaviss, Joanna [1 ]
de la Cruz, Carmen [1 ]
Wong, Ruth [1 ]
机构
[1] Univ Sheffield, Sch Hlth & Related Res, Sheffield S1 4DA, S Yorkshire, England
关键词
severe combined immunodeficiency; SCID; cost-effectiveness; economic; newborn screening; neonatal screening; STEM-CELL TRANSPLANTATION; TERM IMMUNE RECONSTITUTION; MIXED METHODS; DIAGNOSIS; OUTCOMES; LYMPHOPENIA; EUROQOL; BENEFIT; CHILD;
D O I
10.3390/ijns5030028
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
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 pound 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 pound 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.
引用
收藏
页数:15
相关论文
共 50 条
  • [1] Cost-effectiveness of newborn screening for severe combined immunodeficiency
    Van der Ploeg, Catharina P. B.
    Blom, Maartje
    Bredius, Robbert G. M.
    van der Burg, Mirjam
    Schielen, Peter C. J., I
    Verkerk, Paul H.
    Van den Akker-van Marle, M. Elske
    [J]. EUROPEAN JOURNAL OF PEDIATRICS, 2019, 178 (05) : 721 - 729
  • [2] Cost-effectiveness of newborn screening for severe combined immunodeficiency
    Catharina P. B. Van der Ploeg
    Maartje Blom
    Robbert G. M. Bredius
    Mirjam van der Burg
    Peter C. J. I. Schielen
    Paul H. Verkerk
    M. Elske Van den Akker-van Marle
    [J]. European Journal of Pediatrics, 2019, 178 : 721 - 729
  • [3] Cost-effectiveness of newborn screening for severe combined immunodeficiency.
    McGhee, SA
    McCabe, ERB
    Stiehm, ER
    [J]. JOURNAL OF INVESTIGATIVE MEDICINE, 2004, 52 (01) : S127 - S127
  • [4] THE COST-EFFECTIVENESS OF SCREENING FOR SEVERE COMBINED IMMUNODEFICIENCY (SCID) IN THE UK NHS NEWBORN BLOODSPOT SCREENING PROGRAMME
    Bessey, A.
    Leaviss, J.
    de la Cruz, Galvan C.
    Chilcott, J.
    Wong, R.
    [J]. VALUE IN HEALTH, 2017, 20 (09) : A501 - A501
  • [5] SYSTEMATIC REVIEW OF THE COST EFFECTIVENESS OF NEWBORN SCREENING FOR SEVERE COMBINED IMMUNODEFICIENCY
    Maher, A.
    Comber, L.
    Finnegan, E.
    Byrne, D.
    Carrigan, M.
    O'Neill, M.
    Harrington, P.
    Spillane, S.
    Ryan, M.
    [J]. VALUE IN HEALTH, 2023, 26 (12) : S158 - S158
  • [6] Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency
    Shih, Sophy T. F.
    Keller, Elena
    Wiley, Veronica
    Farrar, Michelle A.
    Wong, Melanie
    Chambers, Georgina M.
    [J]. INTERNATIONAL JOURNAL OF NEONATAL SCREENING, 2022, 8 (03)
  • [7] A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID)
    Chan, Kee
    Davis, Joie
    Pai, Sung-Yun
    Bonilla, Francisco A.
    Puck, Jennifer M.
    Apkon, Michael
    [J]. MOLECULAR GENETICS AND METABOLISM, 2011, 104 (03) : 383 - 389
  • [8] Cost-Effectiveness/Cost-Benefit Analysis of Newborn Screening for Severe Combined Immune Deficiency in Washington State
    Ding, Yao
    Thompson, John D.
    Kobrynski, Lisa
    Ojodu, Jelili
    Zarbalian, Guisou
    Grosse, Scott D.
    [J]. JOURNAL OF PEDIATRICS, 2016, 172 : 127 - 135
  • [9] THE COST-EFFECTIVENESS OF NEWBORN SCREENING FOR METACHROMATIC LEUKODYSTROPHY (MLD) IN THE UK
    Bean, K.
    Jones, S.
    Chakrapani, A.
    Vijay, S.
    Wu, T. H. Y.
    Church, H. J.
    Pang, F.
    [J]. VALUE IN HEALTH, 2022, 25 (01) : S79 - S79
  • [10] Newborn Screening for Severe Combined Immunodeficiency
    Taki, Mohammed
    Miah, Tayaba
    Secord, Elizabeth
    [J]. IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA, 2021, 41 (04) : 543 - 553