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

Clément, M.-C.; Mahlaoui, Nizar; Mignot, C.; Bihan-Benjamin, Christine Le; Rabetrano, Hasina; Hoang, Ly N.; Neven, Bénédicte; Moshous, Despina; Cavazzana, Marina; Blanche, Stéphane; Fischer, Alain; Audrain, Marie; Durand‐Zaleski, Isabelle

doi:10.1016/j.jaci.2015.02.004

Cited by 33 publications

(18 citation statements)

References 27 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…In France, a pilot testing program indicated a cost of $6-$9 per test. 51 These cost estimates are for laboratory-developed tests; if laboratories use a newly available Food and Drug Administration-approved kit, the cost per life-year saved will likely be higher.…”

Section: Discussionmentioning

confidence: 99%

“…Finally, the mean cost for hospital care received by 27 late-treated French infants from birth through 1 year posttransplant (including 10 who died posttransplant) was €226 510, with median costs for late-treated (≤3 months) and early-treated (<3 months) transplanted infants of €195 776 and €86 179, respectively (median costs are lower than mean costs). 51 Taking into account US healthcare prices, the US-equivalent average total treatment cost for late-treated infants might be as much as $500 000.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

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

Ding

Thompson

Kobrynski

et al. 2016

The Journal of Pediatrics

View full text Add to dashboard Cite

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.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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

Ding

Thompson

Kobrynski

et al. 2016

The Journal of Pediatrics

View full text Add to dashboard Cite

show abstract

“…Early HSCT has demonstrated to cure several of the most severe forms of PI. Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia using the T-cell receptor excision circle quantification method is available in very few countries around the world but has shown to reduce dramatically the cost of an HSCT [3].…”

mentioning

confidence: 99%

Into Action: Improving Access to Optimum Care for all Primary Immunodeficiency Patients

et al. 2016

View full text Add to dashboard Cite

“…2 Newborn screening of T cell deficiencies including severe combined immunodeficiency (SCID) is increasingly being implemented in the United States, and similar efforts are proposed internationally. Clement et al 3 reported an economic analysis of newborn screening for T cell defects in France. In addition to medical benefits of early detection to minimize the risk of fatal infections, the authors estimated a reduction of medical costs between 50,000 to 100,000 euros per patient identified and treated.…”

mentioning

confidence: 99%

Advances in clinical immunology in 2015

Chinen

Notarangelo

Shearer

2016

Journal of Allergy and Clinical Immunology

View full text Add to dashboard Cite

Advances in clinical immunology in the past year included the report of practice parameters for the diagnosis and management of primary immune deficiencies (PIDs) to guide the clinician in the approach to these relatively uncommon disorders. We have learned of new gene defects causing immunodeficiency and of new phenotypes expanding the spectrum of conditions caused by mutations found in one gene, such as a specific RTEL1 mutation causing isolated NK cell deficiency, and mutations in RAB27 resulting in immunodeficiency without albinism. Advances in diagnosis included the increasing use of whole exome sequencing to identify gene defects, and the measurement of serum free light chains to identify secondary hypogammaglobulinemias. For several PIDs, improved outcomes have been reported after definitive therapy with hematopoietic stem cell transplantation and gene therapy.

show abstract

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

Cited by 33 publications

References 27 publications

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

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

Into Action: Improving Access to Optimum Care for all Primary Immunodeficiency Patients

Advances in clinical immunology in 2015

Contact Info

Product

Resources

About