Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data - 05/06/15
Abstract |
Background |
The inclusion of severe combined immunodeficiency (SCID) in a Europe-wide screening program is currently debated.
Objective |
In making a case for inclusion in the French newborn screening program, we explored the costs incurred and potentially saved by early management of SCID.
Methods |
For test costs, a microcosting study documented the resources used in a laboratory piloting a newborn screening test on Guthrie cards using the T-cell receptor excision circle quantification method. For treatment costs, patients with SCID admitted to the national reference center for primary immunodeficiency in France between 2006 and 2010 were included. Costs of admission were estimated from actual national production costs. We estimated the costs for patients who underwent early versus delayed hematopoietic stem cell transplantation (HSCT; age, ≤3 vs >3 months, respectively).
Results |
The unit cost of the test varied between €4.69 and €6.79 for 33,800 samples per year, depending on equipment use and saturation. Of the 30 patients included, 27 underwent HSCT after age 3 months. At 1 year after HSCT, 10 of these had died, and all 3 patients undergoing early transplantation survived. The medical costs for HSCT after 3 months were €195,776 (interquartile range, €165,884-€257,160) versus €86,179 (range, €59,014-€272,577) when performed before 3 months of age. In patients undergoing late transplantation, active infection contributed to high cost and poor outcome.
Conclusion |
Early detection of SCID could reduce the cost of treatment by €50,000-100,000 per case. Assuming a €5 unit cost per test, the incidence required to break even is 1:20,000; however, if the survival advantage of HSCT before 3 months is confirmed, universal screening is likely to be cost-effective.
Le texte complet de cet article est disponible en PDF.Key words : Primary immunodeficiency, severe combined immunodeficiency, severe T-cell lymphopenia, newborn screening, health economics, cost-effectiveness, health policy
Abbreviations used : CEREDIH, HSCT, NBS, PID, SCID, TCL, TREC
Plan
| The study was financed in part by Nantes and AP-HP Paris University hospitals and by an unrestricted grant from Baxter Biosciences to the Laboratory of Immunology of Nantes University Hospital for the estimation of the screening cost. CEREDIH is supported by the French Association of Patients with Primary Immunodeficiencies (IRIS) and is funded by the French Ministry of Health. It additionally received unrestricted educational grants from LFB, CSL Behring, Baxter Biosciences, GlaxoSmithKline, Pfizer, Octapharma, and Orphan-Europe. This study received support from the French Association of Patients with PID (IRIS), the International Patient Organisation for Primary Immunodeficiencies (IPOPI), and the head of the Social Worker Unit of Necker-Enfants Malades University Hospital (Mrs C. Maurey-Forquy). |
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| Disclosure of potential conflict of interest: I. Durand-Zaleski's institution has received funding from the French government. The rest of the authors declare that they have no relevant conflicts of interest. |
Vol 135 - N° 6
P. 1589-1593 - juin 2015 Retour au numéro
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