Marie Caroline Clément1, Nizar Mahlaoui2, Cécile Mignot3, Christine Le Bihan4, Hasina Rabetrano1, Ly Hoang1, Bénédicte Neven5, Despina Moshous5, Marina Cavazzana6, Stéphane Blanche5, Alain Fischer7, Marie Audrain8, Isabelle Durand-Zaleski9. 1. URC Eco (Clinical Research Unit in Health Economics), Assistance Publique-Hôpitaux de Paris, Hôtel Dieu Hospital, Paris, France. 2. Pediatric Hematology-Immunology and Rheumatology Unit, Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; CEREDIH (French National Reference Center for Primary Immune Deficiencies), Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; Laboratory of Human Genetics of Infectious Diseases, Necker Branch, Inserm U1163, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Imagine Institute, Paris, France. 3. CEREDIH (French National Reference Center for Primary Immune Deficiencies), Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France. 4. Medical information unit, Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France. 5. Pediatric Hematology-Immunology and Rheumatology Unit, Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; CEREDIH (French National Reference Center for Primary Immune Deficiencies), Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Imagine Institute, Paris, France. 6. Paris Descartes University, Sorbonne Paris Cité, Imagine Institute, Paris, France; Biotherapy Department, Necker Children's University Hospital, AP-HP, Paris, France; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, Paris, France. 7. Pediatric Hematology-Immunology and Rheumatology Unit, Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; CEREDIH (French National Reference Center for Primary Immune Deficiencies), Assistance Publique-Hôpitaux de Paris, Necker-Enfants Malades University Hospital, Paris, France; Paris Descartes University, Sorbonne Paris Cité, Imagine Institute, Paris, France; Collège de France, Paris, France. 8. Immunology Laboratory, Nantes University Hospital, Nantes, France. 9. URC Eco (Clinical Research Unit in Health Economics), Assistance Publique-Hôpitaux de Paris, Hôtel Dieu Hospital, Paris, France; Public Health Unit, Henri Mondor Hospital, Assistance Publique-Hôpitaux de Paris, INSERM UMR 1123, Créteil, France. Electronic address: isabelle.durand-zaleski@hmn.aphp.fr.
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.
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.
Keywords:
Primary immunodeficiency; cost-effectiveness; health economics; health policy; newborn screening; severe T-cell lymphopenia; severe combined immunodeficiency
Authors: Francisco J Espinosa-Rosales; Antonio Condino-Neto; José L Franco; Ricardo U Sorensen Journal: J Clin Immunol Date: 2016-04-06 Impact factor: 8.317
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