Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data
Background: Phenylketonuria (PKU) is an inherited metabolic disease. If left untreated, it can lead to severe irreversible intellectual disability and can cause seizures, behavior disturbance, and white matter disease. This study aimed at evaluating the health economic impact of patients with PKU in...
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Elsevier
2024-12-01
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| Series: | Molecular Genetics and Metabolism Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426924000879 |
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| author | Jean-Baptiste Arnoux Claire Douillard Francois Maillot Stéphane Bouée Christian Jacob Kim Maren Schneider Julia Theil Sybil Charrière |
| author_facet | Jean-Baptiste Arnoux Claire Douillard Francois Maillot Stéphane Bouée Christian Jacob Kim Maren Schneider Julia Theil Sybil Charrière |
| author_sort | Jean-Baptiste Arnoux |
| collection | DOAJ |
| description | Background: Phenylketonuria (PKU) is an inherited metabolic disease. If left untreated, it can lead to severe irreversible intellectual disability and can cause seizures, behavior disturbance, and white matter disease. This study aimed at evaluating the health economic impact of patients with PKU in France. Methods: This retrospective observational study used health insurance claims data from the French SNDS (Système National des Données de Santé) database, which contains data from over 66 million French inhabitants. Patients with PKU were identified by ICD-10 diagnosis codes E70.0 (PKU) and E70.1 (Other hyperphenylalaninemia) documented as a chronic condition (affection de longue durée – ALD) or in the inpatient setting in the SNDS database between 2006 and 2018. Patients with PKU were matched to controls without PKU by age, sex, and region. Patients with early- and late-diagnosed PKU were defined as patients born after and before the implementation of nationwide newborn screening in France in 1972, respectively. Outcomes were analyzed for the year 2018. Results: Overall, 3549 patients with PKU were identified in the database on January 1st, 2018. Of those, 3158 patients versus 15,703 controls with at least one healthcare consumption in 2018 were available for outcome analyses. Patients with PKU had 7.7 times higher healthcare costs than non-PKU controls in 2018 (€11,144 versus 1456 mean costs; p < 0.0001). Pharmaceutical costs including dietary amino acid supplements were the cost driver and contributed 80.0% of the overall mean difference (MD) between patients with PKU and matched non-PKU controls. More than half (52.4%) of the mean pharmaceutical costs per patient with PKU was attributable to medical foods including dietary amino acid supplements.Of the 3158 patients with PKU, 2548 (80.7%) were classified as early-diagnosed and 610 (19.7%) as late-diagnosed. Increased healthcare costs, in comparison to non-PKU controls, were more evident in early-diagnosed patients (€11,263 versus €855 mean costs; 13.2-fold increase; p < 0.0001). For patients with late-diagnosed PKU, healthcare costs were 2.7-fold higher compared to matched non-PKU controls (€10,644 versus €3951 mean costs; p < 0.0001). Outpatient pharmaceutical costs accounted for 89.1% of the MD between early-diagnosed patients and controls. Among late-diagnosed patients, 55.5% of the MD were attributable to costs for inpatient care, followed by costs for outpatient care (23.9%) and outpatient pharmaceutical costs (20.6%). Conclusion: The results indicate that PKU is associated with substantially increased health care costs compared to non-PKU controls in France. The health economic impact was most evident in patients with early-diagnosed PKU due to increased outpatient pharmaceutical costs, especially for medical foods including dietary amino acid supplements. For late-diagnosed and by definition older patients with PKU, the excess costs compared with matched controls were mostly driven by costs for inpatient care. |
| format | Article |
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| institution | Kabale University |
| issn | 2214-4269 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Elsevier |
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| series | Molecular Genetics and Metabolism Reports |
| spelling | doaj-art-12fa43473b034ec3b0f8760465a67c5e2024-12-17T04:59:41ZengElsevierMolecular Genetics and Metabolism Reports2214-42692024-12-0141101134Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims dataJean-Baptiste Arnoux0Claire Douillard1Francois Maillot2Stéphane Bouée3Christian Jacob4Kim Maren Schneider5Julia Theil6Sybil Charrière7Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Necker-Enfants Malades, APHP, 149 rue de Sèvres 75015, Paris, France; Corresponding author.Service d'endocrinologie, diabétologie, métabolisme et nutrition, Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Huriez, CHU de Lille, 59037 Lille, FranceService de médecine interne, CHRU et Université de Tours, UMR INSERM 1253 « iBrain », Tours, FranceCEMKA, 43 boulevard du Maréchal Joffre, 92340 Bourg-La-Reine, FranceXcenda GmbH, Lange Laube 31, 30159 Hannover, GermanyXcenda GmbH, Lange Laube 31, 30159 Hannover, GermanyXcenda GmbH, Lange Laube 31, 30159 Hannover, GermanyHospices Civils de Lyon, Department of endocrinology, diabetologia, metabolic diseases and nutrition, Hôpital Louis Pradel, 69600 BRON, France; CarMen laboratory, INSERM, INRAE, Université Claude Bernard Lyon 1, 69310 Pierre Bénite, FranceBackground: Phenylketonuria (PKU) is an inherited metabolic disease. If left untreated, it can lead to severe irreversible intellectual disability and can cause seizures, behavior disturbance, and white matter disease. This study aimed at evaluating the health economic impact of patients with PKU in France. Methods: This retrospective observational study used health insurance claims data from the French SNDS (Système National des Données de Santé) database, which contains data from over 66 million French inhabitants. Patients with PKU were identified by ICD-10 diagnosis codes E70.0 (PKU) and E70.1 (Other hyperphenylalaninemia) documented as a chronic condition (affection de longue durée – ALD) or in the inpatient setting in the SNDS database between 2006 and 2018. Patients with PKU were matched to controls without PKU by age, sex, and region. Patients with early- and late-diagnosed PKU were defined as patients born after and before the implementation of nationwide newborn screening in France in 1972, respectively. Outcomes were analyzed for the year 2018. Results: Overall, 3549 patients with PKU were identified in the database on January 1st, 2018. Of those, 3158 patients versus 15,703 controls with at least one healthcare consumption in 2018 were available for outcome analyses. Patients with PKU had 7.7 times higher healthcare costs than non-PKU controls in 2018 (€11,144 versus 1456 mean costs; p < 0.0001). Pharmaceutical costs including dietary amino acid supplements were the cost driver and contributed 80.0% of the overall mean difference (MD) between patients with PKU and matched non-PKU controls. More than half (52.4%) of the mean pharmaceutical costs per patient with PKU was attributable to medical foods including dietary amino acid supplements.Of the 3158 patients with PKU, 2548 (80.7%) were classified as early-diagnosed and 610 (19.7%) as late-diagnosed. Increased healthcare costs, in comparison to non-PKU controls, were more evident in early-diagnosed patients (€11,263 versus €855 mean costs; 13.2-fold increase; p < 0.0001). For patients with late-diagnosed PKU, healthcare costs were 2.7-fold higher compared to matched non-PKU controls (€10,644 versus €3951 mean costs; p < 0.0001). Outpatient pharmaceutical costs accounted for 89.1% of the MD between early-diagnosed patients and controls. Among late-diagnosed patients, 55.5% of the MD were attributable to costs for inpatient care, followed by costs for outpatient care (23.9%) and outpatient pharmaceutical costs (20.6%). Conclusion: The results indicate that PKU is associated with substantially increased health care costs compared to non-PKU controls in France. The health economic impact was most evident in patients with early-diagnosed PKU due to increased outpatient pharmaceutical costs, especially for medical foods including dietary amino acid supplements. For late-diagnosed and by definition older patients with PKU, the excess costs compared with matched controls were mostly driven by costs for inpatient care.http://www.sciencedirect.com/science/article/pii/S2214426924000879Phenylketonuria (PKU)Healthcare costsHospitalizationsOutpatient careSNDS health insurance dataFrance |
| spellingShingle | Jean-Baptiste Arnoux Claire Douillard Francois Maillot Stéphane Bouée Christian Jacob Kim Maren Schneider Julia Theil Sybil Charrière Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data Molecular Genetics and Metabolism Reports Phenylketonuria (PKU) Healthcare costs Hospitalizations Outpatient care SNDS health insurance data France |
| title | Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data |
| title_full | Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data |
| title_fullStr | Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data |
| title_full_unstemmed | Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data |
| title_short | Health economic impact of patients with phenylketonuria (PKU) in France – A nationwide study of health insurance claims data |
| title_sort | health economic impact of patients with phenylketonuria pku in france a nationwide study of health insurance claims data |
| topic | Phenylketonuria (PKU) Healthcare costs Hospitalizations Outpatient care SNDS health insurance data France |
| url | http://www.sciencedirect.com/science/article/pii/S2214426924000879 |
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