Household financial burden of phenylketonuria and its impact on treatment in China: a cross‐sectional study. Issue 3 (10th November 2016)

• Record Type: Journal Article
• Authors: Wang, Lin; Zou, Hui; Ye, Fang; Wang, Kundi; Li, Xiaowen; Chen, Zhihua; Chen, Jie; Han, Bingjuan; Yu, Weimin; He, Chun; Shen, Ming
• Is Part Of: Journal of inherited metabolic disease. Volume 40:Issue 3(2017)
• Page Start: 369
• Page End: 376
• Publication Date: 2016-11-10
• Subjects: Metabolism, Inborn errors of -- Periodicals; Metabolism -- Disorders -- Periodicals; 616.39042
• DOI: 10.1007/s10545-016-9995-0
• Languages: English
• ISSNs: 0141-8955

Abstract

Summary: Background: Phenylketonuria (PKU) is a rare inborn disease, which, untreated, leading to severe neurobehavioral dysfunction. Considering its complexity, the management of PKU may bring a formidable economic burden to parents and caregivers. It is still unknown what the out‐of‐pocket expenses are for a patient with PKU in China. This paper explores the household financial burden of classical PKU and its impact on Chinese families in a quantitative manner for the first time. Methods: A non‐interventional and observational study was conducted at the China‐Japan Friendship Hospital, one of the national centers for inherited metabolic disorders in China. The medical and non‐medical household financial burdens were consolidated into a questionnaire to evaluate the out‐of‐pocket costs (OOPCs) of PKU treatment and follow‐up. Findings: The total OOPCs were USD$3766.1 (0y), USD$3795.2 (1–2 ys), USD$4657.7 (3–4 ys), USD$5979.9 (5–8 ys), and USD$5588.7 (9 ys and older) for PKU patients of different age groups. The median economic burden of classical PKU was 75.0 % of total annual family income (range 1.0–779.1 %), and 94.4 % of the families exceeding the threshold considered as catastrophic expenditure. There was a negative correlation between the financial burden and the proportion of time when Phe concentrations were in the desired target range (120–250 μmol/L) in 0–4‐ys group ( r = ‐0.474, p = 0.026). Conclusions: The management of PKU is associated with a severe financial burden on patients' families, which may lead to insufficient treatment or variation of blood Phe concentration. The current reimbursement policies are as yet inadequate. A national reimbursement system targeting treatment practices for PKU patients and other rare diseases across China is imperative.