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Household financial burden of phenylketonuria and its impact on treatment in China: a cross-sectional study
- Source :
- Journal of Inherited Metabolic Disease
- Publication Year :
- 2016
- Publisher :
- Springer Netherlands, 2016.
-
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. Electronic supplementary material The online version of this article (doi:10.1007/s10545-016-9995-0) contains supplementary material, which is available to authorized users.
- Subjects :
- Male
congenital, hereditary, and neonatal diseases and abnormalities
China
Phenylketonurias
Cross-sectional study
Disease
03 medical and health sciences
0302 clinical medicine
Rare Diseases
Cost of Illness
Japan
Surveys and Questionnaires
Genetics
Cost of illness
Medicine
Humans
Genetics(clinical)
030212 general & internal medicine
Genetics (clinical)
Finance
Newborn screening
Family Characteristics
business.industry
Family characteristics
nutritional and metabolic diseases
Middle Aged
Cross-Sectional Studies
Observational study
Original Article
Female
business
030217 neurology & neurosurgery
Subjects
Details
- Language :
- English
- ISSN :
- 15732665 and 01418955
- Volume :
- 40
- Issue :
- 3
- Database :
- OpenAIRE
- Journal :
- Journal of Inherited Metabolic Disease
- Accession number :
- edsair.doi.dedup.....c0718cc97d30d78d23bc6d6e764c9dc9