Patients with phenylketonuria (PKU) and their families feel burdened by the high cost and preparation time required to treat the condition, a survey has found.
The results of the survey were described in the study “The financial and time burden associated with phenylketonuria treatment in the United States,” which was published in the journal Molecular Genetics and Metabolism Reports.
Phenylketonuria (PKU) is an inherited disorder in which the body is unable to metabolize the amino acid phenylalanine. As a result, people with PKU require a lifetime of treatment, including a strict low phenylalanine (low protein) diet, daily medications such as Kuvan, and, in some cases, regular monitoring of amino acids in the blood.
Living with PKU can impose a substantial burden on patients and their families, and little is known about the cost in terms of time and money for patients and their caregivers.
To quantify this burden, a team of researchers based at the University of Michigan and collaborators conducted a survey of people living with PKU or parents of children with PKU to identify out-of-pocket expenses and the use of resources for dietary and medical treatments.
A total of 114 participants (47 children and 67 adults), found through the Michigan’s Newborn Screening Program, responded and returned the 60-minute survey, which had been mailed to them in October and November 2014. Of the respondents, 32 children and 52 adults were PKU patients.
The survey collected information on the number and type of medical visits per year, travel time, total time of medical visits, and out-of-pocket expenses for each visit, such as co-pays and parking.
Information was also collected on the use of medical formula, low phenylalanine foods, and Kuvan therapy, and the time costs for shopping and preparing the special PKU diet.
Additionally, the team gathered information from insurance claims in databases containing data from U.S. private health plans provided by employers. This information was analyzed separately for 391 children up to 11 years old, 137 individuals from 12 to 17 years old, and 441 adults older than 18.
The survey found that respondents who used medical foods for children reported an average annual cost of the diet was $4,418 for formula and $1,961 for low-protein foods, for a total of about $6,400 per year.
Of the adults who reported costs, the annual cost of the formula was $7,753 and low-protein foods was $1,274, for an overall total of about $9,000.
Parents of PKU children spent more than 300 hours in the last year shopping for and preparing special diet foods for their affected children, while adults reported spending approximately 310 hours.
For metabolic care visits, parents reported an average of 1.5 visits in the past year, with a round-trip travel time (including wait times) of 5.9 hours on average, and a total out-of-pocket cost of $499. Parents reported missing 3.4 days of work per year and 23% of those parents quit working due to their child’s PKU.
Adults reported similar results with 1.5 metabolic care visits per year, a total travel time of six hours per visit, and out-of-pocket costs of $588.
The total annual cost of Kuvan for children was $88,855 and $191,382 for adults, although the out-of-pocket cost was $302 for children and $25 for adults.
The highest cost for medical expenses based on insurance claims data was $54,147 for children 12 to 17, while the cost was $19,057 for children up to 11 years old, and $40,705 for adults.
Overall, medications were the largest driver of medical costs, with Kuvan as the biggest contributor, accounting for 85% of child medication expenses and 92% for adults.
In addition, more than 85% of parents and 79% of adults found it difficult to follow the recommended diet due to the cost and burden of the diet, and the emotional and social awkwardness of packing meals for social gatherings.
“To the best of our knowledge, this study represents the first US estimates of the financial and time burden of PKU on affected individuals and their families,” the authors wrote.
“These estimates can be used as inputs into cost-effectiveness analyses of the value of PKU treatment or newborn screening for PKU and also inform persons with PKU and their families about aspects of treatment and care for planning purposes.”
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