The direct and indirect annual costs of care for Friedreich’s ataxia (FA) can be as high as £11,000 to £19,000 per person, according to a study titled “Impact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany,” published in BioMed Central’s open access journal Orphanet Journal of Rare Diseases.
Although a rare disease, FA is the most frequent of all inherited ataxias, which affect muscle control. FA, which results from mutations in the gene encoding the protein frataxin, usually begins in childhood, with patients needing continual monitoring by specialists, nurses, and caregivers of developing symptoms.
“The costs of more common progressive diseases such as Parkinson’s Disease are known, but it is not really possible to base budget allocations of one disease on the expectations of another without understanding the differences in need,” Dr. Paola Giunti, a study author from University College London’s Institute of Neurology and University College London Hospitals, said in a news release. “Our results show that the needs of people with Friedreich’s Ataxia are different, with different costs, … and that a ‘one size fits all’ strategy is unlikely to provide the best care to people with rare diseases.”
Importantly, indirect medical costs of treating FA, such as home modifications and loss of earnings, should also be considered.
Cross-sectional observational studies were preformed in the U.K. (75 patients) and Germany (28 patients) to assess the FA burden on patients and healthcare systems. Analysis of yearly direct and indirect resource use was conducted for both countries, although costs were calculated only for the U.K.
Results showed that the cost is significant, and spread over social and healthcare systems, society, caregivers and the patients themselves. The annual per-person cost of healthcare, including medications, was £3,230 (about $4,570) in the U.K. When non-healthcare resources were considered, such as education and caregiver support, the figure rose to £7,537 (about $10,670). If indirect costs such as work loss, mobility aids, and living circumstances were also included, annual costs jumped to between £11,818 and £18,774 (about $16,740 to $26,590). The benefits of improved treatment options would be important from a social perspective, the study said, as the need for caregiver support could be reduced if patients were capable of participating in the workforce and needed few assistive modifications to go about their lives.
The authors concluded that, given the disease’s duration (estimated at 40–50 years), management strategies should seek to minimize FA’s impact on patients and caregivers, while maximizing quality of life.