Impact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany

Giunti, Paola; Greenfield, Julia; Stevenson, Alison J; Parkinson, Michael; Hartmann, Jodie L; Sandtmann, R; Piercy, James; O’Hara, Jamie; Casas, Leo Ruiz; Smith, Fiona

doi:10.1186/1750-1172-8-38

Cited by 15 publications

(18 citation statements)

References 12 publications

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“…The top 3 drivers of health care cost were similar for ALS. A prospective study that evaluated the annual cost related to Friedreich’s Ataxia, a neuromuscular disorder due to a genetic mutation in the mitochondrial protein frataxin, showed that hospitalizations contributed to 21% of total direct medical costs in the UK [ 20 ], which is consistent with our finding. In MS, prescription drug was the single biggest driver of health care cost, as evident by the number and cost of disease modifying treatments approved for MS.…”

Section: Discussionsupporting

confidence: 90%

A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008–2015

Cohen

Balcells²,

Hotchkiss

et al. 2018

Orphanet J Rare Dis

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BackgroundMitochondrial disease (MD) is a heterogeneous group of disorders characterized by impaired energy production caused by abnormal oxidative phosphorylation. Diagnosis of MD is challenging given the variability in how the disease can affect an individual’s neurologic, cardiovascular, ophthalmologic, or gastroenterological systems. This study describes the health care utilization and cost in patients diagnosed with MD.MethodsThis study was a retrospective claims analysis based on data from the Truven Health Analytics MarketScan Database and Milliman’s Consolidated Health Cost Guidelines Sources Database. For the purpose of this study the diagnosis of MD was defined by ICD-9-CM (prior to October 2015), and ICD-10-CM (October 2015 or later), and included patients identified between January 1, 2008 to December 31, 2015. ICD-9-CM code of 277.87 (disorders of mitochondrial metabolism) and the ICD-10-CM codes of E88.40, E88.41, E88.42 and E88.49 (mitochondrial metabolism disorders) were used as inclusive criteria. Patients were included if they had at least six months of exposure after the first MD-related claim occurrence, and either one MD claim in the inpatient setting OR two MD claims in an outpatient setting. Claims of MD patients are compared to those of a general insured total member population, as well as to those from multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) patients.ResultsDuring the study period, 3825 patients between the ages of 0 and 15 (pediatric) and 4358 patients 16 years of age and greater (adult) were identified. Total allowed per member per month (PMPM) cost for pediatric patients was $4829 and $3100 for adults, compared with an average of $202 and $486, respectively, for the total member population. The greatest drivers of costs based on allowed claims came from inpatient, surgery, and prescription medications. In the adult population, MD imposes a PMPM cost burden that was comparable to that observed for multiple sclerosis ($3518) and ALS ($3460) patients.ConclusionsThis retrospective claim study highlights the significant differences in the cost of medical care for MD patients compared to those of a general population. Mitochondrial disorders are associated with multisystem disease manifestations and a greater care and cost burden similar to other devastating neuromuscular diseases.

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Section: Discussionsupporting

confidence: 90%

A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008–2015

Cohen

Balcells²,

Hotchkiss

et al. 2018

Orphanet J Rare Dis

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“…The private and public funding ratios were compared for persons with FRDA and the general population to identify any discrepancies. This study came second to a study on healthcare resource utilization of FRDA patients in the United Kingdom and Germany (Giunti et al, 2013). …”

Section: Objectivesmentioning

confidence: 89%

Burden of Friedreich’s Ataxia to the Patients and Healthcare Systems in the United States and Canada

Polek¹,

Roach²,

Andrews³

et al. 2013

Front. Pharmacol.

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Objective: The study intended to substantiate healthcare resource utilization, costs, and funding patterns of US and Canadian Friedreich’s Ataxia (FRDA) populations, to assess compliance with treatment guidance and to identify areas where novel healthcare measures or improved access to existing care may improve patients’ functional and social capabilities and reduce the financial impact on the healthcare systems.Methods: Healthcare resource utilization and costs were collected in a cross-sectional study in the US (N = 197) and Canada (N = 43) and analyzed across severity of disease categories. Descriptive statistics, correlation analysis, and hypothesis testing were applied.Results: In the US, healthcare costs of FRDA patients were higher than those of “adults with two and more chronic conditions.” Significantly higher costs were incurred in advanced stages of the disease, with paid homecare being the main driver. This pattern was also observed in Canada. Compliance with the recommended annual neurological and cardiological follow-up was high, but was low for the recommended regular speech therapy. In the US public and private funding ratios were similar for the FRDA and the general populations. In Canada the private funding ratio for FRDA was higher than average.Conclusion: The variety of healthcare measures addressing the broad range of symptoms of FRDA, and the increasing use of paid home care as disease progresses made total US healthcare costs of FRDA exceed the costs of US adults with two and more chronic conditions. Therefore, measures delaying disease progression will allow patients to maintain their independence longer and may reduce costs to the healthcare system. Novel measures to address dysarthria and to ensure access to them should be further investigated. The higher than average private funding ratio in Canada was due to the relatively high cost of the pharmacological treatment of FRDA.

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“…This is in part because large samples are required to generate sufficient numbers of affected patients. One longitudinal prospective study from Europe 15 demonstrated significant annual costs related to Friedreich’s Ataxia (FA), a neuromuscular disorder that impacts mitochondrial function. They found that hospitalizations contributed to approximately 20% of total direct medical costs in the UK.…”

Section: Discussionmentioning

confidence: 99%

Hospitalizations for mitochondrial disease across the lifespan in the U.S.

McCormack

Xiao

Kilbaugh

et al. 2017

Molecular Genetics and Metabolism

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Importance Mitochondrial disease is being diagnosed with increasing frequency. Although children with mitochondrial disease often have severe, life-limiting illnesses, many survive into adulthood. There is, however, limited information about the impact of mitochondrial disease on healthcare utilization in the U.S. across the lifespan. Objectives To describe the characteristics of inpatient hospitalizations related to mitochondrial disease in the U.S., to identify patient-level clinical factors associated with in-hospital mortality, and to estimate the burden of hospitalizations on individual patients. Design Cross-sectional and longitudinal observational studies. Setting U.S. hospitals. Participants Individuals with hospital discharges included in the triennial Healthcare Cost and Utilization Project (HCUP) Kids Inpatient Database (KID) and the National Inpatient Sample (NIS) in 2012 (cross-sectional analysis); individuals with hospital discharges included in the HCUP California State Inpatient Database from 2007 2011, inclusive (longitudinal analysis). Exposure Hospital discharge associated with a diagnosis of mitochondrial disease. Main outcome measures Total number and rate of hospitalizations for individuals with mitochondrial disease (International Classification of Diseases, 9th revision, Clinical Modification code 277.87, disorder of mitochondrial metabolism); in-hospital mortality. Results In the 2012, there were approximately 3,200 inpatient pediatric hospitalizations (1.9 per 100,000 population) and 2,000 inpatient adult hospitalizations (0.8 per 100,000 population) for mitochondrial disease in the U.S., with associated direct medical costs of $113 million. In-hospital mortality rates were 2.4% for children and 3.0% for adults, far exceeding population averages. Higher socioeconomic status was associated with both having a diagnosis of mitochondrial disease and with higher in-hospital mortality. From 2007–2011 in California, 495 individuals had at least one admission with a diagnosis of mitochondrial disease. Patients had a median of 1.1 hospitalizations (IQI, 0.6 2.2) per calendar year of follow-up; infants under 2y were hospitalized more frequently than other age groups. Over up to five years of follow up, 9.9% of participants with any hospitalization for mitochondrial disease were noted to have an in-hospital death. Conclusions and Relevance Hospitalizations for pediatric and adult mitochondrial diseases are associated with serious illnesses, substantial costs, and significant patient time. Identification of opportunities to prevent or shorten such hospitalizations should be the focus of future studies.

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Impact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany

Cited by 15 publications

References 12 publications

A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008–2015

A retrospective analysis of health care utilization for patients with mitochondrial disease in the United States: 2008–2015

Burden of Friedreich’s Ataxia to the Patients and Healthcare Systems in the United States and Canada

Hospitalizations for mitochondrial disease across the lifespan in the U.S.

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