Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres

Nerich, Virginie; Tissot, Edgar; Faradji, A.; Demesmay, K.; Bertrand, Marie Anne; Lorenzini, J. L.; Briquel, Marie‐Élisabeth; Pouzol, P; Woronoff‐Lemsi, Marie‐Christine

doi:10.1007/s11096-007-9181-4

Cited by 23 publications

(24 citation statements)

References 14 publications

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“…Comparing mean and median costs, it is evident some of the high cost cases cause the mean cost to be significantly higher than the cost for a typical patient (77,78,82).…”

Section: Schramm Et Al Examined Costs Incurred By Patients In Both Pmentioning

confidence: 99%

“…The distribution of costs over different patients tends to be skewed with either a small proportion of patients accounting for a large proportion of resource utilisation (82,95,96) or particular subgroups of patients incurring higher costs. For example, haemophilia patients with HIV were four times as expensive to treat as HIV negative (85), and seropositive polyarthritis was five times more expensive to treat than persistent oligoarthritis both in total and direct cost estimates for JIA patients (96).…”

Section: Evidence and Policy Implicationsmentioning

confidence: 99%

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Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

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A c c e p t e d M a n u s c r i p t 2 Highlights  We systematically reviewed the socioeconomic cost literature for 10 rare diseases. Direct and indirect costs incurred by patients, carers and society were searched. 77 studies were included with an unequal distribution of studies over disease types. Level of existing evidence is highest for diseases with available drug treatments. Indirect costs are in most cases of similar or higher magnitude than direct costs. found to range between 1-2 per 100,000 population (PWS, a sub-type of Histiocytosis, and EB) up to 42 per 100,000 population (Scleroderma). Overall, cost evidence on rare diseases appears to be very scarce (a total of 77 studies were identified across all diseases), with CF (n=29) and Haemophilia (n=22) being relatively well studied, compared to the other conditions, where very limited cost of illness information was available. In terms of data availability, total lifetime cost figures were found only across four diseases, and total annual costs (including indirect costs) across five diseases.Overall, data availability was found to correlate with the existence of a pharmaceutical treatment and indirect costs tended to account for a significant proportion of total costs.Although methodological variations prevent any detailed comparison between conditions, most of the rare diseases examined are associated with significant economic burden, both direct and indirect.

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“…Comparing mean and median costs, it is evident some of the high cost cases cause the mean cost to be significantly higher than the cost for a typical patient (77,78,82).…”

Section: Schramm Et Al Examined Costs Incurred By Patients In Both Pmentioning

confidence: 99%

Section: Evidence and Policy Implicationsmentioning

confidence: 99%

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

2015

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“…The low consumption of clotting factor concentrate in these countries [6] arises from the economic constraints of accessing this high-cost treatment product. In developed countries, hospital records provide accurate treatment data and can be used for studies on haemophilia economics [7][8][9][10]. These data are important as they describe the impact of lack of subsidized services on treatment of patients.…”

Section: Introductionmentioning

confidence: 99%

Out‐of‐pocket and catastrophic expenditure on treatment of haemophilia by Indian families

et al. 2013

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In low-income countries, haemophilia treatment is not supported by national health services. Data on the burden of out-of-pocket (OOP) expenditure on households are unavailable from these countries. This study measured the OOP expenditure on treatment of haemophilia by Indian households. We used 20 weeks of follow-up data of 24 haemophilia A patients to estimate the annual bleeding rate for each patient and the actual OOP expenditure on treatment. We used this observational data to calculate the annual OOP expenditure on treatment if all bleeding episodes were to be treated with clotting factor concentrate. Using previously published methodology, we estimated if the expenditure was catastrophic to households or not. The observed monthly expenditure on treatment ranged from 1.5% to 12% of monthly income as not all bleeding episodes were treated with clotting factor concentrate. The estimated monthly expenditure if all bleeding episodes occurring over 1 year were to be treated would range from 21 to 314 times the monthly income of families. Nearly 68% of households would have experienced catastrophic expenditure. Treatment for haemophilia results in significant OOP expenditure for households, which is avoided by not providing standard treatment to patients. There is a need to mobilize prevention and care services for haemophilia in India and other low-income countries to mitigate the suffering due to lack of affordable treatment.

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“…The number is even higher in individuals with inhibitors treated with immune tolerance therapy. [20][21][22] In addition to direct health care costs, studies have also shown considerable indirect costs related to hemophilia. 18 These indirect costs include individuals' and caregivers' lost productivity, caregivers' unpaid time costs, and hemophilic individuals' disability.…”

Section: Cost Of Care In Hemophiliamentioning

confidence: 99%

Costs of Care in Hemophilia and Possible Implications of Health Care Reform

Johnson

Zhou

2011

Hematology

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Economic evaluation in health care is increasingly used to assist policy makers in their difficult task of allocating limited resources. The high cost of care, including that for clotting factor concentrates, makes hemophilia a potential target for cost-cutting efforts by health care payers. Although the appropriate management of hemophilia is key to minimizing and preventing long-term morbidity, comparative effectiveness studies regarding the relative benefit of different treatment options are lacking. Cost-of-illness (COI) analysis, which includes direct and indirect costs from a societal perspective, can provide information to be used in cost-effectiveness and other economic analyses. Quality-of-life assessment provides another methodology with which to measure outcomes and benefits of appropriate disease management. Health care reform has implications for individuals with hemophilia and their families through changes in payment, insurance coverage expansion, and health care delivery system changes that reward quality and stimulate cooperative, team-based care. Providers will benefit from the expansion of insurance coverage and some financial benefits in rural areas, and from the expansion of coverage for preventive services. Accountable care organizations will potentially change the way providers are paid and financial incentives under reform will reward high quality of care.

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Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres

Cited by 23 publications

References 14 publications

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

Out‐of‐pocket and catastrophic expenditure on treatment of haemophilia by Indian families

Costs of Care in Hemophilia and Possible Implications of Health Care Reform

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