Haemophilia prophylaxis: how can we justify the costs?

Severe haemophilia results in increased mortality and poorer quality of life. Factor prophylaxis leads to a more normal life, but is very costly; most of the cost is due to the high cost of replacement factor. Despite its high cost, factor prophylaxis has been adopted throughout the developed world...

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Bibliographic Details
Published in:Haemophilia : the official journal of the World Federation of Hemophilia 2012-09, Vol.18 (5), p.680-684
Main Authors: FELDMAN, B. M., BERGER, K., BOHN, R., CARCAO, M., FISCHER, K., GRINGERI, A., HOOTS, K., MANTOVANI, L., WILLAN, A. R., SCHRAMM, W.
Format: Article
Language:English
Subjects:
Blood Coagulation Factors - economics
Blood Coagulation Factors - therapeutic use
cost
Drug Costs
haemophilia
Hemophilia A - economics
Hemophilia A - therapy
Hemorrhage - economics
Hemorrhage - prevention & control
Humans
Male
Preventive Medicine - economics
prophylaxis
treatment
willingness to pay
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Summary:Severe haemophilia results in increased mortality and poorer quality of life. Factor prophylaxis leads to a more normal life, but is very costly; most of the cost is due to the high cost of replacement factor. Despite its high cost, factor prophylaxis has been adopted throughout the developed world – even in different health care systems. We argue that there are at least five possible reasons why societies may value factor prophylaxis despite its cost: (i) it is directed towards an inherited disease, (ii) the treatment is largely directed towards children, (iii) the disease is rare and the overall cost to society is small, (iv) the treatment is preventative, and v) the high cost is largely the result of providing safe products. In an era of rising health care costs, there is a strong research agenda to establish the factors that determine the value of expensive therapies for rare diseases like haemophilia.
ISSN:1351-8216
1365-2516
DOI:10.1111/j.1365-2516.2012.02790.x