title: Haemophilia prophylaxis: how can we justify the costs?
abstract: 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.
authors.affiliation: Division of Rheumatology, The Hospital for Sick Children, Department of Pediatrics, Institute of Health Policy Management & Evaluation, and the Dalla Lana School of Public Health, University of Toronto, Toronto, Canada. brian.feldman@sickkids.ca
authors.forename: B M
authors.initials: BM
authors.lastname: Feldman
authors.forename: K
authors.initials: K
authors.lastname: Berger
authors.forename: R
authors.initials: R
authors.lastname: Bohn
authors.forename: M
authors.initials: M
authors.lastname: Carcao
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.forename: A
authors.initials: A
authors.lastname: Gringeri
authors.forename: K
authors.initials: K
authors.lastname: Hoots
authors.forename: L
authors.initials: L
authors.lastname: Mantovani
authors.forename: A R
authors.initials: AR
authors.lastname: Willan
authors.forename: W
authors.initials: W
authors.lastname: Schramm
mesh_terms: D001779:Blood Coagulation Factors / Q000191:economics* / Q000627:therapeutic use*; D016527:Drug Costs; D006467:Hemophilia A / Q000191:economics* / Q000628:therapy*; D006470:Hemorrhage / Q000191:economics / Q000517:prevention & control; D006801:Humans; D008297:Male; D011315:Preventive Medicine / Q000191:economics
chemical_list: D001779:Blood Coagulation Factors