Abstract
Hemophilia is a set of lifelong bleeding disorders linked to the X chromosome. Standard treatment for patients with severe hemophilia is intensive replacement therapy with intravenous injection of coagulation factor concentrates to prevent spontaneous recurrent joint bleed. In the 1980s, many hemophilia patients were infected with HIV and/or hepatitis C transmitted by plasma derived coagulation factor concentrates. In the future, new pathogens could appear and a risk remains that the current manufacturing methods will not be able to eliminate those. The aim of the study is to estimate the value of eliminating the risk of being infected by blood-borne diseases in the treatment of hemophilia which could provide decision-makers with information on how much resource to spend on this purpose to be in line with societal preferences. Individual preferences for safety were elicited from a sample of 821 individuals from the Swedish general population using a web-based questionnaire. The preferences were estimated using the “chained approach” which combines the contingent valuation (CV) and standard gamble (SG) methods. The respondents were asked (1) to state their willingness-to-pay to avoid a temporary, non-fatal injury presented in a health state derived from the EQ-5D instrument, and (2) to choose between living with this injury with certainty or receive a treatment that could restore the respondent to full health immediately, but which entails a risk of being infected by a fatal, blood-borne disease. A value of a statistical life (VSL) in the context of blood-borne diseases of SEK47 million was calculated by “chaining” the answers to the CV question and SG question, and from the VSL we derived a value of a QALY in the context of blood-borne diseases of SEK2.8 million. Using the current mean annual dose of factor concentrate of 268,000 international units (IU) per patient in Sweden, the value of eliminating the risk of being infected by blood-borne diseases would be SEK1.80 per IU corresponding to a price premium of around 45 %.Published: Online April 2016. In print August 2016.
Highlights
Medical treatment of disease may entail risks and rigorous safety reporting for medicines are standard since decades
Hemophilia is included among the rare diseases (Mannucci and Tuddenham, 2001) with a prevalence of 1 in 10,000 in the general population.(Berntorp and Shapiro, 2012) The replacement treatment with intravenous infusions of the deficient clotting factor introduced in hemophilia care in the 1960s has drastically increased the life expectancy which approaches that of the general population. (Darby et al, 2007, Lovdahl et al, 2013) Besides the risk of life threatening bleeds, repeated bleeding episodes in joints lead to crippling hemophilic arthropathy
In the 1980s, many people with hemophilia were infected with human immunodeficiency virus (HIV), acquired immunodeficiency syndrome (AIDS) and/or hepatitis C (HCV), all of which were acquired through infected plasma-derived factor concentrates.(Darby et al, 2007, Evatt, 2006, Soucie et al, 1998) HIV infection occurred in up to 70% of people with severe factor VIII deficiency in many countries and in 30% to 40% of the hemophilia population as a whole
Summary
Medical treatment of disease may entail risks and rigorous safety reporting for medicines are standard since decades. For diseases where medicines are manufactured from human blood, the risk of virus transmission is a central concern. In the 1980s, many people with hemophilia were infected with human immunodeficiency virus (HIV), acquired immunodeficiency syndrome (AIDS) and/or hepatitis C (HCV), all of which were acquired through infected plasma-derived factor concentrates.(Darby et al, 2007, Evatt, 2006, Soucie et al, 1998) HIV infection occurred in up to 70% of people with severe factor VIII deficiency in many countries and in 30% to 40% of the hemophilia population as a whole. Prior to the HIV and HCV outbreaks, life expectancy amongst the hemophilia community in developed countries had been increased from 16 years at the start of the 20th century, to 60 years in 1980.(Arnold et al, 2006, Darby et al, 2007, The Swedish Haemophilia Society, 2003, O'mahony, 1999) by 1994 it had fallen to 40 years, almost entirely as a result of AIDS.(Chorba et al, 1994)
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