Hemophilia A: Patient Impact and Economic Burden of the Disease

Hemophilia A is a rare but serious bleeding disorder caused by a deficiency in blood clotting factor VIII (FVIII). To examine the impact of hemophilia A and replacement FVIII therapies from a humanistic and economic perspective, a targeted literature review was conducted.

Searches were conducted in MEDLINE^® and the National Health Service Economic Evaluation Database, using combinations of disease, patient-reported outcome and economic-related key words, limited to articles published in English between January 2000 and January 2010 (inclusive). A total of 34 full-text articles (from 653 abstracts retrieved) were selected for detailed consideration.

Findings revealed increased mortality rates and decreased life expectancy among people with hemophilia A, compared with the general population. This is largely a result of the transmission of blood-borne viruses (e.g. HIV and Hepatitis-C) due to use of plasma-derived FVIII (pdFVIII) concentrates. Improvements in viral attenuation processes plus donor screening practices for pdFVIII products have reduced the risks of viral transmission, but risk of transmission still exists in relation to non-enveloped viruses and other unknown pathogens. Newly developed recombinant FVIII therapies minimizes these risks; however, such therapies are not currently widely available globally. All available FVIII therapies use demanding regimens requiring regular time-consuming injections, which can be detrimental to patients’ health-related quality of life and limit adherence to replacement FVIII therapy. Non-adherence to factor replacement therapy is associated with diminished product efficacy, poorer health outcomes and increasing economic expenditure. Treating hemophilia A is costly, primarily due to the high acquisition cost of replacement FVIII products. However, indirect costs related to patient disability and level of care also contribute to economic burden. Prophylactic treatment initially may appear to be more expensive than on-demand treatment but is associated with greater clinical efficacy and improved long-term outcomes, which may lead to cost savings over the course of a patient's lifetime.

Hemophilia A is associated with considerable humanistic and economic burden. There remain substantial unmet needs with regard to the safety, convenience, global access to treatment incl. reliable product supply and total care costs associated with factor replacement therapies.

Strandberg-Larsen:Novo Nordisk A/S: Employment. Gater:Novo Nordisk A/S: Research Funding. Marlow:Novo Nordisk A/S: Research Funding. Thomson:Novo Nordisk A/S: Research Funding.