The price of care versus the cost of caring

Abstract

The high drug costs of enzyme replacement therapy (ERT) of lysosomal storage diseases (LSD), including Fabry disease, make the overall per-patient costs of treatment extremely high. According to conventional cost-utility analyses, ERT does not meet generally accepted criteria for public reimbursement. However, research has shown that public involvement in healthcare resource allocation invariably includes consideration of other societal concerns, including severity of the disease, the potential for a positive response to treatment, and the life-threatening nature of the disease. The formal incorporation of societal values in decisions relating to financial support for expensive treatments for rare diseases is illustrated by the establishment of the Citizen's Council of the National Institute for Health and Clinical Excellence (NICE) in the UK. The need for ERT for LSD is generally deemed to be life-long, and other, non-medical, costs ultimately play a role in decisions by patients to continue treatment indefinitely. © 2010 Springer Science+Business Media B.V.