Fellows & Alumni

Orphan drugs are those used in the treatment of rare diseases, and are often very expensive, with costs exceeding $600,000 per year for one patient. This high cost is related to several factors including recouping the manufacturer’s research and development costs, the limited number of patients with the disease and lack of competition within the market. The potential financial burden and opportunity cost of treating patients with rare diseases is significant and the available treatments and disease prevalence are expanding rapidly.  Currently there is not a framework or priority setting decision-making model in place in order to address the growing demand for therapeutics used to treat rare diseases. A key component of developing a policy framework for orphan drugs is cost-effectiveness. Given the costs of drugs for rare diseases and the often marginal effectiveness, treatments for rare diseases often do not meet the current thresholds, and do not incorporate the potential societal altruism for the treatment of those with a rare disease. The aim of our research is to determine the societal value for the treatment of patients with rare diseases, given the opportunity costs.  Through a media-supported public forum, we will capture and analyze society’s preferences for paying for drugs for rare diseases. This project will evaluate what society considers an appropriate cost of treatment of individual patients with rare diseases, and allow us to apply this information to future funding and policy decisions.