OBJECTIVES: The market for orphan drugs presents challenges in balancing incentives for innovation with accessibility for patients. We assessed availability and prices of United States (US) Food and Drug Administration (FDA) orphan-designated drugs approved for treatment of leukemia in the US and England. METHODS: Regulatory information for leukemia drugs with FDA orphan designation was collected from the FDA, the European Medicines Agency, and the Medicines and Healthcare Products Regulatory Agency. Generic and brand drug names, dosing, administration, and US pricing were collected from the Lexicomp online database. The National Institute for Health and Care Excellence database was then used to determine dosage and pricing for leukemia drugs in England. Prices were converted to cost per treatment or cost per year, as appropriate, to allow for comparison. Descriptive statistics, chi square, and paired t-test were used for the analysis. RESULTS: As of 31Dec2014, there were 38 active ingredients (28 chemical entities and 10 biologic) with a total of 43 orphan drug designations available in the US for treatment of leukemia, as compared to 29 drugs with 35 indications available in England (p<0.003). A higher percentage of drugs had orphan designation in the US than in England (p<0.001). Of the 6 drugs with orphan designation in both countries, ofatumumab (FDA approval in 2009; +53.0% more expensive in the US), dasatinib (2006; 72.4%), nilotinib (2007; 36.3%), and idelalisib (2007; 50.0%) were more expensive in the US. Two drugs, obinutuzumab (2013; -4.8%) and fludarabine (1990;-109.1%) were more expensive in England. The difference in prices was not statistically significant. CONCLUSIONS: More orphan drug treatment options for patients with leukemia were available in the US, whereas England provides a smaller number of drugs typically at a lower price than the US.