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56; figure 3 inset). A significant inverse association was also observed for the relationship between annual cost and the prevalence for 21 drugs approved for managing ultra-orphan diseases (p=0.04; figure 4). However, a scatter plot of the subset of the 53 drugs approved for orphan diseases with prevalence >1 per 100?000 revealed a non-significant relationship (p=0.18). Scatter plots of prevalence selleckchem against annual cost did not reveal significant relationships when cancers or inborn errors of metabolism were individually tested (data not shown). Figure?3 Scatter plot of orphan disease prevalence against annual cost. Figure?4 Scatter plot of ultra-orphan disease prevalence against annual cost. Association between clinical effectiveness and annual cost Because of discrepancies in outcome measures and time points for outcome measurements, we could not use scatter plots to explore associations between clinical effectiveness and annual costs. All orphan drugs approved for managing pulmonary arterial hypertension showed comparative level of effectiveness at improving 6?min work distance, and decreasing clinical worsening irrespective of annual cost (see web appendix 4). Similar findings were observed for progression-free survival and overall survival for orphan drugs approved for treating cancers. The annual costs of two drugs approved for treating Pseudomonas in cystic fibrosis were comparable. We could not determine whether the risk or occurrence of serious adverse events played a role in the annual costs of approved orphan drugs. Association between year of approval and annual cost Two or more orphan drugs were approved by the EMA for treating seven orphan conditions (see web appendix 4). Scatter plot of annual cost against year of approval for the five drugs approved for pulmonary arterial hypertension suggested a trend towards a significant relationship for higher annual costs with more recent approvals (p=0.06). A significant relationship between annual cost and year of approval was observed with four drugs approved for the management of chronic myeloid leukaemia (p=0.03). There was no significant relationship between annual cost and year of approval for drugs approved for treatment of acute lymphoblastic leukaemia (p=0.38); however, exclusion of the most recently approved drug of which approval was based on a historical perspective (Xaluprine) resulted in a significant relationship being observed (p=0.01). Branded orphan drugs versus generic or unlicensed versions We found 15 approved drugs with generic versions, of which data on annual cost of generic or unlicensed versions for 10 (13.5%) were available. Figure?5 shows a price comparison in annual costs of branded orphan drugs compared with their unlicensed/generic equivalents. While branded mercaptopurine (Xaluprine) was only 1.