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Drugs for Ultra-Rare Disorders: Cost Drivers and Budget Impact

by Angelika Oberhof

In this new paper, the Institute reports on cost drivers and budgetary impact of drugs for rare and ultra-rare disorders in Europe. In a systematic search, a total of seven studies with specific estimates of the budget impact of drugs for rare diseases for a total of nine countries were identified. Annual per-capita spending for orphan drugs ranged from €0.48 in Russia to €16 in France. Only one study on URDs was identified. In Germany, annual treatment costs per patient for drugs for non-oncological URDs vary between €1,175 and €726,890. In a regression analysis, a significant inverse relationship between availability of alternative treatments for the same indication and annual treatment costs was found. In addition, log prevalence was found to have a significant inverse relationship with log annual treatment cost.

Thus, an inverse relationship between prevalence and annual treatment costs was found specifically for drugs for non-oncological URDs. Despite annual treatment costs in the range of several hundreds to thousands of euros for some of the URD drugs, per-capita spending for URD drugs was found to be relatively small.

Also see Discussion Papers.