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How did we get here and where do we go now? An economist’s perspective on EU orphan drug policy

Adam Hutchings
February 2021

The divergence between the rapid pace of scientific progress in rare diseases and declining sentiment towards orphan medicines reflects a paradox that is at the heart of the European orphan drug debate. While the last twenty years have witnessed a sharp increase in the authorisation of orphan medicines, and the greatest scientific innovation of any sector of the biopharmaceutical industry, it has also become an embodiment of the concerns of politicians, policymakers, payers, as well as patients and physicians, about the same biopharmaceutical industry model that has spawned these innovations.

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