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Rare innovation: How it happens, when it doesn’t, and what can be done to sustain it

Isabelle Laurence, Adam Hutchings, Elena Nicod
October 2023

Addressing remaining unmet needs in rare diseases hinges on a positive and enduring policy environment which helps to achieve the necessary clinical and economic conditions for drug development in small, hard-to-treat populations. This white paper examines through historic case analysis the factors which make up those conditions and how these are shaped by policy to encourage (or deter) innovation.


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