Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement.

There has been an increasing trend of relying on financial-based instead of outcomes-based MEAs because they are simpler and easier to implement, despite the fact that they can only mitigate some types of uncertainty, (e.g., around the size of the treatment population to limit budget impact) but do not de-risk uncertainties about the drug effect itself (e.g., size or persistence of effect) ( Whittal et al., 2022 ).

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