Knight C, Pearson I. Economic modelling considerations for rare diseases. Presentation to be given at the ISPOR 2019 European Conference; November 4, 2019. Copenhagen, Denmark.


When seeking orphan drug approval, demonstrating value for therapies can be a challenge. The limited amount of data available affects the feasibility of developing models that meet good practice recommendations for health technology assessment submissions. We will discuss different approaches for developing a robust model structure that can help you with orphan drug reimbursement.

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