Implementing outcomes based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel

PharmacoEconomics

10 August 2021 - Enthusiasm for the use of outcomes based managed entry agreements to manage uncertainties apparent at the time of appraisal/pricing and reimbursement of new medicines has waned over the past decade, as challenges in establishment, implementation and re-appraisal have been identified. 

With the recent advent of innovative treatments for rare diseases that have uncertainties in the clinical evidence base, but which could meet a high unmet need, there has been renewed interest in the potential of outcomes based managed entry agreements.

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Michael Wonder

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Michael Wonder