Recent Publication Considers Outcome-Based Approaches for Rare Disease Therapies

A recent article published in PharmacoEconomics considers outcomes-based approaches that may be used when considering the approval of rare disease therapies throughout the globe. Such a system ties a therapy’s price to patient outcomes. The article provides case studies from several countries and compares their approaches.

“The publication hopes to highlight practices that could aid in the development of tools to support future implementation of such agreements, when appropriate. Outcomes-based managed entry agreements represent one method of potentially mitigating the impacts of uncertainties in the clinical evidence base, for example, relating to the long-term clinical effectiveness of innovative therapies, at the time of their pricing/reimbursement and market entry.” Read more here.

(Source: The Evidence Base, 7/7/21)