Addressing the Socioeconomic Burden and Policy Barriers in Rare Diseases

Moderator

Ka Keat Lim, MSc, RPh, PhD, Queen Mary University of London, London, United Kingdom

This session addresses challenges and innovations in rare disease research, policy, and care. Presentations cover the social and economic burden of inherited retinal disease in Sweden, the evolution of risk-sharing agreements for orphan drugs over the past decade, and a catalogue of actions to overcome policy barriers to drug repurposing. The session concludes with a decision-analytic framework for evaluating personalized, patient-centered care solutions in multiple system atrophy.

Code

129

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