Model-based DDI Extrapolation Strategy from Adults to Children – A Case Study with Risdiplam

Risdiplam (EVRYSDI) is approved for the treatment of patients with spinal muscular atrophy ≥ 2 months old. CYP3A-mediated DDI risk in pediatric patients was evaluated by physiologically-based pharmacokinetic (PBPK) modelling and informed the label. This talk will illustrate a modeling strategy for the extrapolation of DDIs from adults to children covering: Development of a patient-specific … Continued

The Scary Future of Rare Disease Management

In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug interventions that do not meaningfully benefit their covered lives. Rare disease therapies, often viewed as attractive areas for pharmaceutical & biotech manufacturers, represent some of the most expensive interventions available today given inherently limited competition. Yet, as more manufacturers enter … Continued

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