Supporting Drug Development for Rare Diseases using QSP

In Supporting Drug Development for Rare Diseases using QSP, we explore how Quantitative Systems Pharmacology is transforming every stage of therapeutic discovery and development for conditions that lack traditional commercial focus. Each episode dives into the power of multi‑scale mechanistic modeling to connect molecular interactions with cellular, tissue, and whole‑body dynamics, especially when patient data are scarce. You’ll learn how virtual patient cohorts and in silico trials can predict efficacy and safety across diverse subpopulations, reducing reliance on large clinical studies. We unpack strategies for identifying novel targets and repurposing existing compounds, optimizing dosing regimens and trial endpoints, and ensuring regulatory readiness through standardized model qualification. Along the way, we spotlight cutting‑edge AI advances—rapid literature mining, automated data extraction, and intelligent trial‑design workflows—that turbocharge model construction, hypothesis generation, and decision‑making. Whether you’re a systems pharmacologist, drug developer, or curious innovator, tune in to discover how QSP and AI are enabling faster, more efficient, patient‑centric therapies for rare diseases.

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