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From Lab to Clinic: Model-Based Approaches in Rare Disease Drug Development

ASCPT Members-Only Webinar Presented by the Rare Diseases (RD) Community

Speaker: Arijit Chakravarty, PhD
Moderators: Tao Long, PhD; and Mariam Ahmed, PhD

Rare Diseases pose a dual challenge for drug development as they often lack predictive preclinical pharmacology models, and the patient recruitment and retention are rate-limiting in the clinic. In common diseases, drug development follow sequential process of Phase 1 single and multiple ascending dose studies that typically start at very low conservative dose, Phase 2a proof of concept study, Phase 2b dose ranging study, two pivotal phase 3 studies. Unfortunately, due to the rarity of the condition, the unmet medical need and the need to bring medication faster, drug development typically follows faster paradigm with many cases where the development programs involve Phase 1/2 study followed by a Phase 2/3 confirmatory efficacy study. Additionally, due to ethical considerations imposed by the modalities targeting rare disease, Phase 1 ascending dose study sometimes needs to be in patient. This webinar aims at discussing ways to use parsimonious model-based approaches to facilitate the path to clinical proof-of-concept for Rare Diseases. Specifically, the focus of this webinar will be on the use of modeling at the stage of candidate selection, as a translational tool to project drug disposition and inform first in human dose selection, to inform disease progression for early go/no go decision making. Successful case studies will be presented, and the view of sponsor and regulatory side will be shared through a third-party consulting company.
Learning Objectives:
1. Understand the quantitative toolbox available for clinical pharmacologist to ensure informed decisions.
2. Understand how to design an impactful model-based strategy for translational and clinical pharmacology that can inform regulatory decision.

Dec 15, 2022 11:00 AM in Eastern Time (US and Canada)