2023 ACCP Virtual Journal Club Webinar: Model-Informed Approach Supporting Drug Development and Regulatory Evaluation for Rare Diseases | ON DEMAND

On Demand webinar recording: May 17th, 2023 to May 17th, 2026

Why is this article important to your practice? 

For rare diseases, it is challenging to enroll a large number of patients and obtain all critical information to support drug approval through traditional clinical trial approaches. In addition, over half of the population affected by rare diseases are children, which presents additional drug development challenges. In the context of rare diseases, maximizing the use of all available data is in the interest of drug developers and regulators. This provides opportunities for model-informed drug development(MIDD) to use and integrate all available sources and knowledge to quantitatively assess the benefit/risk of a new product under development and to inform dosing. Learners that complete this activity will be able to explain the application of MIDD in rare diseases.

Target Audience:

 Physicians, Pharmacists, PhDs and other healthcare professionals interested in the application of model-informed drug development.

Learning Objectives

After completing this activity, the learner will be able to:

1. Explain why MIDD is important for rare diseases;
2. Discuss how MIDD can be applied to enhance drug development for rare diseases;
3. Learn about the future direction for MIDD application to enhance drug development for rare diseases.