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The Application of Clinical Pharmacology for the Treatment of Rare Diseases
ASCPT Members-Only Webinar Presented by the Pharmacometrics & Pharmacokinetics (PMK) and Rare Diseases (RD) Communities

Speakers: Mariam Ahmed, PhD; Michael Pacanowski, PharmD; and Ramona Walls, PhD
Moderators: John Crowley, JD; and Tao Long, PhD

The drug development for rare disease have unique challenges, both from scientific and operational perspectives. The webinar will discuss how clinical pharmacology tools (e.g., MIDD, In Vitro models) can be used to overcome some of these challenges and help bring efficient, safe, and effective treatment to patients. Examples that will be presented include but not limited to approval expansion based on In Vitro determination of efficacy, use of exposure-response (E-R) to support efficacy, determination of similarity of E-R in adults and pediatric patients with systemic lupus erythematosus (SLE), and qualification of surrogate endpoint in pediatric patients with pulmonary arterial hypertension. The webinar will also highlight the need for information/data sharing and collaborations to advance therapeutic development for rare diseases. An example is the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) initiative. The regulatory history and advancement in regulatory science in the risk benefit assessment of orphan drug approvals will also be discussed.

Learning Objectives:
1. Develop a better understanding on the current status and challenges in drug development for rare diseases.
2. Familiarize with the applications the state-of-the-art clinical pharmacology tools as well as pharmacometrics tools to facilitate the drug development for rare diseases.
3. importance of collaboration and data sharing will be highlighted using Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) initiative as an example.

Dec 13, 2022 02:00 PM in Eastern Time (US and Canada)

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