Advisory Committee for Pharmaceutical Science and Clinical Pharmacology

Advisory Committee for Pharmaceutical Science and Clinical Pharmacology

Committee Meeting on March 2, 2011, from 7:15 a.m. to 3 p.m.

Agenda: The committee will discuss innovative approaches to the development of drugs for orphan and rare diseases to support decisions such as dose and trial design selection. FDA will seek input and comment on how to optimally utilize mechanistic biomarkers and apply clinical pharmacology tools, such as pharmacogenetics and modeling and simulation, to facilitate efficient and informative drug development and regulatory review. FDA will present and seek input from the committee on how lessons learned from other applications of clinical pharmacology tools in pediatrics and oncology can be applied to orphan and rare disease drugs. The committee will be asked to comment on the current status and future direction for clinical pharmacology studies (e.g., dose-response, drug-drug interactions, pharmacokinetics in patients with renal or hepatic impairment) as they pertain to drug development for orphan and rare diseases