Back to: FDA Clinical Investigator Training Course (CITC) 2024
Presenter
James P. Smith, MD, MS
Director
Office of New Drug Policy (ONDP)
Office of New Drugs (OND)
Center for Drug Evaluation and Research (CDER)
US Food and Drug Administration (FDA)
Dr. Smith is the Director of the Office of New Drug Policy in the Office of New Drugs (OND), previously having held various leadership positions within the office since its formation in 2018. Prior to his roles in OND Policy, he held positions as a primary reviewer, team leader, and deputy division director of the former Division of Metabolism and Endocrinology Products after joining FDA in 2011. Dr. Smith has overseen development programs targeting diseases ranging from the very rare to the very common, and his specific interests include clinical, scientific, and policy considerations related to generating evidence to demonstrate the safety and effectiveness of new drugs and biological products. Dr. Smith is a graduate of the University of Michigan Medical School and completed an Internal Medicine residency at the same institution before fellowships in both nephrology and clinical pharmacology at Vanderbilt University Medical Center. He also holds a master’s degree in Clinical Research Design and Statistical Analysis from the University of Michigan School of Public Health
Abstract
Jim Smith’s presentation, “Basics of Clinical Trial Design,” outlines the fundamental principles for conducting adequate and well-controlled studies to distinguish a drug’s effect from other influences like spontaneous changes, placebo effects, or biased observations. He emphasizes the importance of clear objectives and a design allowing valid comparisons with control groups. Key to minimizing bias is randomization, which ensures group comparability at baseline, and blinding, which conceals treatment assignments to ensure similar treatment and observation throughout the trial. Smith details five types of control groups: placebo, no treatment, dose-response, active, and externally controlled trials. He also discusses optimal study population selection, including enrichment strategies to enhance drug effect detection. The presentation covers the critical role of well-defined endpoints, differentiating between clinical outcomes and surrogate endpoints, and outlines various study designs like parallel group, crossover, and factorial. Smith concludes by stressing the need for assay sensitivity in non-inferiority trials and advocates for focusing resources on essential study activities while incorporating broad stakeholder input to optimize trial design.