Case Study Example #2: Leveraging the 505(b)(2) Pathway for an Investigational New Drug Formulation Using New Administration Modalities (CE1-3)

Sponsors can leverage the Agency’s previous findings of safety and efficacy using the 505(b)(2) pathway. However, identifying data gaps and knowing when additional nonclinical studies are warranted can be challenging. This talk will present a case study involving a proposed intravenously administered new drug product formulation for short term use containing an active ingredient already approved for acute and chronic uses. The audience will be asked to evaluate the overall program design, species selection and challenges for toxicity assessment including formulation composition and quality issues (excipients, impurities, CMC-related changes) arising during clinical development. Learning objectives: 1) identify how regulatory pathways and expectations can impact the nonclinical program design, 2) understand the technical challenges of program execution, 3) understand how to identify and assess the impact of CMC related changes during development, and 4) understand risk mitigation through effective regulatory interactions with health authorities.