Achieving Acceleration to First-in-Human: MSD’s Learnings on Platform Method Validation Strategy

Abstract

Over the past decades, the number of therapeutic protein pipelines in early-phase clinical studies has increased dramatically. The rapid growth in the pipeline underscores the need to accelerate early-stage development and enable fast first-in-human (FIH) trials to bring novel therapies to patients. Across the industry, various efforts have been developed to achieve this goal. In this communication, a platform analytical method validation approach developed and used by MSD for FIH programs is described. A case study from the release panel, a polysorbate 80 (PS-80) platform method is utilized to illustrate the workflow. In this approach, historical validation data within the same modality are summarized and supplemented with statistical analyses to justify a limited validation for future pipeline projects. The platform method validation strategy has reduced the overall validation timeline from up to 4 months to 1–2 months and has successfully been implemented in FIH filings. This communication provides insights to pharmaceutical companies interested in developing platform analytical method validation approaches for fast FIH studies.