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

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.