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

Area of Science:

• Biopharmaceutical Development
• Analytical Chemistry
• Regulatory Science

Background:

• The number of therapeutic protein pipelines has surged, necessitating accelerated early-stage development for novel therapies.
• Fast first-in-human (FIH) trials are crucial for bringing innovative treatments to patients promptly.
• Existing analytical method validation processes can be time-consuming, potentially delaying clinical progression.

Purpose of the Study:

• To describe a platform analytical method validation approach implemented by MSD for first-in-human (FIH) programs.
• To demonstrate how historical data and statistical analyses can support limited validation for new pipeline projects.
• To showcase the efficiency gains and successful implementation of this strategy in FIH filings.

Main Methods:

• Summarizing historical validation data within the same therapeutic protein modality.
• Applying statistical analyses to historical data to support limited validation for future projects.
• Utilizing a polysorbate 80 (PS-80) platform method as a case study for the workflow.

Main Results:

• The platform analytical method validation strategy significantly reduced the overall validation timeline from up to 4 months to 1-2 months.
• This approach has been successfully implemented in multiple FIH filings.
• The strategy streamlines the validation process without compromising scientific rigor.

Conclusions:

• Platform analytical method validation offers a viable strategy to accelerate early-phase biopharmaceutical development.
• This approach enables faster progression to first-in-human trials, benefiting patients awaiting novel therapies.
• Pharmaceutical companies can leverage this strategy to optimize their development timelines and regulatory submissions.