Liaison(s): Marisa Hewitt Ryan Shade
BioMarin Pharmaceutical, Inc. is a world leader in developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes. The KGI student team aims to help BioMarin make a big impact on small patient populations through a Team Master’s Project entitled Evaluation of Clinical Manufacturing Process Changes and Their Impact on Commercialization. The team has developed a tool to evaluate parameters of the manufacturing process based on their impact on commercialization. This tool is a stage-gated, real options-based decision tree, highlighting what would be required to implement each type of change at a given stage of development, considering cost, timeline, and regulatory requirements. The tool developed by the team is generic to BioMarin’s manufacturing processes and product pipeline, but takes into account the short development timelines relative to industry standards. The development of this tool required research in areas such as rare disease clinical development, regulatory affairs, bioprocessing and manufacturing. Research required for the development of the decision tree was interdisciplinary in nature, and was facilitated by generous contributions from BioMarin and KGI subject matter experts. The team successfully collected data outlining the detailed costs and timelines associated with implementing manufacturing process changes, considering the regulatory requirements associated with making these changes. This analysis includes numerous process changes and provides actionable information for each stage of clinical development. The tool is adaptable and provides the user with a customizable evaluation of implementing manufacturing process changes during clinical development, considering the impact on time to market, which is a key deciding factor at BioMarin.