Year: 2014-15
Company: BioMarin
Liaison(s): Javier Femenia Thomas Lester
BioMarin Pharmaceuticals, Inc. was founded in 1997 in Novato, California to develop and commercialize therapies for rare genetic diseases. Their strategy to develop first-to-market therapies with an orphan disease status has been remarkably successful and the current product portfolio comprises five approved products as well as multiple pre-clinical and clinical candidates. Given the limited manufacturing capacity of any industrial plant, the BioMarin TMP team has been tasked with investigating strategies to increase BioMarin’s production capacity and capability by analyzing the feasibility of implementing a number of changes to the manufacturing process for one of their products. The benefits of the proposed changes include decreasing the production time and costs while building in-house capabilities and increasing the available resources in BioMarin’s manufacturing facility. The team has been assessing the technical, regulatory, and financial issues that BioMarin must address in order to implement these post-approval process changes. Historically, companies have avoided implementing post approval process changes in order to avoid large capital outlays and extensive regulatory investments in additional clinical trials and other comparability tests. However, companies might gain significantly from the post approval process changes if they were to make these investments. Typically, process capacity and efficiency could be enhanced in additional to other potential benefits. The team’s task was to investigate and fully understand the potential risks and benefits of a number of post approval process changes. With this information in hand, the BioMarin Pharmaceuticals TMP Team was expected to outline a recommended course of action. To accomplish the project objectives, the team implemented two semester-phase milestones. The first milestone was to conduct interviews to understand the industry’s best practices with respect to manufacturing processes. As part of this effort the team classified each manufacturing change and identified critical regulatory hurdles. As part of this effort the team also provided a comprehensive risk-benefit analysis and risk mitigation plan associated with the different manufacturing changes. Finally, the BioMarin Pharmaceuticals TMP Team assessed the economic feasibility, and provided recommendations for post approval process changes from a technical, regulatory and economical perspective.