Medical Scientific Director

Overview

In this role you will oversee BioMarin-sponsored and collaborative studies across therapeutic areas, ensuring rigorous conduct and timely reporting. You will partner with RWE, epidemiology, and global medical teams to shape study strategy and deliver evidence that informs medical practice and regulatory decisions. You will lead observational and interventional studies, contribute to protocol design and regulatory responses, and present findings to internal and external audiences. This is a cross-functional, impact-focused position at the Integrated Evidence function, offering meaningful opportunities to advance rare-disease therapies.

Responsibilities

• Execute evidence generation activities for Phase IIIb/IV studies to fill priority gaps
• Collaborate with Global Medical Leads, RWE, and HEOR to align on global/local needs and study dissemination
• Partner with operations to implement complex study solutions and address issues
• Contribute to study concept development, protocols, statistical analysis plans, and regulatory responses
• Lead development of annual/interim/periodic study reports and manage SME input
• Design secondary data analysis plans for clinical and real-world data
• Ensure medical monitoring oversight including eligibility, safety data, and coding
• Present results at scientific conferences and relevant forums
• Coordinate Medical Affairs input for regulatory submissions and inspections
• Participate in efficiency initiatives in study implementation

Key requirements

• PharmD/PhD/MD with at least 5 years of experience in observational and Phase IIIb/IV studies
• MS/MPH with 10+ years of similar experience
• Experience delivering multiple protocols aligned with program objectives
• Experience addressing regulatory and IRB/Ethics Committee questions on study design and conduct

• cross-functional collaboration
• scientific writing and communication
• regulatory liaison and negotiation
• observational and interventional study design
• Phase IIIb/IV study execution
• statistical analysis and data interpretation

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