Medical Scientific Director

Who We Are

Bio Marin is a leading rare disease biotechnology company focused on genetically defined conditions.

Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options.

More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world‑class manufacturing capabilities. At the heart of Bio Marin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

From research and discovery to post‑market clinical development, our R&D engine involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first‑in‑class and best‑in‑class therapeutics that provide meaningful advances to patients who live with rare diseases.

The Medical Scientific Director is accountable for the day‑to‑day oversight of numerous Bio Marin‑sponsored and collaborative studies across a therapeutic area, ensuring consistency of approach, conduct, monitoring, analysis, and reporting of studies within one or more programs. The Medical Scientific Director will work in partnership with Real World Evidence (RWE)/Epidemiology, Global Medical Leads, Data Sciences and Global Study Operations to develop practical study strategies and ensure optimal hands‑on study operations and execution support for Medical Affairs‑led studies from cross functional partners.

The Medical Scientific Director is part of the Integrated Evidence function comprised of subject matter experts in evidence planning, observational study design and data interpretation. The function works to drive excellence in evidence planning and implement novel study design approaches to generate robust evidence to enhance understanding of unmet medical need, support patient and endpoint selection for clinical studies and inform medical practice on appropriate use and long‑term safety and effectiveness of Bio Marin products to drive clinical adoption.

• Executes evidence generation activities (non‑interventional, low‑interventional and interventional Phase IIIb/IV studies) that will fill priority evidence gaps identified in product Integrated Evidence Plans (IEPs)
• Collaborates closely and builds alliances with Global and Country Medical Leads, RWE Lead, and HEOR to understand key global and local (key market) needs and opportunities in order to develop strategic study pull through and dissemination plans
• Collaborates closely with operational strategic partners to ensure successful implementation of GMAF‑led studies; identifies issues in a timely manner, leads implementation of complex solutions, and/or escalates as appropriate
• Provides scientific input into the development of study concepts, protocols and protocol amendments, statistical analysis plans, data review plans, and responses to regulatory agency questions regarding scientific aspects of study protocols
• Leads the development of annual, interim and periodic study reports for Medical Affairs‑led studies, including coordination of SME input, data interpretation and presentation, and ensuring that regulatory agency questions (where relevant) have been adequately addressed
• Designs secondary data analysis plans of Bio Marin clinical and real‑world data to support program strategy
• Ensures adequate medical…