Medical Scientific Director

BioMarin Pharmaceutical Inc.

141d

About The Position

The Medical Scientific Director provides day-to-day oversight of BioMarin-sponsored and collaborative studies across a therapeutic area, ensuring consistent study approach, conduct, monitoring, analysis, and reporting across one or more programs. The role partners with Real World Evidence/Epidemiology, Global Medical Leads, Data Sciences, Global Study Operations, and other cross-functional teams to develop practical study strategies and support hands-on execution of Medical Affairs-led studies. As part of the Integrated Evidence function, the Medical Scientific Director contributes expertise in evidence planning, observational study design, and data interpretation. The function aims to improve evidence planning and apply novel study designs to generate robust evidence about unmet medical need, support patient and endpoint selection for clinical studies, and inform medical practice concerning appropriate use and long-term safety and effectiveness of BioMarin products. The role executes evidence-generation activities, including non-interventional, low-interventional, and interventional Phase IIIb/IV studies, to address priority evidence gaps identified in product Integrated Evidence Plans. It provides scientific input into study concepts, protocols, analysis plans, reports, and regulatory responses; oversees assigned studies where relevant; and presents study results to internal and external stakeholders.

Requirements

PharmD, PhD, or MD with at least 5 years of experience conducting observational and Phase IIIb/IV studies within Clinical Development and/or Medical Affairs; alternatively, an MS or MPH with 10 or more years of similar experience.
Experience delivering a portfolio of protocols and ensuring they meet strategic program objectives.
Experience responding to regulatory agency and IRB/Ethics Committee questions regarding study designs and protocol conduct.
Proven ability to work with cross-functional partners to deliver studies end to end in support of product Integrated Evidence Plans.
Track record of delivering high-quality novel observational and Phase IIIb/IV studies.
Ability to work effectively in cross-functional matrix settings and shape study strategy for assigned projects.
Understanding of statistical and data analysis, research methods, and study design.
Experience in study monitoring.
Advanced knowledge of the clinical research process and Good Clinical/Pharmacoepidemiology Practice standards and requirements for conducting research and disseminating scientific information.
Strong scientific writing and document-development skills.

Nice To Haves

Awareness of novel prospective observational and interventional study designs.

Responsibilities

Execute evidence-generation activities, including non-interventional, low-interventional, and interventional Phase IIIb/IV studies, to address priority evidence gaps in product Integrated Evidence Plans.
Collaborate with Global and Country Medical Leads, the RWE Lead, and HEOR to understand global and local needs and opportunities and develop study pull-through and dissemination plans.
Work with operational strategic partners to implement GMAF-led studies, identify issues promptly, lead complex solutions, and escalate issues as appropriate.
Provide scientific input into study concepts, protocols and amendments, statistical analysis plans, data review plans, and responses to regulatory agency questions about scientific aspects of study protocols.
Lead development of annual, interim, and periodic reports for Medical Affairs-led studies, coordinating subject matter expert input, data interpretation, and presentation, and ensuring relevant regulatory questions are addressed.
Design secondary data analysis plans using BioMarin clinical and real-world data to support program strategy.
Provide medical monitoring oversight of assigned studies where relevant, including subject eligibility, safety and study-data quality, informed consent forms, case report forms, tables/listings/graphics, adverse event and concomitant medication coding, and inspection readiness.
Present assigned study results to internal and external stakeholders, including at scientific conferences and other relevant forums.
Coordinate Medical Affairs input for regulatory submissions, regulatory responses, IRB/Ethics Committee questions, and preparation for program inspections and audits.
Participate in initiatives to improve or increase the efficiency of study implementation.