Medical Scientific Director

The Medical Scientific Director is accountable for the day-to-day oversight of numerous BioMarin-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 role partners with Real World Evidence (RWE)/Epidemiology, Global Medical Leads, Data Sciences and Global Study Operations to develop practical study strategies and provide 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 comprising subject matter experts in evidence planning, observational study design and data interpretation. The function drives excellence in evidence planning and implements 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 BioMarin products to drive clinical adoption.

Responsibilities

• Execute evidence generation activities (non-interventional, low-interventional and interventional Phase IIIb/IV studies) that fill priority evidence gaps identified in product Integrated Evidence Plans (IEPs).
• Collaborate with Global and Country Medical Leads, RWE Lead, and HEOR to understand key global and local needs and opportunities and develop strategic study pull-through and dissemination plans.
• Work with operational strategic partners to ensure successful implementation of GMAF-led studies; identify issues, lead complex solutions, and escalate as appropriate.
• Provide scientific input into study concepts, protocols and protocol amendments, statistical analysis plans, data review plans, and responses to regulatory questions regarding scientific aspects of study protocols.
• Lead the development of annual, interim and periodic study reports for Medical Affairs-led studies, coordinating SME input, data interpretation and presentation, and addressing regulatory questions where relevant.
• Design secondary data analysis plans of BioMarin clinical and real-world data to support program strategy.
• Ensure adequate medical monitoring oversight of assigned studies including monitoring and arbitration on subject eligibility, safety data quality, development and review of ICFs, CRFs, TLGs, accurate AE and concomitant medication coding, and inspection readiness activities.
• Present results of assigned studies to internal and external stakeholders, including at scientific conferences and other relevant forums.
• Coordinate Medical Affairs input in preparation of regulatory submissions, responses to regulatory queries, IRB/Ethics Committee questions, and program regulatory inspections and audits for assigned programs.
• Participate in initiatives to enhance or drive efficiency in study implementation.

Qualifications

• PharmD/PhD/MD with at least 5 years of experience conducting observational and Phase IIIb/IV studies within Clinical Development and/or Medical Affairs; MS/MPH with 10+ years of similar experience.
• Experience delivering a portfolio of protocols and ensuring they meet strategic program objectives.
• Experience responding to regulatory agency, IRB/Ethics Committee questions regarding study designs and protocol conduct.
• Proven track record of working with cross-functional partners to ensure successful end-to-end delivery of studies 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 a cross-functional matrix to shape study strategy for one or more assigned projects.
• Awareness of novel prospective observational and interventional study designs.
• Understanding of statistical and data analysis, research methods and design.
• Experience in study monitoring.
• Advanced knowledge of the clinical research process and Good Clinical / Pharmacoepidemiology Practice standards and requirements related to conducting research and disseminating scientific information.