Medical Director, Clinical Science

BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need.

BioMarin Clinical Science (CLS) is responsible for overseeing clinical programs across various phases, from proof-of-concept to Phase 3 and BLA/NDA/MAA filing. The Clinical Science team provides leadership for clinical strategy and oversight to ensure excellence in clinical trial conduct, data analysis and interpretation, publication preparation, and safety monitoring.

The Medical Director has a key scientific and analytical leadership role in the development of CLS core deliverables across study planning, design and execution, results analysis and regulatory filings. Key focus areas include:

• Acting as a study Medical Monitor and providing the scientific input into the creation and review of all CLS study deliverables.
• Monitoring subject eligibility and study data.
• Contributing critically to the scientific interpretation and integration of clinical study results.
• Providing medical input into the creation and review of all CLS study deliverables to support clinical decision making, problem solving, safety surveillance, and collaboration with external physician stakeholders.

Key Responsibilities:

• Scientific Leadership: Support the definition of the core clinical and scientific strategy that serves as the basis for the Clinical Development Plan (CDP).
• Participate in the development of CLS-assigned sections of regulatory filings and in drafting regulatory responses in collaboration with Medical Writing.
• Establish relationships with investigators and KOLs as appropriate in support of the CDP.
• Stay up to date with advances in literature in therapeutic/disease area including mechanism of action, diagnostic tests, treatment, drug development trends, and regulatory requirements.
• Develop therapy-specific publication plans and work with internal and external colleagues to prepare study results for timely publication.
• Attend and contribute to relevant scientific conferences, seminars or presentations.

Clinical Study Planning and Monitoring:

• Act as the scientific subject matter expert, and primary contact, for assigned clinical study; take a proactive approach to identifying issues and mitigating risk.
• Initiate and provide the medical and scientific content and insight for development and review of protocols, protocol amendments, ICFs, CRFs, statistical tables and listings.
• Provide clinical input to statistical analysis plan to ensure alignment with Regulatory and business interests.
• Facilitate the review and approval of all study related CLS deliverables and content.
• Act as study scientific subject matter expert and main point of contact for Principal Investigators (PIs) and sites.
• Conduct periodic review of protocol deviations in collaboration with Clinical Operations Study Lead or designee.
• Attend and present at Investigator Meetings, as needed.
• Conduct data review, assessment and interpretation of clinical data to ensure that the data are correct and presented with the appropriate interpretation.
• Review and analyze SAEs, safety and efficacy trends on an ongoing basis.
• Work closely with the Pharmacovigilance representatives providing medical input into safety reports.
• Participate in and provide scientific advice during key Database Lock (DBL) activities.
• Collaborate with Biometrics to identify key issues, prepare content and facilitate discussions at study Data Review Board or Data Monitoring Committee.

Governance and Communication:

• Provide timely and high-quality functional deliverables and contributions to Study Execution Team (SET).
• Act as primary point of contact between SET and BCLS to ensure appropriate dissemination of information and communication.
• Provide agenda topics to be discussed during SET meetings.
• Act as the medical and scientific subject matter expert to the SET.
• Proactively identify and communicate potential risks and mitigations relevant to the BCLS deliverables.
• Contribute to the development and maintenance of study-specific plans.

Competencies: The Medical Director is expected to exhibit mastery-level understanding of multiple technical competencies, including scientific knowledge, data analysis, and scientific writing. S/he should also have advanced-level competence in several areas, including strategic thinking and problem solving.

Education and Experience: MD, MD/PhD (or equivalent) Advanced degree in life or health sciences (e.g. PhD/PharmD/RN). 5 or more years of relevant experience in Clinical Development, with experience in genetic diseases, specialty care, and/or rare diseases desired; Clinical experience preferred.