Medical Director, Clinical Science

Who We Are
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. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical, and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options.

About Worldwide Research And Development
From research and discovery to post-market clinical development, our WWRD 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 genetic diseases.

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.

Summary Description:
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 and monitoring subject eligibility, study data, and contributing critically to the scientific interpretation and integration of clinical study results. The Medical Director will also contribute to the scientific strategy and plan as outlined in the Clinical Development Plan (CDP). The Medical Director is also responsible for providing medical input into the creation and review of all CLS study deliverables to support clinical decision making, problem solving, and safety surveillance, as well as 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 CDP and provide CLS expertise in the design and execution of clinical studies under the purview of the CDP.
• Participate (as assigned) 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 including accurate AE (MedDRA) and concomitant medications (WHODrug) coding; audit reports; clinical study reports; inspection readiness activities, regulatory submissions, and other key study deliverables.
• 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 to assess subject eligibility, provide scientific rationale and manage ongoing protocol issues.
• Provide leadership to sites by developing or participating in training, answering investigator/site questions about the protocol.
• Respond to site and Health Authority questions about the protocol.
• 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 including thorough review of SAEs and other important AEs.
• Review and analyze SAEs, safety and efficacy trends on an ongoing basis.
• Work closely with the Pharmacovigilance representatives providing medical input into safety reports including, SAE narratives and analysis of similar events, Development Safety Update Reports (DSURs) and Suspected Unexpected Serious Adverse Reactions (SUSARs) reports, Company Core Safety Information (CCSI), Investigator Brochure (IB), Risk Management Plans, Integrated Summaries of Safety and Efficacy, Clinical Study Reports, and preparation of labels.
• Participate in and provide scientific advice, as appropriate, during key Database Lock (DBL) activities and actively participate in all data snapshots taken.
• Collaborate with Biometrics to identify key issues, prepare content and facilitate discussions at study Data Review Board (internal) or Data Monitoring Committee (DMC).
• Lead the selection of and interactions with independent Data Monitoring Committees (DMC).
• Act as the medical subject matter expert for review of clinical and safety data to ensure data are correct and presented with the appropriate medical interpretation and for discussing safety concerns with sites.

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 (SME) to the SET with the ability to make decisions and recommendations on behalf of BCLS.
• Proactively identify and communicate potential risks and mitigations relevant to the BCLS deliverables.
• Contribute to the development and maintenance of study-specific plans; manage the development of study-specific plans that are the responsibility of BCLS.
• Collaborate with BCLS Therapeutic Area Lead, CDTL, and CSL on all study-related decisions, as appropriate.
• Escalate issues affecting BCLS function deliverable quality, timelines, resources, or budget, as appropriate.

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. The Medical Director should:

• Identify scientific and medical knowledge gaps in therapeutic area and target populations to drive research and publication strategies.
• Assess the clinical and medical impact of clinical and external research findings and data on overall therapeutic area and clinical development strategies.
• Conduct comprehensive document evaluations including evaluation of statistical presentations, research methods, quality, and completeness of content.
• Identify internal and external best practices, trends, developments, or alternative approaches that can be leveraged for strategy development.
• Demonstrate ability to look beyond the obvious toward innovative approaches, avoiding biases and historical crutches.

The Medical Director will also demonstrate mastery of relevant clinical and therapeutic area knowledge to support clinical decision making, problem solving, safety surveillance, and as well as collaboration with external physician stakeholders.

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
Note: This description is not intended to be all-inclusive or a limitation of the duties of the position. It is intended to describe the general nature of the job that may include other duties as assumed or assigned.

Equal Opportunity Employer/Veterans/Disabled
An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.