BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options.
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 Director / Medical Director has a key scientific and analytical leadership role in the development of Clinical Science 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 Clinical Science study deliverables and monitoring subject eligibility, study data and contributing critically to the scientific interpretation and integration of clinical study results.
The Director / Medical Director may also contribute to the scientific strategy and plan as outlined in the Clinical Development Plan.
The Medical Director is also responsible for providing medical input into the creation and review of all Clinical Science study deliverables to support clinical decision making, problem solving, and safety surveillance, as well as collaboration with external physician stakeholders
Scientific Leadership
- Support the definition of the core clinical and scientific strategy that serves as the basis for the CDP and provide Clinical Science expertise in the design and execution of clinical studies under the purview of the CDP
- Participate (as assigned) in the development of clinical science 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 Clinical Science 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 rational 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 (per protocol specific Protocol Deviation Plan)
- 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 (per the study specific Medical Monitoring Plan)
- 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 (final listing review, review of blinded tables, listings and figures (TLFs), etc.) and actively participate in all data snapshots taken (not limited to final DBL)
- 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)
- The Medical Director will also 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 Clinical Science to ensure appropriate dissemination of information and communication (including functional managers and other relevant individuals)
- 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 Clinical Science
- Proactively identify and communicate potential risks and mitigations relevant to the Clinical Science deliverables
- Contribute to the development and maintenance of study- specific plans; manage the development of study-specific plans that are the responsibility of the Clinical Science
- Collaborate with CLS Therapeutic Area Lead, CDTL and CSL on all study related decisions, as appropriate
- Escalate issues affecting CLS function deliverable quality, timelines, resources or budget, as appropriate
Competencies:
The Director / 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 Director / 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/Experience:
- Medical Director: MD, MD/PhD (or equivalent)
- Director: Advanced degree in life or health sciences (e.g. PhD/PharmD/RN)
- 4 or more years of relevant experience in genetic diseases, metabolic diseases, and/or rare diseases desired;
- Early clinical development experience desirable
- Clinical development experience in Duchenne Muscular Dystrophy preferred
- No people management expected
- Domestic and International Travel Required
We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law
