Genetic Disease Specialist Leader

About BioMarin Pharmaceutical Inc.

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. With an unparalleled expertise in genetics and molecular biology, we develop transformative medicines for patients with significant unmet medical need.

Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with genetic diseases. 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.

Clinical Science at BioMarin

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. Our teams oversee clinical programs across various phases, from proof-of-concept to Phase 3 and BLA/NDA/MAA filing.

Associate Director / Associate Medical Director Role

We are seeking an experienced Associate Director / Associate Medical Director to join our Clinical Science team. As an emerging leader within Clinical Science, you will lead tasks related to Clinical Science core deliverables across all stages of program and study design and execution, data analysis and regulatory filings as delegated by the Clinical Science Therapeutic area lead or other Clinical Science leader.

You will be responsible for building and leveraging relationships with external parties (KOLs, Investigators); conducting data analysis in support of reports and key messaging. The Associate Director / Associate Medical Director may also take on the role of Medical Monitor for a study depending on clinical experience.

Key Responsibilities

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• Scientific Expertise
  
  
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  • Develop key documents to support the components of trial execution and regulatory submission (including IND Annual Reports, Investigator Brochures, Briefing Books and Pediatric Plans).
    
    
  • Support the design, update and implementation of Clinical Development Plans (CDPs), and work with Project Management to ensure progress in line with current plans and timelines.
    
    
  • Develop scientific rationale for methods, design and implementation of clinical protocols, data collection systems and final reports.
    
    
  • 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.
    
    
  • Engage and establish rapport with KOLs and scientific or clinical experts.
    
    
  • Contribute to content and submission of abstracts to key symposia.
    
    
  • Work with CLS and Clinical Operations to ensure the success of Investigator Meetings by contributing and presenting scientific content.
    
  
  
  
• Data Analysis
  
  
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  • Develop thorough understanding of clinical trial data to help guide the drafting of CSRs and Key Messages.
    
    
  • Perform preliminary analyses on clinical study data as it becomes available to help build and guide Statistical Analysis Plans (SAPs).
    
    
  • Help define study quality metrics and perform study data reviews during study execution to ensure integrity of accruing study data.
    
  
  
  
• Clinical Study Monitoring
  
  
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  • 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 scientific content and insight for development and review of: protocols, protocol amendments; statistical tables and listings including accurate AE (MedDRA) and concomitant medications (WHODrug) coding; clinical study reports; inspection readiness activities, regulatory submissions and other key study deliverables.
    
    
  • Act as study scientific subject matter expert and main point and 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/HA questions about the protocol.
    
    
  • Conduct review, assessment and interpretation of clinical and safety 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).
    
    
  • Conduct periodic review of protocol deviations in collaboration with Clinical Operations Study Lead or designee (per protocol specific Protocol Deviation Plan).
    
    
  • 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.
    
    
  • 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).
    
    
  • 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).
    
    
  • Contribute to and review Clinical Study Report (CSR) writing (review final TLFs, work with medical writer to review & revise CSR document, draft discussion and conclusions, participate in comment resolution meetings).
    
    
  • Recommend and develop Key Opinion Leader (KOL) and Advisory Panel Networks, as appropriate.
    
    
  • Provide representation at regulatory meetings, as needed.
    
    
  • The Associate 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
  
  
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  • 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).
    
    
  • 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.
    
  
  
  

Competencies

We expect the Associate Director / Associate Medical Director to demonstrate advanced skills in communication and collaboration, proactivity, scientific knowledge, drug development, data analysis, and scientific writing. You will have a strong understanding of clinical study protocol scientific rationale, methodology, biostatistical concepts and data analysis and procedures through the life of the clinical trial.

In this role, you will anticipate risks or issues to program or business and take the necessary steps to mitigate or address. You will critique clinical and scientific evidence and research and interpret how findings may impact BioMarin clinical development plans and publication plans.

Education and Experience

The ideal candidate will hold an MD, MD/PhD (or equivalent). You will have 2 or more years of relevant experience in genetic diseases, metabolic diseases, specialty care, and/or rare diseases.

This is an excellent opportunity to join our Clinical Science team and contribute to the development of transformative medicines for patients with significant unmet medical need. Salary range: $120,000 - $180,000 per year.