Director, Medical Affairs, US Fabry
Description
Who We Are
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with multiple commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.
About Research and Development
From research and discovery to post-marketing clinical development, our R&D engine involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing breakthrough medicines that provide meaningful advances to patients living with rare genetic conditions.
The US Fabry Medical Director is a critical strategic and execution focused role that reports into the US Medical Lead for Enzyme Conditions and represents Medical Affairs on core cross-functional teams.
Key Responsibilities:
Disease Area Strategy & Execution
- Develop and implement the US Medical Plan and lead US medical activities in coordination with Medical Affairs, Commercial and Regulatory colleagues
- Ensure adherence to US regulatory and clinical practice guidelines in supporting development and marketing of BioMarin compounds for Fabry
- Act as the US product and disease area expert and primary contact point for all compound or disease related issues both within Medical Affairs and for all other BioMarin department
- Contribute to the development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, development of tools to support execution, and mechanisms are in place to measure impact
- Conceptualize and lead US medical advisory boards as needed as part of the US Medical Affairs strategy
- Collaborate with Scientific Communications on relevant publications related to Fabry disease state and management
- Review US Medical Information requests, standard response documents and FAQs
- Contribute US perspective to scientific messaging discussions
Launch and Pre-Launch Excellence
Act as a key member on the Medical SMART team to provide medical recommendations and collaboration with the Clinical Development Team, Integrated Evidence Team, Asset Team and Integrated Brand Team
Develop US launch plans in collaboration with Medical Affairs and Commercial colleagues and execute on the US specific tactics in support of global product launches
Filing / Regulatory
Assist in preparation and review of relevant product BLAs or sBLAs and other regulatory documents including protocols, protocol amendments and briefing materials
Post Marketing Commitments
Lead for development/design of US post-marketing commitments, registries and studies falling under Medical Affairs scope in coordination with Global Study Operations
Active member of Study Engagement Teams (SET) related to ongoing Fabry post-marketing studies
Collaboration with pharmacovigilance to evaluate safety issues associated with products and promotional activities for BioMarin products in accordance with respected regulatory requirements
- Represent Medical Affairs on relevant US Commercial teams including Brand Team
Represent Medical Affairs in the Promotional Review Board (PRB) and Medical Material Review Committee (MMRC) to ensure maximum effectiveness of materials while adhering to the company's ethical standards and relevant Codes of Practice for the region
Provide medical oversight for Commercial international symposiums and advisory boards
External Research Projects
Presentation of US external research project proposals to SMART and External Research Committee for endorsement
Assist and collaborate with internal and external stakeholders in critically evaluating external research protocols, study results for statistical and final reports; Ongoing reviews with study investigators, resolving problems arising in the course of clinical investigations
Training
Support internal disease and product training to cross-functional colleagues in collaboration with Medical and Commercial Learning and Development
Desired Skills:
- The Medical Director is expected to exhibit mastery-level understanding of multiple competencies, including communication, scientific knowledge, medical diagnostics and management, as well advances skills in strategic thinking, problem solving, business acumen and drug development, regulatory and study design.
The Medical Director must be able to:
Be recognized as a highly capable facilitator of information sharing across all levels of the organization
Actively foster and maintain product strategy working relationships with key influential stakeholders inside and outside of BioMarin
Be the US subject matter expert, demonstrating deep medical, technical and scientific knowledge for relevant BioMarin therapeutic areas
Demonstrate understanding of cross-functional inter-dependencies across the drug development life cycle
Ability to support study design and protocol development for pre and post marketing studies
People Management
The US Fabry Medical Director may have the opportunity to manage direct reports
ducation and Experience
MD, PhD, or PharmD, or other advanced life science degree required
Fabry knowledge/clinical experience ideal
- 3 or more years of relevant experience in clinical science, medical affairs, and/or drug development desired
Equal Opportunity Employer