Director, Medical Affairs, US Fabry

BioMarin Pharmaceutical Inc.

About The Position

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. This role involves developing and implementing the US Medical Plan, leading US medical activities, and serving as the primary product and disease area expert. The position requires collaboration with various departments, including Commercial, Regulatory, and Scientific Communications, to ensure adherence to guidelines, develop engagement strategies, and manage medical information requests. The role also plays a key part in launch and pre-launch activities, post-marketing commitments, and external research projects, including study design, protocol development, and investigator collaboration. Additionally, the Medical Director will support internal training and may have direct reports.

Requirements

Mastery-level understanding of communication, scientific knowledge, medical diagnostics and management
Advanced skills in strategic thinking, problem solving, business acumen and drug development, regulatory and study design
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
MD, PhD, or PharmD, or other advanced life science degree required
3 or more years of relevant experience in clinical science, medical affairs, and/or drug development desired

Nice To Haves

Fabry knowledge/clinical experience ideal

Responsibilities

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
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
Assist in preparation and review of relevant product BLAs or sBLAs and other regulatory documents including protocols, protocol amendments and briefing materials
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
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
Support internal disease and product training to cross-functional colleagues in collaboration with Medical and Commercial Learning and Development