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Medical Director, Hemophilia Gene Therapy, Medical Sciences (TEMPORARY)

Location San Rafael, California
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 will continue to focus on advancing therapies that are the first or best of their kind.

BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development 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 rare diseases. Come join our team and make a meaningful impact on patients’ lives. 


The Medical Director is a critical role assisting with strategy and execution in the Medical Sciences function, leading North American Regional Medical Affairs activities for BioMarin's Hemophilia product from pipeline through post approval status.  Key product responsibilities include, but are not limited to, cross functional leadership, strategic alignment with commercial business partners, Medical Plan development, product launch strategy and execution, scientific message development and product scientific materials review, KOL relationship management and medical monitoring of post-approval commitments.


North American Product Strategy & Execution

•       Assist the Medical Lead, with the development and communication of the Global Medical Plan in collaboration with Medical Affairs stakeholders across BioMarin

•       Act as product and disease area expert and primary contact point for all global compound or disease area related issues both from within Medical Affairs and all other BioMarin departments

•       Identify, develop and maintain the scientific messaging platform with Medical Communications and other relevant stakeholders

•       Assist with the critical appraisal of available scientific and medical knowledge, identify gaps and identify and lead activities to address deficiencies

•       Review all product materials (dossiers, medical Information response documents, slide decks, etc.)

•       Assist with developing scientific messaging and gap analysis, as well as, communications regarding product or disease area

•       Contribute to the development of, review and approval of global publication plans

Cross Functional Leadership

•       Act as the Medical Affairs representative on key cross-functional product and development strategic focused teams

•       Collaborate with the Medical Affairs cross-functional Product Team

•       Represent Medical Affairs on relevant commercial teams: Launch Team, and Global Brand Teams for marketed products

Market relationship and access development

•       Manage contact with KOLs, particularly for Pipeline products, ensure appropriate access to KOLs during entire product lifecycle

•       Assist with international symposiums, advisory boards, manuscripts

Product Launch

•       Align with the Medical Affairs product launch strategy and roadmap and lead all Medical Affairs launch activities in collaboration with Commercial and other key stakeholders

Filing / Regulatory

•       Assist in writing medical need, natural history and risk/benefit sections of BLA and MAA

•       Assist with representation of Medical Affairs at FDA/EMA advisory board meetings


Skills Cont... 

Post Marketing – Registries, IR, observational

•       Participate in the development/design of PMC/PMRs and studies falling under Medical Affairs scope in coordination with Medical Affairs Study management

•       Act as medical monitor for PMR/PMC and studies falling under the Medical Affairs scope in collaboration with Medical Affairs Study management

•       Collaborate with clinical registries and evaluate safety issues associated with products and promotional activities for BioMarin products in accordance with respected regulatory requirements

•       Assist in critically evaluating study results in statistical and final reports and ongoing reviews with study investigators, resolving problems arising in the course of clinical investigations and preparing reports to the management and medical community

•       In collaboration with Regional Medical Product leadership and Area Medical Leads, conduct critical scientific and medical review of IR protocols


•       Support early disease training across the company. In collaboration with Medical Affairs learning and development department develop content and modules for training

The Medical Director is expected to exhibit strong understanding of multiple competencies, including communication and medical and scientific knowledge as well as, 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

·        Demonstrate medical and scientific knowledge for key BioMarin therapeutic areas

·        Demonstrate solid understanding of cross-functional inter-dependencies across the drug development life-cycle

·        Have ability to assist with the design of studies and develop protocols in a wide range of types of study phases pre and post marketing


·        MD with US licensure or fellowship required; BS in life or health sciences

·        1 or more years of industry experience in Hemophilia or genetics ideal

·        3 or more years of relevant experience in clinical science, medical affairs, and/or drug development desired

Skills and Experience

We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, sex, national origin, disability status, protected veteran status, or any other characteristic protected by law.

About our location

San Rafael


Hiring Insights

We treat rare diseases, and that gives us rare opportunities. Chris Brodeur, Director, Corporate Compliance
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