Associate Director, Lysosomal Storage Disorder Programs

BioMarin Pharmaceutical Inc.
United States, California, San Rafael
770 Lindaro St (Show on map)
Jan 11, 2025
Who We Are 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. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. 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. With this distinctive approach to drug discovery, we've produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options. Our Culture From research and discovery to post-market clinical development, our WWRD engine 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 genetic diseases. Title: Medical Director, Lysosomal Storage Disorder (LSD) Programs, United States Location: Remote Who We Are 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. Global Medical Affairs (GMAF) is an expert scientific and medical function that develops and maintains non-promotional, evidence-based, transparent and balanced scientific and educational programs and resources in support of BioMarin's products and disease states of interest. We do this by assuring safe and appropriate product use, support of continuous scientific exchange and through ongoing and new data generation. The Role The US Lysosomal Storage Disorder (LSD) Medical Director is a critical strategic and execution focused role covering the following disease areas: MPS IVA (Vimizim), MPS VI (Naglazyme) and CLN2 (Brineura). The role reports into the US Medical Lead for PKU and LSDs 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 compound Act as the US product and disease area expert and primary contact point for all compound or disease area related issues both from within Medical Affairs and all other BioMarin departments Support internal and external communications related to diagnostic testing programs Cross Functional Leadership Represent Medical Affairs on relevant US Commercial teams including Brand Team Represent Medical Affairs in the Promotional Review Board (PRB) and provide medical review of promotional material to ensure maximum effectiveness 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 Launch and Pre-Launch Excellence 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 PMC/PMRs and studies falling under Medical Affairs scope in coordination with Global Study Operations Oversee clinical registries and evaluate safety issues associated with products and promotional activities for BioMarin products in accordance with respected regulatory requirements External Research Projects Presentation of US external research project proposals to the appropriate review 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 Learning and Development Medical Engagement Develop and strengthen key US medical expert relationships Contribute to the development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, tools are developed to support execution, and mechanisms are in place to measure impact Contribute to relevant publications on diagnostics, disease management and treatment guidelines Conceptualize and lead US medical advisory boards as needed as part of the US Medical Affairs strategy Review US Medical Information requests, standard response documents and FAQs Participate in, and contribute US perspective to scientific messaging discussions 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 solid 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 Medical Director Medical Sciences may have the opportunity to manage direct reports Education and Experience MD, PhD, or PharmD, or other advanced life science degree required LSD or genetics knowledge/experience ideal 3 or more years of relevant experience in clinical science, medical affairs, and/or drug development desired Note: This description is not intended to be all-inclusive, or a limitation of the duties of the position. It is intended to describe the general nature of the job that may include other duties as assumed or assigned. Equal Opportunity Employer/Veterans/Disabled An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability. In the U.S., the salary range for this position is $ 158,100 to $ 237,100 per year, which factors in various geographic regions. The base pay actually offered will take into account internal equity and also may vary depending on the candidate's geographic region, job-related knowledge, skills, and experience among other factors. A discretionary bonus and/or long-term incentive units may be provided as part of the compensation package, in addition to the full range of medical, financial, and/or other benefits, dependent on the level and position offered.