Lead, Research​/Development, Clinical Research

Bio Marin is a leading rare disease biotechnology company focused on genetically defined conditions. Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options.

More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions. Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world‑class manufacturing capabilities. At the heart of Bio Marin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

About Worldwide Research and Development:
From research and discovery to post‑market 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 first‑in‑class and best‑in‑class therapeutics that provide meaningful advances to patients who live with rare diseases.

The Real World Evidence Leads are part of the Integrated Evidence function comprised of subject matter experts in evidence planning, observational study design and data interpretation. The function works to drive excellence in evidence planning and implement novel study design approaches to generate robust evidence to enhance understanding of unmet medical need, support patient and endpoint selection for clinical studies and inform medical practice on appropriate use and long‑term safety and effectiveness of Bio Marin products to drive clinical adoption.

For one or more assigned programs or therapy areas, the Real World Evidence Lead (Director) is accountable for:

• Setting and leading the strategy of real‑world/observational evidence generation across assigned program(s) or therapy area(s), aligning to the Integrated Evidence Plan and key value, access, and regulatory decision points
• Leading implementation of real‑world evidence studies in‑line with a product’s Integrated Evidence Plan. Key activities include:
• Lead protocol development and reporting for disease understanding studies (retrospective and prospective), ensuring fit‑for‑purpose design to characterize target populations (risk factors, natural history, burden of illness, treatment patterns, comorbidities/comedications, outcomes, and unmet need) and to inform program decisions
• Lead the post‑marketing evidence strategy and delivery (including safety/effectiveness studies and registries), overseeing evaluation of risk mitigation strategies and ensuring alignment to risk management plans and real‑world regulatory commitments
• Establish and lead implementation of research plans with external collaborators (CROs, academia, data partners), working effectively to oversee external groups through the research process to ensure delivery of high‑quality outputs
• Define and oversee secondary data analysis strategy using Bio Marin clinical and real‑world data, ensuring analytic rigor and interpretation that directly supports program strategy and evidence gaps
• Lead interpretation and synthesis of real‑world evidence to deliver clear implications and recommendations for internal governance and senior stakeholders, and drive external scientific dissemination (manuscripts, abstracts, posters, presentations) consistent with publication strategy
• Serve as a strategic evidence partner to Commercial, Market Access, and Regulatory, ensuring real‑world evidence is appropriately reflected in forecasts, value narratives, and key regulatory deliverables (e.g., Orphan Drug Designation applications and regulatory strategy documents)

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