Real World Evidence Lead

BioMarin 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, PKU, CLN2, and various forms of MPS 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 BioMarin 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 (RWE) 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 BioMarin products to drive clinical adoption.

Key Responsibilities:

• Setting and leading the strategy for 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.
• Lead protocol development and reporting for disease understanding studies, ensuring fit-for-purpose design to characterize target populations and to inform program decisions.
• Lead the post-marketing evidence strategy and delivery, 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, 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 BioMarin 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.
• 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.

Desired Experience:

• A combination of academic training and practical experience in Epidemiology is required.
• Doctoral degree or Master’s of Public Health in Epidemiology/Pharmacoepidemiology and at least 8 years of experience leading epidemiologic research in the pharmaceutical industry.
• Doctoral degree or Master’s degree in a relevant discipline with at least 10 years of experience leading epidemiologic research in the pharmaceutical setting.

Skills:

• Demonstrated leadership in pharmacoepidemiologic and real-world evidence research within the pharmaceutical/biotechnology industry.
• Proven ability to translate integrated evidence strategy into executable study concepts and protocol portfolios.
• Extensive experience authoring and/or providing final scientific accountability for protocol concept documents, study protocols, statistical analysis plans, and study reports.
• Deep expertise in observational study design and advanced analytic methods.
• Strong understanding of healthcare data ecosystems and standards.
• Demonstrated capability to lead high-stakes engagement with regulators and ethics bodies.
• Ability to set priorities and drive delivery in a business-driven, matrix environment.
• Proven ability to build credibility and influence at all levels.
• Experience in rare disease epidemiology and/or genetic epidemiology, including working with small populations, registries, and novel endpoints (preferred).

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.

An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, colour, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.