Real World Evidence Lead

Description

Who We Are 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 (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 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 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

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

• Setting and leading the strategy of 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. 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 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, 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)

Desired Experience A combination of academic training and practical experience in Epidemiology is required. This may consist of:

• Doctoral degree (PhD, ScD, or DrPH) 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 (PhD, ScD or DrPH) or Master’s degree in a relevant discipline (ie. Health services research, health outcomes research, public health, statistics, or pharmaceutical sciences) 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 (or in support of) the pharmaceutical/biotechnology industry, with accountability for study strategy and delivery
• Proven ability to translate integrated evidence strategy into executable study concepts and protocol portfolios, ensuring alignment to program priorities and measurable decision needs
• Extensive experience authoring and/or providing final scientific accountability for protocol concept documents, study protocols, statistical analysis plans, and study reports (including directing contributions from matrix partners and vendors)
• Deep expertise in observational study design and advanced analytic methods, with strong grasp of bias/confounding mitigation, data quality assessment, and fit-for-purpose inference
• Strong understanding of healthcare data ecosystems and standards (e.g., HL7, FHIR), clinical terminologies (e.g., ICD-10), EMR/claims data, and data governance/privacy requirements (e.g., GDPR, HIPAA)
• Demonstrated capability to lead high-stakes engagement with regulators and ethics bodies (e.g., HA queries, IRB/EC questions), shaping responses that protect scientific integrity, timelines, and program objectives
• Ability to set priorities and drive delivery in a business-driven, matrix environment—balancing multiple workstreams through strong planning, stakeholder management, risk management, and operational discipline
• Proven ability to build credibility and influence at all levels (including senior leadership), with the ability to translate complex evidence into clear implications and recommendations for internal decision-making and external scientific dissemination
• Experience in rare disease epidemiology and/or genetic epidemiology, including working with small populations, registries, and novel endpoints (preferred)

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.