Real World Evidence Lead
BioMarin Pharmaceutical Inc.
August 31, 2026
Remote friendly (San Rafael, CA)
United States
Market Access
Key Responsibilities:
- Set and lead the strategy for real-world/observational evidence generation across assigned program(s) or therapy area(s), aligned to the Integrated Evidence Plan and key value, access, and regulatory decision points.
- Lead implementation of real-world evidence studies in line with the Integrated Evidence Plan, including:
- Lead protocol development and reporting for disease understanding studies (retrospective and prospective) to characterize target populations and inform program decisions (risk factors, natural history, burden of illness, treatment patterns, comorbidities/comedications, outcomes, unmet need).
- Lead post-marketing evidence strategy and delivery (safety/effectiveness studies and registries), evaluating risk mitigation strategies and ensuring alignment to risk management plans and real-world regulatory commitments.
- Establish and lead research plans with external collaborators (CROs, academia, data partners) to ensure high-quality outputs.
- Define and oversee secondary data analysis strategy using BioMarin clinical and real-world data with analytic rigor.
- Interpret and synthesize real-world evidence for internal governance and senior stakeholders; drive external scientific dissemination (manuscripts, abstracts, posters, presentations).
- Partner with Commercial, Market Access, and Regulatory to reflect real-world evidence in forecasts, value narratives, and regulatory deliverables (e.g., Orphan Drug Designation applications).
Desired Experience/Qualifications:
- Academic training and practical experience in Epidemiology.
- Options: PhD/ScD/DrPH or MPH in Epidemiology/Pharmacoepidemiology + 8+ years epidemiologic research in pharma; or PhD/ScD/DrPH or Master’s in relevant discipline (health services/outcomes research, public health, statistics, pharmaceutical sciences) + 10+ years in pharma.
Skills (Required/Preferred):
- Leadership in pharmacoepidemiologic and real-world evidence research; accountability for study strategy and delivery.
- Ability to translate integrated evidence strategy into study concepts/protocol portfolios.
- Experience authoring/owning protocol concepts, protocols, statistical analysis plans, and study reports.
- Expertise in observational study design and advanced analytics (bias/confounding mitigation, data quality, fit-for-purpose inference).
- Knowledge of healthcare data standards/ecosystems (HL7, FHIR), clinical terminologies (ICD-10), EMR/claims, and governance/privacy (GDPR, HIPAA).
- Ability to lead regulator/ethics engagements (HA queries, IRB/EC).
- Stakeholder management and operational discipline in a matrix environment.
- Translate complex evidence into clear internal recommendations and external dissemination.
- Preferred: rare disease and/or genetic epidemiology experience with small populations, registries, novel endpoints.
- Set and lead the strategy for real-world/observational evidence generation across assigned program(s) or therapy area(s), aligned to the Integrated Evidence Plan and key value, access, and regulatory decision points.
- Lead implementation of real-world evidence studies in line with the Integrated Evidence Plan, including:
- Lead protocol development and reporting for disease understanding studies (retrospective and prospective) to characterize target populations and inform program decisions (risk factors, natural history, burden of illness, treatment patterns, comorbidities/comedications, outcomes, unmet need).
- Lead post-marketing evidence strategy and delivery (safety/effectiveness studies and registries), evaluating risk mitigation strategies and ensuring alignment to risk management plans and real-world regulatory commitments.
- Establish and lead research plans with external collaborators (CROs, academia, data partners) to ensure high-quality outputs.
- Define and oversee secondary data analysis strategy using BioMarin clinical and real-world data with analytic rigor.
- Interpret and synthesize real-world evidence for internal governance and senior stakeholders; drive external scientific dissemination (manuscripts, abstracts, posters, presentations).
- Partner with Commercial, Market Access, and Regulatory to reflect real-world evidence in forecasts, value narratives, and regulatory deliverables (e.g., Orphan Drug Designation applications).
Desired Experience/Qualifications:
- Academic training and practical experience in Epidemiology.
- Options: PhD/ScD/DrPH or MPH in Epidemiology/Pharmacoepidemiology + 8+ years epidemiologic research in pharma; or PhD/ScD/DrPH or Master’s in relevant discipline (health services/outcomes research, public health, statistics, pharmaceutical sciences) + 10+ years in pharma.
Skills (Required/Preferred):
- Leadership in pharmacoepidemiologic and real-world evidence research; accountability for study strategy and delivery.
- Ability to translate integrated evidence strategy into study concepts/protocol portfolios.
- Experience authoring/owning protocol concepts, protocols, statistical analysis plans, and study reports.
- Expertise in observational study design and advanced analytics (bias/confounding mitigation, data quality, fit-for-purpose inference).
- Knowledge of healthcare data standards/ecosystems (HL7, FHIR), clinical terminologies (ICD-10), EMR/claims, and governance/privacy (GDPR, HIPAA).
- Ability to lead regulator/ethics engagements (HA queries, IRB/EC).
- Stakeholder management and operational discipline in a matrix environment.
- Translate complex evidence into clear internal recommendations and external dissemination.
- Preferred: rare disease and/or genetic epidemiology experience with small populations, registries, novel endpoints.