Senior Director, Evidence Generation

Ultragenyx Pharmaceutical

1d•$254,200 - $314,000•Remote

About The Position

Ultragenyx is seeking a highly experienced and strategic Senior Director, Evidence Generation to lead global post-approval and late-stage evidence strategy across the company’s rare disease portfolio. Working in close partnership with Global Medical Leads, this leader will define and execute integrated evidence plans that support patient access, inform clinical practice, and strengthen the scientific understanding of real-world disease and treatment outcomes. The successful candidate will bring deep expertise in rare/ultra-rare disease research, strong experience in real-world evidence and non-interventional methodologies, and a proven ability to lead complex, cross-functional evidence generation programs.

Requirements

Advanced degree (PhD, PharmD, or equivalent)
12+ years of experience in Medical Affairs, Clinical Development, Epidemiology, HEOR, or Evidence Generation
Proven experience designing and executing real-world evidence, observational studies, registries, or non-interventional research
Deep expertise in integrated evidence planning and evidence generation strategy
Strong background in rare diseases or small population research preferred
Demonstrated leadership of cross-functional global teams
Experience establishing governance models, operating frameworks, and scalable processes
Expertise in externally sponsored research (ISR/IIR/CR) programs and partnerships
Strong analytical, strategic thinking, and scientific communication skills
Experience aligning evidence generation with medical affairs, regulatory, HTA, and market access needs

Responsibilities

Define and lead the framework, standards, and operating model for integrated evidence generation, including processes and cross-functional ways of working, to enable consistent, high-quality development and execution of Integrated Evidence Plans (IEPs) across programs
Partner with Global Medical Leads and cross-functional teams to support development of IEPs and align on medical evidence generation approaches
Translate IEP priorities into execution-ready evidence generation plans, including study concepts, protocols, data sourcing strategies, analytical approaches, and input into Disease Monitoring Programs (DMPs)
Lead and coordinate evidence generation activities (e.g., RWE studies, registries, chart review studies, natural history studies) and contribute to DMP strategy and design, in collaboration with cross-functional stakeholders, clarifying roles and responsibilities for execution as operating models evolve
Serve as an internal subject matter expert on real-world data (RWD) and real-world evidence (RWE), providing strategic guidance on data source selection, methodological approaches, and application of RWD/RWE to inform evidence generation and decision-making
Establish and advance structured data planning approaches and enable selection of fit-for-purpose data sources (e.g., EMR, claims, registries, PROs, and DMP-derived data), ensuring consistency, quality, and utility of data across programs
Lead and guide the analysis, interpretation, and dissemination of data generated from prioritized evidence generation activities, including DMP outputs, to support publications, scientific communications, and post-marketing and real-world evidence activities supporting regulatory, payer, and clinical decision-making
Implement and evolve enterprise-level processes and KPIs to support prioritization, tracking, and delivery of evidence generation activities, and partner cross-functionally (Clinical, HEOR, Regulatory, Market Access) and with external researchers and academic partners to ensure alignment with IEP priorities and evidence generation objectives; participate as a subject matter expert in IST/IIR/CR review processes