Why Join Us?
Be a hero for our rare disease patients
At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them.
Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work.
If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team.
Position Summary:
ultrafocused – Work together to fearlessly uncover new possibilities
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.
Work Model:
Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.
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
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
#LI-CS1 #LI-Remote
Full Time employees across the globe enjoy a range of benefits, including, but not limited to:
· Generous vacation time and public holidays observed by the company
· Volunteer days
· Long term incentive and Employee stock purchase plans or equivalent offerings
· Employee wellbeing benefits
· Fitness reimbursement
· Tuition sponsoring
· Professional development plans
* Benefits vary by region and country
Ultragenyx Pharmaceutical is an equal opportunity employer and prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status, and any other status or classification protected by applicable federal, state, and/or local laws. Reasonable accommodation will be provided for all protected statuses or classifications protected by applicable law, including individuals with disabilities, disabled veterans, for pregnancy, childbirth, and related medical conditions, and based on sincerely held religious beliefs. Applicants can request an accommodation prior to accepting a job offer. If you require reasonable accommodation in completing this application, or in any part of the recruitment process, you may contact Talent Acquisition by emailing us at [email protected].
It is unlawful in Massachusetts to require or administer a lie detector test as a condition of employment or continued employment. An employer who violates this law shall be subject to criminal penalties and civil liability.
Note to External Recruiters: All candidate activity and open positions are managed strictly through our Human Resources Department. Our Human Resources Department kindly requests that recruiters not contact employees/hiring managers directly in an attempt to solicit business and present candidates. Please note that failure to comply with this request will be a factor in determining a professional relationship with our organization. Submission of unsolicited resumes prior to an agreement set in place between the Human Resources Department and the recruiting agency will not create any implied obligation. Inquiries on developing a recruiting relationship with us, may be directed to
: [email protected]. 