Ultragenyx Pharmaceutical Inc. Remote USA Full-time

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. During the COVID-19 pandemic, we are committed to the health and safety of our employees and prospective employees, which means at this time our interview processes may include virtual discussions, interviews, and onboarding. Please note that compliance with local regulations and safety protocols is a condition of employment at Ultragenyx. Elements of safety protocols may include, but are not limited to: work from home requirements, regular or symptom-based covid testing, providing valid evidence of vaccination, mask wearing, and physical distancing.
US based roles: Full vaccination against COVID-19 is a condition of employment at Ultragenyx. This requirement applies to almost all roles at Ultragenyx based in the U.S., with very limited exceptions. If you are unable to receive a COVID-19 vaccine due to a disability, serious medical condition, or because it would violate your sincerely held religious belief, you will have an opportunity to request a reasonable accommodation. 
Roles based outside the US:  If the role requires travel to the US or another country that requires evidence of covid vaccination for entry, candidates and employees will be required follow the local regulations and corporate policy regarding vaccination evidence submission.
Position Summary:
ultracurious – Apply your biggest ideas in courageous ways
Ultragenyx Pharmaceutical, Inc. seeks an experienced, energetic and highly motivated advocate who will actively engage with patients, families and care partners battling serious, debilitating genetic diseases, and the local, national and global advocacy organizations that represent them. Patients are at the center of Ultragenyx’s mission to bring to market novel products for the treatment of rare and ultra-rare diseases. This individual will be a part of a growing patient advocacy organization working across several diseases to communicate the enormous potential of Ultragenyx’s ongoing clinical programs and its powerful discovery and drug development platform.
Externally, the Senior Director of Patient Advocacy & Patient Engagement will develop productive partnerships and facilitate dialogue with patient advocacy groups to further mutual interests and transform the care of patients. Internally, this role will be responsible for incorporating the patient voice into the everyday operations of the company, nurturing Ultragenyx’s resolute sense of urgency and patient-focused culture. The Senior Director will report to the Vice President, Global Patient Advocacy & Patient Engagement.
Engagement with patients and patient advocacy groups is a critical success factor for the discovery, development and approval of investigational therapeutic programs in rare and genetically defined diseases. The Senior Director of Patient Advocacy & Patient Engagement will partner closely with internal partners to incorporate patient insights into Ultragenyx’s programs, decision-making and communications. The Senior Director is involved in the development and implementation of the internal and external patient engagement and experience strategy required to support clinical-stage programs, and ultimately, support commercialization and launches in multiple rare, genetically defined diseases.
Work Model:
Remote: officially documented as working full time from home, with travel to Ultragenyx’s offices or other location on occasion as needed


  • Responsible for developing and implementing cross functional strategic plan for patient advocacy engagement in pre-commercialization stage therapeutic areas through commercialization
  • Represent Ultragenyx and serve as the primary interface with rare disease communities
  • Lead relationship development with patient advocacy organizations to ensure productive ongoing partnerships based upon alignment between unmet needs of patient communities and UGX mission and priorities
  • Manage critical relationships with internal stakeholders through the compliant exchange of complex scientific information related to Ultragenyx areas of research, product development and commercialization
  • Engage with patients, families and care partners throughout discovery and development to inform key activities, including but not limited to, trial design and recruitment, engagement with regulatory agencies, and access and reimbursement
  • Interface with cross-functional teams to develop and drive corporate objectives and priorities for multiple programs that may include AAV gene therapy, mRNA, small molecule and biologic platforms
  • Identify and engage in internal opportunities to elevate the clinical and development value of inclusion of the patient experience in support of Ultragenyx’s patient-focused approach and culture
  • Participate in the measurement, reporting and impact of engagement with patients, families, care partners and patient advocacy groups
  • Assist in updating and informing the internal organization on patient advocacy trends, news and disease-specific development, while incorporating these opportunities and developments into Ultragenyx patient advocacy and engagement strategies as appropriate
  • Manage budget and finance process for portfolio
  • Ensure compliance with all internal and external guidelines


  • 12+ years within the biotechnology/pharmaceutical industry 
  • 10+ years of patient advocacy experience
  • Bachelor’s degree is required; advanced degree is desired 
  • Strong understanding of Patient Focused Drug Development from early transitional research through commercialization
  • Experience with working with Patient Advocacy Groups on public policy issues
  • Experience in managing a team
  • Able to think strategically, with extensive tactical planning and execution experience
  • Ability to summarize and communicate complex medical and scientific information using terms and concepts that are most relevant, understandable, and appropriate for rare disease communities
  • Rare Disease experience is preferred
  • Extensive knowledge of HIPAA and all appropriate regulatory, compliance and legal requirements
  • Ability to travel 25%