Sr Director Medical Affairs, Pipeline Lead

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:

ultradedicated – Your biggest challenges yield rare possibilities

Ultragenyx is seeking a dynamic and experienced leader to join the Global Medical Affairs team with responsibility for the pipeline portfolio. The Sr. Director, Pipeline Lead will provide strategic early medical affairs leadership across multiple early-stage assets in our neurology and inborn errors of metabolism portfolios, including, for example, GNE Myopathy, Creatine Transporter Deficiency, Wilson’s Disease, and Ornithine Transcarbamylase Deficiency. This role requires an adaptable leader willing to take a “hands on” approach in their work and who is comfortable working in a fast-paced intellectual environment.  

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:

• Provide early medical affairs leadership to assigned programs, serving as an active core member of the Program Team and decision-making sub-teams, and contributing to the progression of program milestones 

• Demonstrate and apply deep rare disease expertise across assigned programs, actively building and maintaining a global network of therapeutic area experts. Lead insight-generation activities, including scientific advisory boards and steering committees that inform program strategy  

• Lead the development of an aligned early-stage Integrated Evidence Generation Strategy for assigned programs, incorporating real world evidence generation, ensuring plans are strategically designed and executed to establish a robust foundational scientific evidence base 

• Lead proactive competitive landscape assessments and translate emerging science and therapeutic area dynamics into strategic recommendations for asset positioning 

• Establish and lead the Global Medical Sub Team(s), and drive the development and implementation of the medical strategy for assigned pipeline programs, aligned with program objectives and through close collaboration with global medical functions and regional medical leads 

• Partner with scientific and medical communications to guide development of the end-to-end scientific communication platform, including the global scientific narrative, ensuring a consistent external global scientific education strategy 

• Ensure timely and effective data dissemination by leading publication strategies, congress planning, and scientific education initiatives 

• Provide deep disease area and product expertise, ensuring accurate and consistent communication of clinical and scientific data, including sign off in review committees  

• Provide guidance, tools, and high-quality compliant training to US field and regional medical affairs teams to elevate consistent scientific exchange, and other internal teams as needed 

• Collaborate effectively with clinical development, HEOR, diagnostics, scientific