Executive Director, Global Medical Expert – Gene Therapies

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:

ultraimpact – Make a difference for those who need it most

Ultragenyx is seeking a dynamic and experienced physician leader to join the Global Medical Affairs team to lead the implementation of gene therapy programs at launch.  The leader will play a key role within the gene therapy portfolio, initially focused on Inborn Errors of Metabolism.  The company is launching over the next 2 years two gene therapies in the US requiring insightful management of clinical centers implementing gene therapy.

Reporting to the VP Global/US Medical Affairs and Strategy, this leader will partner closely with the US Field Medical, Global/US Medical Strategy Leads and Clinical Development through a largely externally facing role which requires peer-to-peer interactions with KOLs who are conducting gene therapy treatments.  It will also involve clinical and scientific education, evidence creation and dissemination and addressing information needs of physicians treating patients as well as patient organizations or others who may be stakeholders for commercialized Ultragenyx gene therapies. This in an innovative high-profile position that requires a “hands on” adaptable and clinically capable leader, comfortable working in a fast-paced intellectual environment, understanding patient care, immunomodulation and collaborating at the interface between clinical development and medical affairs leveraging their expertise to advance our mission to change the future of rare disease medicine.

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:

• Be a core member of the Medical Affairs Gene Therapy Treatment Team, collaborating closely with Clinical Development to proactively identify and resolve challenges, and compliantly deliver timely scientific information and education regarding the use of gene therapies to inform clinical decision-making. We need every site doing these critical therapies to be well prepared to conduct treatment and manage treated patients
• Collaborate closely with Global/US Medical Leads and US Field Medical team across the Inborn Errors of Metabolism (IEM) portfolio to ensure execution of medical plans by leading/supporting initiatives to expand the knowledge of KOLs and centers of excellence
• Provide the leadership both internally and externally to drive successful implementation of gene therapy and not just be a passive contributor
• Provide expertise and contribute as needed to key regulatory agency communication, including filings and information request responses
• Provide expertise and contribute as needed to protocol development, steering committee meetings, data generation initiatives of Disease Monitoring Programs
• Through partnership with Patient Advocacy, Government Affairs and Market Access teams, meet the engagement and education needs of stakeholders relevant to patient education, advocacy and access  
• Represent Ultragenyx at key scientific meetings to achieve conference strategy objectives including: leading engagement meetings with KOLs, gathering and sharing of strategic insights, executing education initiatives, presenting data 
• Stay abreast of internal and external developments, trends and dynamics and provide direction for strategic implications to the development and commercialization of gene therapy