Medical Director, Medical Affairs, Inborn Errors of Metabolism (IEM)

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 physician leader with the passion and creativity to develop gene therapies for patients with rare and ultra-rare diseases. Reporting to the Sr. Director, Global Medical Affairs Strategy – Gene Therapy, the Medical Director will be a key contributor to shaping strategy and implementing the medical affairs plans for various gene therapies, including within the inborn errors of metabolism therapeutic area. Furthermore, the Medical Director will be responsible for supporting development of an integrated evidence plan, forging strong relationship with key opinion leaders, supporting payer-related activities and educational events, and partnering cross-functionally with Medical Affairs colleagues, Commercial, Clinical, and Patient Advocacy.        

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 strategic medical and scientific leadership to assigned program(s) to support development of the global medical strategy
• US Launch: Support the execution of the medical launch plan for the relevant programs.
• Support development of the integrated evidence generation plan for assigned program(s), including input to Disease Monitoring Programs and leading data generation activities
• Support the execution of the relevant medical affairs activities including advisory boards, medical education initiatives, congress activities, and payer interactions
• Provide medical expertise across the organization to compliantly assist in the development and execution of commercial and clinical development activities
• Participate in scientific engagement and maintain strong relationships with external stakeholders (including healthcare professionals, clinical investigators, and societies) to generate insights and to educate and advance scientific understanding of relevant disease area
• Provide scientific and medical leadership as needed for publication development and serve as medical affairs reviewer for promotional materials and non-promotional scientific materials
• Provide support for Medical Science Liaison activities. This will include training as well as development of materials
• Partner cross functionally within medical affairs on assigned programs with all key stakeholders e.g., regional medical teams, US field medical, training, health economics and outcomes research, molecular diagnostics, medical services, patient find, patient advocacy, commercial, regulatory, clinical development and other related departments
• Exhibit leadership skills within and outside of the organization (including collaboration, mentoring, presentation skills and professional interaction)

Requirements:

• Medical Doctor (MD, DO) with Board certification (or equivalent) in Biochemical/Metabolic Genetics or Endocrinology (required)
• Experience in the management of patients with inherited metabolic disorders
• At least 2 years of experience in the pharmaceutical industry is strongly preferred, with Medical Affairs experience being ideal; however, extensive relevant clinical experience may be considered as an alternative
• Clinical or industry experience in gene therapy is preferred
• Comfortable with a