Position Summary:

ultradedicated – Your biggest challenges yield rare possibilities

Ultragenyx is seeking a physician with experience in clinical development to lead the ongoing clinical development of a gene therapy treatment for Wilson’s disease. The individual will be a scientifically minded, curious, collaborative, team leader who will be creative and resourceful in addressing clinical, scientific and operational challenges for an ongoing clinical trial. This individual will conduct medical monitoring, interpret clinical data, contribute to study design and clinical development strategy, participate in regulatory interactions, and lead the clinical development team. This person will establish long-lasting relationships with study investigators, CRO partners, and patient organizations.

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:

• Create and refine the clinical development plan
• Lead the team to execute the clinical development plan
• Perform medical monitoring for ongoing clinical trial(s)
• Provide medical and scientific input in review of clinical data, patient medical safety data, and laboratory values; maintain an ongoing assessment of the safety profile and efficacy data.
• Support study execution, including review and cleaning of study data, updating study protocols, reviewing consent forms and other patient-facing materials, reviewing electronic case report forms, reviewing periodic safety reports, and training study sites
• Formulate clinical responses to regulatory, institutional review board (IRB) and ethics committee (EC) inquiries
• Exhibit leadership skills within and outside of the organization (including people skills, mentoring, presentation skills and professional interaction). Inspire a cross-functional team to promote high performance and keep team members focused on the highest priority activities for study
• Critical review and scientific contribution to clinical study reports and manuscripts
• Work with the clinical operations and finance teams to assist with the production of budgets and timelines for clinical trials.
• Participate in pre-submission, pre-market and other meetings with the FDA and other regulatory agencies, as needed

Requirements:

• MD or MD/PhD graduating from a high-quality medical school
• Originally boarded in a medical specialty, optimally in gastroenterology, hepatology, medical biochemical genetics, or other complex fields dealing with rare, chronic diseases and complex pathophysiology
• At least 2-3 years of industry experience or leadership of industry-sponsored clinical trials
• Trained in GCP regulations, familiar with ICH and FDA guidelines relevant to clinical development
• Experience designing successful clinical studies in complex diseases requiring the management of patient variation, chronic irreversible disease and related complexities.
• Facile with statistical issues and be able to manage working with a statistician to design the analysis of clinical studies
• Travel to study sites, team meetings, scientific conferences, and patient organization meetings approximately 1-2 times per quarter
• Ability to integrate and interpret complex clinical data and present them succinctly and clearly to an audience
• Actively listen to and value others’ thoughts and ideas. Demonstrate effective partnership both within and outside your team. Achieve results with and through others.
• Demonstrate self-awareness, humility, and curiosity in service of self-development. Provide regular coaching and feedback that will inspire growth and improvement in others. #LI-CS1 #LI-Remote