Position Summary:

ultraimpact – Make a difference for those who need it most

Ultragenyx is a leading fully integrated biotechnology company in rare disease development. We are seeking a physician-scientist with experience in clinical development to be a member of the expanding Neurology Therapeutic Area. This individual will have clinical neurology or pediatrics with neuroscience training with expertise in early clinical development and translational research. They will be curious and collaborative, creative and resourceful in addressing clinical, scientific and operational challenges for ongoing clinical studies and new INDs. This individual will conduct medical monitoring and participate in interpretation of clinical data for our late stage Angelman’s Syndrome program and contribute to study design and strategy for INDs entering clinical studies. This position will provide clinical development representation at team and regulatory interactions and responsible for establishing long-lasting relationships with key thought leaders, 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:

• 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
• Formulate first-in-human concepts and study protocol design and present to corporate governance for approval
• Author early clinical development plans and present to corporate governance for approval
• 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
• Originally boarded in neurology (peds or adult) or pediatrics with clear record of neuroscience research
• Academic scientific research in a related field
• Prefer 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 to collaborate 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