Ultragenyx

Director

• Lead the Global Medical Sub Team (GMST), ensuring regional medical affairs are involved in and inform the development of global medical strategy
• Develop and drive the timely execution of the Global Medical Plan for the bone franchise assets across pre-launch, launch, and post-launch phases
• Serve as the strategic medical leader on the Program Core Team and related sub teams, providing expertise to shape development, commercialization, and lifecycle decisions, taking a “hands on” approach to proactively identify and resolve challenges
• Lead Key Opinion Leader advisory boards, steering committees, and investigator meetings to gather key insights that inform program strategy and activities
• Lead updates of the Integrated Evidence Generation Plan (IEP), ensuring alignment with HEOR and clinical development to address global/regional evidence gaps
• Lead and successfully complete global medical affairs data generation activities, including real-world evidence (RWE), investigator-sponsored trials (ISTs), and other strategic evidence initiatives
• Accountable as the medical affairs lead for Disease Monitoring Programs for rare bone disease, providing strategic medical leadership on all aspects of the studies and accountable for leading data generation and publication activities aligned to the IEP
• Provide deep disease area and product expertise, ensuring accurate and consistent communication of clinical and scientific data
• Lead global publication strategy and congress planning activities, ensuring timely, high-impact dissemination of data and other education initiatives
• Ensure development of impactful core scientific resources (slide decks, FAQs, educational materials) for adaptation by regional teams
• Provide strategic guidance, tools, and training to regional and affiliate medical affairs teams to ensure launch readiness and consistent scientific exchange, and lead internal scientific training for the programs
• Build and maintain relationships with global key opinion leaders (KOLs), societies, and advocacy groups to inform strategy and amplify scientific reach
• Accountable for the medical assessment of the competitor environment and providing direction on strategic implications
• Collaborate effectively with cross-functional partners including clinical development, HEOR, diagnostics, scientific communications and publications, regulatory, market access, commercial, training, and patient advocacy

Senior Director, Global Medical Lead – Bone

• Provides data management oversight and support to project teams on all data management matters, including optimizing overall performance, generic systems and procedures
• Collaborates cross-functionally to lead program deliverables to support program objectives
• Accountable for the quality and timely completion of project deliverables and responsible for multiple or broad areas of significant impact
• Accountable for strategy and functional management across assigned critical programs through resourcing (including contingent workers) and individual contributions
• Leads data management efforts within assigned key company programs
• Identifies and leads critical DM process improvement initiatives
• Identifies, evaluates and selects DM vendors; establishes and manages key vendor partnerships
• Leads vendor qualification and oversight audits as DM SME as needed
• Ensures compliance with regulatory and industry process and quality standards and represents the function in regulatory inspections
• Proactively identifies potential study or process issues/risks and spearheads solutions
• Provides leadership/oversight to ensure high quality CDM deliverables and study-related documents (e.g., eCRF and edit check requirements, Clinical Data Management Plan, CRF Completion Guidelines)
• Participates in the selection, training and evaluation of DM personnel (internal and contract) to ensure efficient and effective department function

Director, Clinical Data Management

• Provide DSPV leadership in support of development, registration, and life-cycle management for assigned Ultragenyx portfolio of products
• Guide, coach, and manage Safety Science professionals and their managers to ensure professional development and advancement
• Lead and contribute to safety aspects of interactions with regulatory authorities worldwide (FDA, EMA etc.) at type C, B, pre-BLA/MAA/NDS meetings, Scientific Advice, Advisory Committee meetings, or other scientific advice forums for portfolio products
• Provide DSPV leadership to support Ultragenyx Commercial organization global launch activities for portfolio products
• Manage pre- and post-marketing safety surveillance activities in compliance with ICH guidelines, Good Pharmacovigilance Practices (GVP), and applicable global health authority regulations, guidance and best practices
• Build partnerships with key senior stakeholders from other functions to ensure strategic and operational business goals are met
• Liaise and negotiate with global regulatory authorities on drug safety issues and to expedite approvals
• Maintain awareness of the global regulatory environment, interpret PV regulations, and assess impact of changes on business and PV systems
• Proactively manage critical and urgent safety issues and lead DSPV contributions for portfolio products
• Develop and implement department policies, processes, and SOPs
• Support inspection readiness activities, internal audits, and external inspections for Safety Science
• Contribute to the generation and maintenance of Pharmacovigilance Agreements (PVAs)

Executive Director, Safety Science – Global

Vice President

• Lead the Biostatistics/Epidemiology and Statistical Programming teams
• Ensure statistical integrity of clinical studies conducted to support projects across the portfolio
• Set strategy for the department, including enhancing technical expertise in support of drug development and enabling development of staff
• Build/drive relationships and teamwork across the Biostatistics and Programming teams in support of the programs ensuring proper statistical strategies, and understanding of priorities between the project teams and the teams
• Provide statistically/epidemiologically sound scientific methodology leadership in experimental design and data analyses
• Review statistical analysis sections of protocols, statistical analysis plans, study reports, regulatory documents or scientific publications, generates/reviews study sample size/power estimations
• Provide leadership in the interpretation and reporting of the results of statistical analyses
• Mentor lead statisticians/epidemiologists in presenting the statistical concepts, evidence, interpretations, and logical arguments to the project team and senior management
• Independently pursue analyses suggested by the data; Propose new/novel statistical methodological approaches leading to improvements in the efficiency and sensitivity of study results
• Contribute to the advancement of research in clinical trials methodology. Establish a healthy culture in the department, including collaboration and quality mindset
• Develop and evaluate options for meeting project team goals under time and budgetary constraints and can change course quickly
• Reporting to the CMO; sit on the CMO’s leadership team and play a critical role in the biostatistical strategic planning for all clinical programs at the company

Senior Vice President, Biostatistics and Statistical Programming

Manager

• Accountable for the overall success of Data Management for their program(s).
• Operational leadership and support of data management activities for one or more programs.
• Represent data management on cross-functional team providing accurate study status updates and proactive communication/escalation of data management issues and risks.
• Development and maintenance of program level DM deliverable timelines in accordance with program development strategy; drive internal and external timeline negotiations.
• Management of data management vendors providing consistent team direction/guidance and monitoring quality of work through vendor audits and key performance metrics.
• Facilitate and perform cross-functional sponsor review meetings of eCRF design, edit check specifications and user acceptance testing.
• Facilitate the development of data transfer agreements with external data vendors (e.g., clinical laboratories, imaging vendors) ensuring data quality from data capture to reporting.
• Perform sponsor review of DM essential documents including annotated CRFs, eCRF Completion Guidelines, Clinical Data Management Plans and DB Go Live and Lock documentation.
• Contribute to the development, review and revision of department processes (e.g., SOPs, job aids, standards/templates) to improve quality, effectiveness and efficiency.

Manager, Clinical Data Management

Compliance

• Performs moderately complex to non-routine submission formatting activities associated with generating hard copy and electronic submission-ready documents and reports.
• Electronically compiles all components of moderately complex submissions using Lorenz DocuBridge™ and associated publishing tools including but not limited to: eCTDs - National and Centralized Procedures; Post Marketing/Life-cycle Management: Type Ia, Ib and II Variations & Renewals; Orphan Drug Documents, Scientific Advice and PIPs.
• Dispatches submissions through the relevant gateways/portals (e.g. EMA Submissions Gateway and US Gateway and/or other channels) and electronically archives them according to the departmental practices.
• Participates in supporting and promoting current routine electronic initiatives in moving the company forward with electronic submissions and electronic archives.
• May interface with cross-functional teams and or content authors to discuss routine submission preparation and content.
• May assist and/or provide training to others on software tools used and educating authors in publishing policies and procedures.
• Identifies and records issues that require resolution prior to finalization and liaises with the author responsible to resolve issues. Assists authors in the completion and compilation of regulatory documents to ensure all components are provided and presented in the correct format.
• Exercises judgment within well-defined procedures and practices to determine appropriate action.

Senior Associate, Global Regulatory Operations

Account Manager

• Responsible for the successful commercialization of Ultragenyx products in the assigned territory; accountable for attaining revenue and profitability goals
• Independently analyze market potential, treatment paradigms, decision-making and patient referral patterns in the territory
• Develop, execute and present account plans for key accounts in the territory
• Analyze effectiveness of commercial tactics and monitor changes in the health care system to identify new business opportunities
• Establish sustainable, mutually beneficial relations with opinion leaders and other key stakeholders; engage in regular exchange with peers to share best practices
• Optimize patient care by aligning decision makers involved in relevant decisions (specialists, nurses, pharmacists, payers)
• Develop and execute strategies to identify undiagnosed or misdiagnosed patients
• Support market access on a local level (regions and hospitals) to ensure patients have sustainable access to pharmaceutical innovation
• Field role: Officially documented as working as a member of the Ultragenyx field team, generally interacting with third parties on behalf of Ultragenyx

Key Account Manager

Clinical Operations

• Position Summary: The Director of Clinical Operations is accountable for the operational strategic planning, oversight and delivery for one or more assigned clinical programs.
• Work Model: Remote—Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.
• Lead the operational strategy and functional management for one or more clinical trial programs, in accordance with the Clinical Development Plans and program plans
• Accountable for the development and implementation of clinical development program strategies; planning horizon is typically 12-24 months
• Develop and present operational plans to management including risk management and mitigation
• Accountable for the development, implementation and execution of clinical trials within agreed timelines, resources and budgets (including proactive planning and management)
• Provide input on the development of protocols, Case Report Forms, Clinical Study Reports, and regulatory submissions (and all study related documents as needed)
• Represent Clinical Operations on the project teams, such as the Development Sub-Team, Core Teams (as applicable)
• Identify program risks, develop and implement mitigation strategies for assigned programs
• Manage and mentor Clinical Operations personnel
• Oversee vendor interactions (from selection through to close) and being a point of escalation for the operational functional activities and identify key risks. Experience working with internal partners (Vendor Management, Finance etc) in the overarching management of the vendors

Director, Clinical Operations

Financial Planning and Analysis

• Lead the development, implementation, and execution of the enterprise-level annual budget and forecast updates, long-range strategic financial plans (e.g., 5-year plan), and assessing and preparing for alternate scenarios, ensuring alignment with corporate strategy and business objectives • Partner with senior leadership across R&D, Commercial, Technical Operations, and Corporate Functions to develop robust financial models and provide strategic financial input for key decision-making, resource allocation, and investment prioritization • Oversee the preparation and presentation of detailed financial reports, analyses, and dashboards to senior management, the executive leadership team, and the Board of Directors, providing clear, concise, and actionable insights • Provide strong leadership in guiding the FP&A team to deliver high-quality financial analysis and insights that support strategic decision-making • Maximize use of current technology and drive adoption of new advanced technologies as needed • Challenge the status quo and identify opportunities for process optimization and efficiency within financial planning and analysis • Collaborate effectively with Accounting, Treasury, Investor Relations, Business Development, and other functional leads to ensure seamless financial operations and data integrity • Act as a key finance partner to business leaders, providing financial expertise and support for strategic initiatives • Serve as Finance representative to one or more of our project teams, providing analytical and strategic support

Senior Director, Corporate Financial Planning and Analysis

Medical writer

• The Medical Writer is responsible for writing and overseeing the writing of various clinical and regulatory documents. The position works closely with the cross-functional team to ensure the documents are high quality and completed in a timely fashion compliant with SOPs, ICH/GCP/regulatory guidelines, and company goals.
• The core duties of the medical writing manager are delineated below.
• 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:
• Write, edit, or collaborate on development of clinical and regulatory documents (e.g., protocols, investigator brochures, clinical study reports, INDs, eCTD/NDA clinical summaries, briefing books)
• Ensure smooth and effective document management from start to finish (i.e., from template to a final approved version), which may include, but is not limited to: first draft authoring, reference retrieval, generating bibliographies, editing (e.g., follow house style guide), and managing various processes (e.g., reviews, QC, QA, formatting, publishing, document archiving)
• Review, edit, and ensure quality of documents or sections of documents prepared by functional area representatives as required and ensure adherence to standards
• Distill large amounts of clinical and scientific data into essential elements for graphical display
• Partnering with lead author, oversee document review and comment resolution meetings with cross-functional teams
• Formulate key messages from clinical study data
• Perform literature-based research to support writing activities
• Develop and maintain templates and outlines for key documents