Associate Director, Patient Centered Outcomes Science

Role Summary

Lead end-to-end Patient Centered Outcomes (PCO) strategy and evidence generation across development to post-market for BioMarin products. Collaborate with cross-functional partners to deliver data that meets regulatory, clinical, and patient access needs. Manage development and deployment of COA tools and PCOS studies. Location: Remote or Hybrid in SRC (San Rafael Campus) and London.

Responsibilities

• Strategy & Planning
  • Collaborate with key cross-functional stakeholders (e.g., clinical, medical, regulatory, advocacy, commercial, global study operations, data science) to lead PCOS data and evidence generation planning and execution to meet cross-functional stakeholder needs
  • Identify, select, and implement fit-for-purpose COA tools in the interventional and non-interventional studies to support regulatory approval, labeling claims, and commercial success
  • Drive de novo COA tool development to fill evidentiary gaps
  • Contribute as key SME for qualitative and quantitative observational studies
  • Prospectively plan for and deliver on key PCOS projects (qualitative, quantitative; COA, DHT, Patient Preference Information) to meet stage-gate, regulatory, and commercial milestones throughout the product lifecycle
  • Lead PCOS publication planning strategy for PCOS-led activities
• Operational Execution
  • Develop and integrate PCOS strategy into the CDP, IEP, and TPP
  • Lead development of robust clinical outcome assessment (COA) endpoint strategy to support clinical development plans for assigned early- and late-stage programs
  • Lead PCO strategy to be incorporated in the TPP
  • Produce comprehensive PCOS Plans that are included in the IEPs
  • Ensure fit-for-purpose clinical outcome assessment (COA) tools are selected and implemented in clinical trials and observational studies and that the study design and data analysis plans are optimized for interpretability
  • Oversee PCOS project execution quality – propose, facilitate and implement plans to resolve issues and execute corrective actions
  • Lead development strategy for de novo COA tool development
  • Lead the design and execution of quantitative and qualitative PCO studies:
    • Select and manage vendor bidding process
    • Partner with legal, finance, and compliance teams to execute contracts and manage book of work
    • Oversee CRO teams
    • Oversee drafting and finalization of study materials, qualitative analysis plans (QAPs), statistical analysis plans (SAPs) and Psychometric Analysis Plans (PAPs)
    • Oversee development of final study reports and dissemination deliverables
  • Act as key SME:
    • Drafting and finalization of PCOS components of interventional and non-interventional study protocols, and other qualitative and quantitative study protocols and SAPs
    • Authoring and reviewing PCOS-relevant sections of regulatory submission materials, site and KOL training materials, investigator and ad board meeting materials, study handbooks and other materials ensuring data and messaging accuracy and alignment
    • Supporting PCOS strategy development and implementation for natural history and RWE evidence studies
  • Organize and drive cross-functional working groups and teams to meet PCOS strategic objectives across the portfolio
• Cross-functional Influence
  • Represent PCOS on cross-functional teams and sub-teams
  • Communicate PCOS information and activities to internal and external stakeholders (including senior leadership)
  • Help develop and disseminate the most current PCO best practices throughout the organization (via email, presentations, desk procedures, reference documents and repositories) to foster enrichment and education of cross-functional stakeholders
  • Represent PCOS on internal and external scientific and advocacy working groups
  • Partner with various stakeholders to transition products from development to marketed stage and to fulfill post-marketing requirements
  • Within cross-functional teams, foster a culture that promotes respect, teamwork, collaboration, openness, and appreciation

Qualifications

• A Master’s or doctoral degree in the field of Epidemiology, Public Health, Psychology, Psychometrics, Health Research, clinical (medicine, pharmacy or nursing) or related is required
• 5 + year experience with PhD; 8+ years with Master’s
• Experience in Patient Focused Drug Development, including Outcomes Research or other related function at a global pharmaceutical or biotechnology company, preferred. Experience interacting with regulators, policy makers, and experts in the field is preferred.