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
o 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
o 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
o drive de novo coa tool development to fill evidentiary gaps
o contribute as key sme for qualitative and quantitative observational studies
o 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
o lead pcos publication planning strategy for pcos-led activities
* operational execution
o develop and integrate pcos strategy into the cdp, iep, and tpp
o lead development of robust clinical outcome assessment (coa) endpoint strategy to support clinical development plans for assigned early- and late-stage programs
o lead pco strategy to be incorporated in the tpp
o produce comprehensive pcos plans that are included in the ieps
o 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
o oversee pcos project execution quality – propose, facilitate and implement plans to resolve issues and execute corrective actions
o lead development strategy for de novo coa tool development
o 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
o 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
o organize and drive cross-functional working groups and teams to meet pcos strategic objectives across the portfolio
* cross-functional influence
o represent pcos on cross-functional teams and sub-teams
o communicate pcos information and activities to internal and external stakeholders (including senior leadership)
o 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
o represent pcos on internal and external scientific and advocacy working groups
o partner with various stakeholders to transition products from development to marketed stage and to fulfill post-marketing requirements
o 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.
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