Associate Director, Patient Centered Outcomes Science

BioMarin Pharmaceutical Inc.

8d•Hybrid

About The Position

BioMarin is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Using our unparalleled expertise in genetics and molecular biology, we develop medicines for patients with significant unmet medical need. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options. About Worldwide Research and Development From research and discovery to post-market clinical development, our WWRD engine involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with genetic diseases. As part of the BioMarin Integrated Evidence Function in the Global Medical Affairs department, the Patient Centered Outcomes Science organization is responsible for the successful development and execution of end-to-end Patient Centered Outcomes (PCO) strategy— delivering evidence that meets regulatory, clinical, and patient access needs.

Requirements

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

Nice To Haves

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.

Responsibilities

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 subject matter expert (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
Work with program manager to develop PCOS risk mitigation plans (i.e., identify risks, scenario planning, facilitate cross-functional input to enable decision-making) for assigned programs
Coordinate PCOS-specific communication across cross-functional teams (CDT, IET, GRT, SET, AT)
Collaborate with PCOS leadership on strategic department and organizational strategy development and execution
Stay current on global best practices for PCOS strategy development and implementation
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
Drafting and finalization of PCOS components of interventional and non-interventional study protocols, and other qualitative and quantitative study protocols and Statistical Analysis Plans
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
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