Associate Director, Patient Centered Outcomes Science
Who We Are
For more than two decades, going our own way has led to countless breakthroughs, bettering the lives of those suffering from rare genetic disease. In 1997 we were founded to make a big difference in small patient populations. Now we seek to make an even greater impact by applying the same science-driven, patient-forward approach that propelled our last 25 years of drug development to larger genetic disorders, as well as genetic subsets of more common conditions. Through our unparalleled expertise in genetics and molecular biology, we will continue to develop targeted therapies that address the root cause of the conditions we seek to treat. Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most. The end goal has always been better lives and now we can reach more.
And the more people we reach, the more our impact can grow. We transform lives through genetic discovery.
Our desire to make a positive impact on our patients extends to our employees and BioMarin is committed to fostering an inclusive environment where every person feels seen, valued, and heard – so employees can thrive in all areas of their lives, in and outside of work. We seek to provide an open, flexible, and friendly work environment to empower people and to provide them with the ability to develop their long-term careers. Ultimately, we want to be an organization where people enjoy coming to work and take pride in our efforts to help patients.
BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class or best-in-class therapeutics that provide meaningful advances to patients. Come join our team and make a meaningful impact on patients’ lives.
As part of the LCD organization, the Patient Centered Outcomes Science (PCOS) team is responsible for leading and ensuring successful development and execution of patient centered outcomes and endpoint strategy in early and late-stage clinical trials and observational studies to support the product lifecycle.
Strategy and Planning
- Oversee the development and implementation of the PCOS strategy to support the overall clinical program strategy, product approval, and commercial success
- Collaborate with key cross-functional stakeholders (e.g., clinical, medical, regulatory, advocacy, commercial) to:
- Identify, select, and implement fit-for-purpose COA tools in the clinical development plan (CDP) to support regulatory approval, labeling claims, and commercial success
- Lead annual program and budget planning in support of corporate objectives
- Prospectively plan for and deliver on key PCOS workstreams to meet stage-gate, regulatory, and commercial milestones throughout the product lifecycle
- Contribute as key subject matter expert (SME) for qualitative and quantitative observational studies
- Lead PCOS data and evidence generation planning and execution to meet cross-functional stakeholder needs
- Drive de novo COA tool development to fill evidentiary gaps
- 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
- 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
- Lead development of robust clinical outcome assessment (COA) endpoint strategy to support clinical development plans for assigned early- and late-stage programs
- Ensure fit-for-purpose clinical outcome assessment (COA) tools are selected and implemented in studies and that clinical trial design and data analyses 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 to fill evidentiary gaps, where appropriate
- Lead the design and execution of psychometric validation studies, including overseeing the development and implementation of Psychometric Analysis Plans
- Act as key SME:
- Drafting and finalization of PCOS components of clinical trial protocols and Statistical Analysis Plans
- Authoring and reviewing PCOS-relevant sections of regulatory submissions, ensuring data and messaging accuracy and alignment
- Supporting PCOS strategy development and implementation for natural history and RWE evidence studies
- Supporting publication planning, drafting, and/or review
- 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 to ensure consistent communication and alignment
- Communicate PCOS information and activities to stakeholders outside of Clinical Science (including external stakeholders)
- Help develop and disseminate the most current PCOS 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
- Within cross-functional teams, foster a culture that promotes respect, teamwork, collaboration, openness, and appreciation
Education & Experience
- 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.
PLEASE NOTE: Absent a Medical or Religious reason that prohibits vaccinations, all our incoming employees must be vaccinated for COVID-19.
We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, sexual orientation, national origin, disability status, protected veteran status, or any other characteristic protected by law.