Director, Quantitative Science/Psychometrician

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 Culture

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.

Summary

BioMarin Biometrics is responsible for designing, capturing, analyzing, and presenting data that can drive key decisions for Clinical Development, Medical Affairs, and other business areas of BioMarin. With a quality-by-design culture, Biometrics builds quality data that is fit-for-purpose to support statistically sound investigation of critical scientific questions. The Biometrics team develops solid analytics that are visually relevant and impactful in supporting key data-driven decisions across BioMarin.

As part of the Quantitative Science organization, the Director/Senior Director 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.

Responsibilities

 

 

Strategy and Planning

 

·         Demonstrated scientific leadership in planning and executing patient-centered research studies including:

•       Evaluating the measurement properties of new and existing patient-centered outcome tools (e.g., clinical outcome assessments) for use in clinical trials

•       Experience with designing and implementing psychometric study protocols, statistical analysis plans, and reports fit for regulatory submission

•       Experience interpreting and addressing Health Authority (FDA, EMA) and HTA feedback on quantitative analytic approach and results

·         Work with the Head of the Quantitative Science and other leaders of the Biometrics Team (namely in Data Management Science, Data Analytics Science, Quantitative Science, and Biostatistics) to organize, plan, manage, and control deliverables in support of company objectives

·         Oversee and guide the Biometrics deliverables across managed programs; ensure Biometrics deliverables are comprehensive and high quality

·         Oversee the performance of Contract Research Organizations (CROs)

·         Manage and resolve escalated issues; mitigate product risks and identify opportunities affecting the Biostatistics activities

·         Ensure statistical best practice methodologies are considered during program planning and protocol/SAP/PAP development, throughout the program lifecycle

·         Monitor project progress and ensure proper resource allocation for successful project deliverables against goals and timelines

·         Ensure Biostatistics processes and procedures are carried out in a compliant and consistent manner according to SOPs, WIs, policies, and published guidelines

·         Stay current on global best practices in measurement science strategy development and implementation

·         Understand and apply modern drug discovery, research development, and measurement science principles and processes

Operational execution

·         Develop statistical and psychometric analysis plans; supporting the analysis in-house, overseeing those conducted by external partners, and communicating results to internal partners.

·         Co-Lead development strategy for de novo COA tool development to fill evidentiary gaps, where appropriate, including the design and execution of psychometric validation studies

·         Act as key statistical/analytical Subject Matter Expert and provide operational statistical leadership in:

•       Drafting and finalization of components of clinical trial protocols, Psychometric Analysis Plans, and Statistical Analysis Plans

•       Authoring and reviewing quantitative sections of regulatory submission documents (e.g., briefing books, evidence dossiers, clinical study reports [CSRs]), ensuring data and messaging accuracy and alignment

•       Supporting analytic strategy development and implementation for natural history and RWE evidence studies

•       Supporting dissemination activity planning, drafting, and/or review for publications, posters, presentations to communicate study outcomes

 

·         Organize and lead cross-functional working groups and teams to organize, plan, manage, and control deliverables against goals and timelines across the portfolio 

·         Statistical software proficiency in:

•       Fluent in SAS (including proc SQL),  and R 

•       Experience with the following software is a plus: IRT programs (e.g., R package MIRT, FlexMIRT), MPLUS, WINSTEPS, BILOG-MG, PARSCALE, HLM

 

·         Proficiency in modern statistical techniques, such as:

•       General and generalized linear models, exploratory and confirmatory factor analysis and SEM, correlation analysis, item response theory and related psychometric techniques, simulation, cluster analysis, survey design and sampling methods, signal detection theory, Bayesian and other nonparametric techniques, clinically meaningful change analyses; discrete choice experiment design and implementation is a plus

Cross-functional Influence & Leadership

·         Ensure effective leadership of drug development process, partnering with Clinical Development and Patient Centered Outcomes (PCO) stakeholders to define analysis planning, and reporting of clinical trial, psychometric and observational study outcomes

·         Ensure key Biometrics functional and cross-functional program teams (Statistical Analysis and Review Team (SART) and Data Analysis and Review Team (DART)) are functioning according to team charters to ensure appropriate cross-functional collaboration, communication, and expert input into program deliverables and outcomes

·         Act as Biometrics primary representative on key cross-functional teams (CDT, BOAT) as a standing member and liaise between the team and Biometrics functions for all Biometrics matters regarding the product

·         Ensure timely and effective communication and alignment among the CDT, BOAT, Biometrics management/team, and the Study team members on all the Biometrics and psychometric issues

·         Participate in or lead elements of key Biostatistics and/or Biometrics-wide strategic change or improvement initiatives

·         Provide innovative statistical problem solving and program design guidance to the Biometrics team, study teams, and other key stakeholders Oversee the development and dissemination of the most current psychometric and analytic best practices throughout the organization (via email, presentations, desk procedures, reference documents and repositories) to foster enrichment and education of cross-functional stakeholders

·         Represent Quantitative Science on internal and external scientific and advocacy working groups

Education & Experience

• A doctoral degree in the field of Biostatistics, Psychometrics, Measurement Science, or Quantitative Health Science or related is required
• 3 + year experience with PhD
• 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.