Medical Scientific Director

BioMarin Pharmaceutical Inc.; Northern (KY); Hybrid; USD 180,000 - 240,000; Full time

Job summary

BioMarin Pharmaceutical Inc. is seeking a Medical Scientific Director to oversee day-to-day oversight of BioMarin-sponsored and collaborative studies across a therapeutic area, ensuring consistency of approach, conduct, monitoring, analysis, and reporting across one or more programs.

The position partners with Real World Evidence (RWE)/Epidemiology, Global Medical Leads, Data Sciences and Global Study Operations to develop practical study strategies and ensure optimal hands-on study operations

Qualifications

• PharmD/PhD/MD with at least 5 years experience in observational and Phase IIIb/IV studies.
• MS/MPH with 10+ years similar experience.
• Experience delivering a portfolio of protocols meeting program objectives.

Responsibilities

• Executes evidence generation activities (Phase IIIb/IV studies) to fill priority gaps.
• Collaborates with Global Medical Leads, RWE, HEOR to develop study pull-through plans.
• Ensures timely issue identification and implements complex solutions.
• Provides scientific input on study concepts, protocols and regulatory responses.
• Presents study results to internal and external stakeholders at conferences and forums.
• Coordinates Medical Affairs input for regulatory submissions and inspections.

Skills

Cross-functional collaboration

End-to-end study delivery

Observational studies

Phase IIIb/IV studies

Study design & analysis

Study monitoring

Regulatory questions handling

Scientific writing & documentation

Education

PharmD/PhD/MD

MS/MPH

Job description

BioMarin is a leading rare disease biotechnology company focused on genetically defined conditions.

Guided by our purpose to develop medicines that make a profound impact on people’s lives, our global teams have delivered a portfolio of therapies since our founding in 1997. Our revolutionary treatments for conditions like achondroplasia (the most common form of dwarfism), PKU (phenylketonuria), CLN2, a form of Batten disease, and a number of forms of MPS (mucopolysaccharidosis) offer new possibilities for patients and families who previously had few, if any, available options. More recently, with the close of the Amicus acquisition, our portfolio has expanded to include therapies for Fabry disease and Pompe disease, expanding our ability to reach more people living with rare genetic conditions.

Our success comes from our unwavering commitment to excellence, our deep understanding of patient needs, our scientific expertise, and our world-class manufacturing capabilities. At the heart of BioMarin is a dedicated team of the brightest minds in the industry working together to deliver innovative therapies to patients and families around the world.

About Worldwide Research and Development

From research and discovery to post-market clinical development, our R&D 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 rare diseases.

The Role

The Medical Scientific Director is accountable for the day-to-day oversight of numerous BioMarin-sponsored and collaborative studies across a therapeutic area, ensuring consistency of approach, conduct, monitoring, analysis, and reporting of studies within one or more programs. The Medical Scientific Director will work in partnership with Real World Evidence (RWE)/Epidemiology, Global Medical Leads, Data Sciences and Global Study Operations to develop practical study strategies and ensure optimal hands-on study operations and execution support for Medical Affairs-led studies from cross functional partners.

The Medical Scientific Director is part of the Integrated Evidence function comprised of subject matter experts in evidence planning, observational study design and data interpretation. The function works to drive excellence in evidence planning and implement novel study design approaches to generate robust evidence to enhance understanding of unmet medical need, support patient and endpoint selection for clinical studies and inform medical practice on appropriate use and long-term safety and effectiveness of BioMarin products to drive clinical adoption.

Key Responsibilities

Study Strategy and Implementation

• Executes evidence generation activities (non-interventional, low-interventional and interventional Phase IIIb/IV studies) that will fill priority evidence gaps identified in product Integrated Evidence Plans (IEPs)
• Collaborates closely and builds alliances with Global and Country Medical Leads, RWE Lead, and HEOR to understand key global and local (key market) needs and opportunities in order to develop strategic study pull through and dissemination plans
• Collaborates closely with operational strategic partners to ensure successful implementation of GMAF-led studies; identifies issues in a timely manner, leads implementation of complex solutions, and/or escalates as appropriate
• Provides scientific input into the development of study concepts, protocols and protocol amendments, statistical analysis plans, data review plans, and responses to regulatory agency questions regarding scientific aspects of study protocols
• Leads the development of annual, interim and periodic study reports for Medical Affairs-led studies, including coordination of SME input, data interpretation and presentation, and ensuring that regulatory agency questions (where relevant) have been adequately addressed
• Designs secondary data analysis plans of BioMarin clinical and real-world data to support program strategy
• Ensures adequate medical monitoring oversight of assigned studies (where relevant) including monitoring and arbitration on subject eligibility, quality of safety and study data, development and review of ICFs, CRFs, TLGs, accurate AE and concomitant medication coding, and inspection readiness activities
• Presents results of assigned studies to internal and external stakeholders, including at scientific conferences, and other relevant forums.
• Coordinates Medical Affairs input in preparation of regulatory submissions, responses to regulatory queries, IRB/Ethics Committee questions, and in preparation for program regulatory inspections and audits for assigned programs.
• Participates in initiatives to enhance or drive efficiency in study implementation

Desired Experience

• PharmD/PhD/MD with at least 5 years experience conducting observational and Phase IIIb/IV studies within Clinical Development and/or Medical Affairs; MS/MPH with 10+ years similar experience
• Experience in delivering a portfolio of protocols and ensuring they meet strategic program objectives
• Experience responding to regulatory agency, IRB/Ethics Committee questions regarding study designs and protocol conduct

Skills

• Proven track record of working with cross-functional partners to ensure successful end-to-end delivery of studies in support of product Integrated Evidence Plans.
• Track record of delivering high quality novel observational and Phase IIIb/IV studies.
• Works effectively in a cross-functional matrix settings to shape the study strategy for one or more assigned projects.
• Awareness of novel prospective observational and interventional study designs.
• Understanding of statistical and data analysis, research methods and design.
• Experience in study monitoring.
• Advanced knowledge of the clinical research process and Good Clinical /Pharmacoepidemiology Practice standards and requirements relating to conducting research and disseminating of scientific information.
• Strong scientific writing and document development.

Note: This description is not intended to be all-inclusive, or a limitation of the duties of the position. It is intended to describe the general nature of the job that may include other duties as assumed or assigned.

Equal Opportunity Employer/Veterans/Disabled

An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.

Equal Opportunity Employer/Veterans/Disabled

An Equal Opportunity Employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, or protected veteran status and will not be discriminated against on the basis of disability.

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