BioSpace
Director, Global Medical Lead – Lysosomal Storage Disorders
BioSpace, Topeka, Kansas, United States
Director, Global Medical Lead – Lysosomal Storage Disorders
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 produce transformative medicines. 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.
Role The Global Medical Lead, Director level, is a critical leadership role in Global Medical Affairs, working on global activities for BioMarin’s Enzyme Conditions portfolio. The role reports into the Global Medical Lead – ENPP1 Deficiency & LSD and represents Medical Affairs in core cross‑functional teams.
Key Responsibilities
In partnership with the Commercial organization, co‑develop the Integrated Brand Plan (IBP), as core member of the 3M (Medical – Marketing – Market Access) Team
Offer scientific and medical expertise to inform the overall product strategy, contributing to clinical development, commercialization, and market access plans
Lead development, execution, and communication of the Global Medical Plan in collaboration with other internal stakeholders across BioMarin
Provide subject matter expertise as well as strategic leadership in key internal medical and clinical teams. The GML is the leader of the Medical Affairs SMART team, where he/she leads development of the medical plan and ensures that all critical components of medical plans are executed and monitored for the intended impact
Act as product and disease area expert and primary contact point for all global compound or disease area related issues both internally and for the external community
Partner with other departments, such as commercial, finance, and clinical development, to ensure that the medical affairs budget supports and aligns with overall business goals
Launch and Pre‑Launch Excellence
Develop Medical Affairs launch strategy and roadmap and lead all Medical Affairs launch activities in collaboration with Commercial and other key stakeholders
Filing / Regulatory
Represent Medical Affairs in the development of regulatory documents and submissions (e.g. briefing books, protocols, label discussions etc.)
Represent Medical Affairs at FDA/EMA meetings
Integrated Evidence Package
As a core member of the Integrated Evidence Team, contribute to the development of the integrated evidence package for therapeutic area, bringing forward scientific and clinical data that demonstrates product value, addresses evidence gaps and helps optimize patient access
Ensure tactics led by Medical Affairs are included in the Medical Plan and delivered in a timely manner
Clinical Research
Responsible for providing medical input into development, execution and interpretation of Clinical Development Plans, clinical protocols, including review of draft external research protocols, reports and manuscripts
Contribute scientific expertise to development of real‑world evidence projects including, disease registries and post‑marketing commitments
Evaluate safety issues associated with product in collaboration with other departments
Assist in critically evaluating study results for statistical and final reports and ongoing reviews with study investigators, resolving problems arising during clinical investigations and preparing reports to management and medical community
Medical Engagement
Guide development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, tools are developed to support execution, and mechanisms are in place to measure impact
Set the aspirational scientific narrative for therapeutic area and contribute to the development, review and approval of relevant global publication plans. Ensure publications are appropriately shared with the external community to optimize patient care
Contribute scientific expertise to the development of Implementation Science projects as part of Health Systems Engagement
Training
Support internal disease and product training in collaboration with Medical Affairs Learning and Development
Compliance
Ensure compliance with policies, SOPs, Code of Business Conduct and Corporate Social Responsibility, as well as relevant legislation and regulations
Desired Experiences
MD, PhD, PharmD or other advanced life sciences degree required
Extensive industry experience, especially in Medical Affairs (≥7 years) and Clinical Development, ideally in a global role with experience in Genetic Medicine or LSDs/Rare Disease inherited metabolic conditions
Desired Skills
Ability to work, influence, and gain consensus across regions and cross‑functional teams
Demonstrate solid understanding of cross‑functional inter‑dependencies across the drug development lifecycle
Excellent communication skills and a strong enterprise mindset required for problem solving and high‑level presentations for senior executive staff review
US and international experience working in orphan or specialty markets
Knowledge and hands‑on skills required to develop the strategy, framework, and scientific content for a wide range of post‑marketing studies
Knowledgeable and current in GCP guidelines and compliance rules globally. Clinical trial and publication experience is desirable
Track record of successfully influencing without authority, partnering across functions especially with but not limited to Commercial
Ability to understand the organization’s financial goals, market landscape, and strategic objectives, and to apply that understanding to support scientific and strategic initiatives that deliver value to both patients and the company
Able to thrive in an ambiguous and demanding environment, with high capacity for effective relationship building and teamwork
Flexibility and adaptability
Sensitivity to a multicultural environment
Willingness to travel, mostly internationally
English proficiency required
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.
Seniority level Director
Employment type Full‑time
Job function Business Development and Sales
Industries Internet News
#J-18808-Ljbffr
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.
Role The Global Medical Lead, Director level, is a critical leadership role in Global Medical Affairs, working on global activities for BioMarin’s Enzyme Conditions portfolio. The role reports into the Global Medical Lead – ENPP1 Deficiency & LSD and represents Medical Affairs in core cross‑functional teams.
Key Responsibilities
In partnership with the Commercial organization, co‑develop the Integrated Brand Plan (IBP), as core member of the 3M (Medical – Marketing – Market Access) Team
Offer scientific and medical expertise to inform the overall product strategy, contributing to clinical development, commercialization, and market access plans
Lead development, execution, and communication of the Global Medical Plan in collaboration with other internal stakeholders across BioMarin
Provide subject matter expertise as well as strategic leadership in key internal medical and clinical teams. The GML is the leader of the Medical Affairs SMART team, where he/she leads development of the medical plan and ensures that all critical components of medical plans are executed and monitored for the intended impact
Act as product and disease area expert and primary contact point for all global compound or disease area related issues both internally and for the external community
Partner with other departments, such as commercial, finance, and clinical development, to ensure that the medical affairs budget supports and aligns with overall business goals
Launch and Pre‑Launch Excellence
Develop Medical Affairs launch strategy and roadmap and lead all Medical Affairs launch activities in collaboration with Commercial and other key stakeholders
Filing / Regulatory
Represent Medical Affairs in the development of regulatory documents and submissions (e.g. briefing books, protocols, label discussions etc.)
Represent Medical Affairs at FDA/EMA meetings
Integrated Evidence Package
As a core member of the Integrated Evidence Team, contribute to the development of the integrated evidence package for therapeutic area, bringing forward scientific and clinical data that demonstrates product value, addresses evidence gaps and helps optimize patient access
Ensure tactics led by Medical Affairs are included in the Medical Plan and delivered in a timely manner
Clinical Research
Responsible for providing medical input into development, execution and interpretation of Clinical Development Plans, clinical protocols, including review of draft external research protocols, reports and manuscripts
Contribute scientific expertise to development of real‑world evidence projects including, disease registries and post‑marketing commitments
Evaluate safety issues associated with product in collaboration with other departments
Assist in critically evaluating study results for statistical and final reports and ongoing reviews with study investigators, resolving problems arising during clinical investigations and preparing reports to management and medical community
Medical Engagement
Guide development of the Medical Engagement strategy with the Medical Engagement Lead ensuring inclusion of key stakeholders, tools are developed to support execution, and mechanisms are in place to measure impact
Set the aspirational scientific narrative for therapeutic area and contribute to the development, review and approval of relevant global publication plans. Ensure publications are appropriately shared with the external community to optimize patient care
Contribute scientific expertise to the development of Implementation Science projects as part of Health Systems Engagement
Training
Support internal disease and product training in collaboration with Medical Affairs Learning and Development
Compliance
Ensure compliance with policies, SOPs, Code of Business Conduct and Corporate Social Responsibility, as well as relevant legislation and regulations
Desired Experiences
MD, PhD, PharmD or other advanced life sciences degree required
Extensive industry experience, especially in Medical Affairs (≥7 years) and Clinical Development, ideally in a global role with experience in Genetic Medicine or LSDs/Rare Disease inherited metabolic conditions
Desired Skills
Ability to work, influence, and gain consensus across regions and cross‑functional teams
Demonstrate solid understanding of cross‑functional inter‑dependencies across the drug development lifecycle
Excellent communication skills and a strong enterprise mindset required for problem solving and high‑level presentations for senior executive staff review
US and international experience working in orphan or specialty markets
Knowledge and hands‑on skills required to develop the strategy, framework, and scientific content for a wide range of post‑marketing studies
Knowledgeable and current in GCP guidelines and compliance rules globally. Clinical trial and publication experience is desirable
Track record of successfully influencing without authority, partnering across functions especially with but not limited to Commercial
Ability to understand the organization’s financial goals, market landscape, and strategic objectives, and to apply that understanding to support scientific and strategic initiatives that deliver value to both patients and the company
Able to thrive in an ambiguous and demanding environment, with high capacity for effective relationship building and teamwork
Flexibility and adaptability
Sensitivity to a multicultural environment
Willingness to travel, mostly internationally
English proficiency required
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.
Seniority level Director
Employment type Full‑time
Job function Business Development and Sales
Industries Internet News
#J-18808-Ljbffr