Associate Director, Compassionate Use / Expanded Access Program Lead

Compassionate Use / Expanded Access Program Lead

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 Commercial Our Commercial organization leads our global sales and marketing strategies around the world. Our integrated team continues to solidify BioMarin's commercial presence in the United States and Europe and is rapidly growing in other regions, such as Latin America, the Middle East and Asia-Pacific.

We are seeking a highly motivated and experienced Compassionate Use / Expanded Access (CU / EA) Program Lead to manage our global compassionate use and expanded access programs. This individual will report into Head of Integrated Evidence, Global Medical Affairs and serve as the primary liaison between internal teams, external healthcare providers, and regulatory authorities to ensure timely, ethical, and compliant access to investigational therapies and approved therapies for patients with serious or life-threatening conditions who lack other treatment options. The ideal candidate will bring strong cross-functional leadership, regulatory knowledge, and a deep commitment to patient-centered care.

Key Responsibilities :

• Program Management
• Design, implement, and manage compassionate use and expanded access strategies and programs for BioMarin products in compliance with global and country level regulatory requirements.
• Lead the coordination of a cross-functional effort that facilitates timely decision-making, ensures appropriate resource allocation, and communicates with external stakeholders to maintain effective information flow
• Maintain systems and databases for tracking requests for CU / EA, approvals, shipments, and outcomes; ensuring timely responses and appropriate documentation, including managing CROs
• For programs with required site training, efficiently develop and deliver customer-focused sessions.
• Oversee all aspects of the program, including timelines, budget, and resources and prepare periodic program updates.
• Support internal and external audits and inspections by providing CU / EA documentation and summaries.
• Regulatory & Compliance Oversight
• Prepare and submit required documentation to global regulatory agencies, including Expanded Access INDs, single-patient INDs, and protocol amendments.
• Ensure all activities align with relevant regulations and guidelines and stay current on global regulatory developments and advise internal stakeholders on relevant changes.
• Stakeholder Engagement & Communication
• Provide training and guidance to internal teams (e.g., Medical Affairs, Clinical, Regulatory) on CU / EA procedures and best practices.
• Collaborate with legal, pharmacovigilance, supply chain, and relevant cross functional teams to support access and distribution.
• Strategic Development
• Contribute to the development of CU / EA policies and strategies aligned with product lifecycle and global access objectives.
• Improve upon current and develop new Corporate policies and Standard Operating Procedures for CU / EA
• Identify potential risks and develop mitigation strategies.

Qualifications :

Preferred Skills and Attributes :

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.