Associate Director, Compassionate Use/Expanded Access Program Lead 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 to the 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. About BioMarinBioMarin is a global biotechnology company that pursues bold science to translate genetic discoveries into new medicines that advance the future of human health. Since 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines. We develop medicines for patients with significant unmet medical need using a range of treatment modalities. Our Commercial organization leads our global sales and marketing strategies worldwide and is growing across regions including the United States, Europe, Latin America, the Middle East and Asia-Pacific. 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. Coordinate cross-functional efforts to facilitate timely decision-making, allocate resources, and communicate with external stakeholders to maintain effective information flow. Maintain systems and databases for tracking CU/EA requests, approvals, shipments, and outcomes; ensure timely responses and appropriate documentation, including managing CROs. Develop and deliver site-training sessions for programs with required training. Oversee all aspects of the program including timelines, budget, and resources; 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 activities align with applicable regulations and guidelines; stay current on global regulatory developments and advise internal stakeholders on changes.

Stakeholder Engagement & Communication

Provide training and guidance to internal teams on CU/EA procedures and best practices. Collaborate with legal, pharmacovigilance, supply chain, and 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 current policies and standard operating procedures for CU/EA. Identify potential risks and develop mitigation strategies.

Qualifications

Bachelor’s degree in Life Sciences, Pharmacy, Nursing, Public Health, or related field (Advanced degree preferred). Minimum 5 years of experience in the pharmaceutical/biotechnology industry or CRO, with at least 5 years focused on initiation and management of expanded access programs or clinical operations. Strong understanding of global regulatory frameworks governing compassionate use and expanded access programs, including country-level provisions. Proven experience working with external stakeholders in a highly regulated environment. Demonstrated ability to manage complex projects, timelines, and budgets. Ability to navigate complex ethical and regulatory landscapes with empathy and professionalism. Proficiency with electronic data management systems and regulatory submission platforms.

Preferred Skills and Attributes

Working knowledge of ICH Good Clinical Practices and other regulatory/health authority experience or clinical/regulatory affairs certification (e.g., RAC, SOCRA, ACRP). Familiarity with rare disease therapeutic areas is a plus. Experience supporting investigational product supply logistics. Ability to manage multiple complex or large clinical/medical programs simultaneously. Demonstrated leadership and problem-solving skills. Passion for patient advocacy and access to care.

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 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.

#J-18808-Ljbffr