Associate Director, Compassionate Use/Expanded Access Program Lead
BioMarin Pharmaceutical Inc.
full-time
Posted on:
Location: 🇺🇸 United States
Visit company websiteJob Level
Senior
About the role
- Report into Head of Integrated Evidence, Global Medical Affairs; serve as primary liaison between internal teams, external healthcare providers, and regulatory authorities
- 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 coordination of cross-functional efforts to facilitate timely decision-making, resource allocation, and external communication
- Maintain systems and databases for tracking CU/EA requests, approvals, shipments, and outcomes; manage CROs and ensure timely responses and documentation
- Develop and deliver customer-focused site training when required
- Oversee program timelines, budget, and resources and prepare periodic program updates
- Support internal and external audits and inspections by providing CU/EA documentation and summaries
- Prepare and submit required documentation to global regulatory agencies, including Expanded Access INDs, single-patient INDs, and protocol amendments
- Stay current on global regulatory developments and advise internal stakeholders on relevant changes
- Provide training and guidance to internal teams (Medical Affairs, Clinical, Regulatory) on CU/EA procedures and best practices
- Collaborate with legal, pharmacovigilance, supply chain, and cross-functional teams to support access and distribution
- Contribute to development of CU/EA policies, corporate policies, and Standard Operating Procedures; identify risks and develop mitigation strategies
Requirements
- Bachelor’s degree in Life Sciences, Pharmacy, Nursing, Public Health, or related field (Advanced degree preferred)
- Minimum 5 years of experience in the pharmaceutical or biotechnology industry or CRO
- Minimum 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
- Working knowledge of ICH Good Clinical Practices and other relevant regulatory/health authority experience or clinical or regulatory affairs certification (e.g., RAC, SOCRA, ACRP) (preferred)
- Familiarity with rare disease therapeutic areas (a plus)
- Experience supporting investigational product supply logistics
- Ability to manage complex or multiple large clinical/medical programs simultaneously
- Demonstrated leadership and problem-solving skills
- Passion for patient advocacy and access to care