Associate Director, Compassionate Use/Expanded Access Program Lead

Role Summary

We are seeking a highly motivated and experienced Compassionate Use/Expanded Access (CU/EA) Program Lead to manage global compassionate use and expanded access programs. This role reports to the Head of Integrated Evidence, Global Medical Affairs, and serves 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.

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

• Required: Bachelor’s degree in Life Sciences, Pharmacy, Nursing, Public Health, or related field (Advanced degree preferred).
• Required: Minimum 5 years of experience in the pharmaceutical or biotechnology industry or CRO with a minimum of 5 years focused on initiation and management of expanded access programs or clinical operations.
• Required: Strong understanding of global regulatory frameworks governing compassionate use and expanded access programs including country level provisions.
• Required: Proven experience working with external stakeholders in a highly regulated environment.
• Required: Demonstrated ability to manage complex projects, timelines, and budgets.
• Required: Ability to navigate complex ethical and regulatory landscapes with empathy and professionalism.
• Required: Proficiency with electronic data management systems and regulatory submission platforms.
• Preferred: Must have 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.
• Preferred: Experience supporting investigational product supply logistics.
• Preferred: Ability to manage complex or multiple large clinical/medical programs simultaneously.
• Preferred: Demonstrated leadership and problem-solving skills.
• Preferred: Passion for patient advocacy and access to care.

Education

• Bachelor’s degree in Life Sciences, Pharmacy, Nursing, Public Health, or related field; Advanced degree preferred.

Skills

• Working knowledge of ICH Good Clinical Practices and other regulatory/health authority experience or related certifications (RAC, SOCRA, ACRP).
• 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.