Senior Medical Director, Global Rare

Role Summary

The Senior Medical Director, Global Rare, provides global strategic and scientific support for the Ultra Rare franchise. This role drives key initiatives of the global medical team, including evidence generation, scientific communication, and stakeholder engagement. The incumbent serves as the primary point of contact for specific regions and their country medical teams, liaising with Clinical Development, Regulatory, Safety, Program Leadership, MAC (Global and Regional) and other medical expertise areas.

Responsibilities

• Strategic Leadership & Governance:
  • Serve as the primary point of contact for global medical affairs activities and act as escalation point for complex regional medical issues.
  • Provide strategic and scientific input to the Rare Disease franchise within the global medical matrix team framework; ensure alignment of global medical strategy with program strategy. Represent medical affairs at Program Leadership Team and Global MAC.
  • Accountable for strategic leadership and oversight of all medical affairs activities in partnership with VESt, Medical Communications and Training, PAE, and Medical Operations.
  • Ensure compliance with global and local regulations, including oversight of pharmacovigilance reporting obligations in collaboration with Safety teams.

• Evidence Generation & Scientific Strategy:
  • Collaborate with Clinical Development and VESt to provide medical affairs input into pivotal study design; work closely with Preclinical, Regulatory, Clinical Operations, Biometrics, and Commercial to integrate product development and lifecycle plans.
  • Define and prioritize therapeutic area-specific evidence gaps; develop and implement evidence generation strategies (e.g., natural history studies, phase 4 trials, registries) to address stakeholder needs.
  • Provide medical oversight for observational studies, comparative effectiveness research, chart reviews, registries, and other evidence generation projects.

• Scientific Communication & Training:
  • Partner with Medical Communications to ensure timely dissemination of scientific data through publications and congress activities.
  • Collaborate with Medical Training to design and deliver high-quality scientific and skill-based training for global and regional medical teams.
  • Review regional commercial materials (CRB) and medical materials (MRB) for scientific accuracy and consistency.

• External Engagement & Representation:
  • Develop and maintain relationships with global KOLs, policy makers, and payer organizations to support education and access.
  • Represent the company at external scientific forums, advisory boards, and rare disease consortia.
  • Work closely with Global Marketing and European Regional Marketing to define stakeholder engagement strategies and drive global KOL engagement activities.

Qualifications

The Senior Medical Director will be part of a highly visible Global medical affairs team within a dynamic, fast-paced biotechnology company. The candidate should possess the following capabilities and characteristics:

• MD (Required)
• Experience in leading Medical Affairs activities in North America and European countries (Required)
• Demonstrated high-level professional knowledge and skills in medical affairs and R&D in the pharmaceutical or biotech industry (Required)
• Experience leading teams/programs in a matrix environment (Preferred)
• Demonstrate experience in rare or genetic diseases (Preferred)
• Knowledge of the biopharmaceutical industry and evidence generation with an understanding of key regulatory bodies (Required)
• High degree of professionalism, maturity, and confidentiality (Required)
• Strong written and verbal communication skills as well as strong leadership and interpersonal skills (Required)
• Demonstrated reputation as a well-respected, dynamic team leader, player and coach (Required)
• Capable of managing shifting priorities in a rapidly changing environment (Required)
• Ability to deliver on company global program goals and objectives as they pertain to medical affairs (Required)
• Ability to affect positive organizational change at the company and team levels (Required)
• Travel will be required (up to 50%) (Required)
• Hybrid onsite location when not traveling: 675 West Kendall Street, Cambridge, MA (Required)