Global Medical Lead, Neurometabolic

Reports to: VP/Head of Global Medical Affairs, Rare Disease
Location: Remote

About the Role

The Global Medical Lead (GML), Neurometabolic is the strategic and medical/scientific owner of all global medical affairs activities across the neurometabolic portfolio, leading the end-to-end global medical strategy, evidence generation plans, and lifecycle medical activities for programs spanning early development through commercialization.

As the senior medical affairs leader for the neurometabolic franchise within Global Medical Affairs, this individual drives integrated medical strategy, ensures robust cross-functional alignment, leads scientific engagement worldwide, and guides the development and implementation of medical plans across geographies.

This role acts as the central node connecting global, regional, and local medical teams, ensuring coherent execution, knowledge flow, and scientific excellence. The GML is accountable for advancing scientific understanding, supporting optimal clinical use, and improving patient outcomes across the neurometabolic disease portfolio.

The ideal candidate will bring experience across multiple stages of asset development, including both early clinical development and launch/commercialization readiness, and will demonstrate the ability to build scientific strategy in complex and evolving rare disease environments.

What You'll Do

Global Medical Strategy Leadership

• Serve as the enterprise medical lead for the Neurometabolic Franchise, responsible for global medical strategy, scientific narrative, and lifecycle medical planning.
• Develop and maintain Global Medical Plans (GMPs) and ensure alignment with integrated evidence generation plans (IEGPs).
• Partner closely with Global Asset Leadership and the Head of Global Medical Affairs to ensure medical strategy aligns with portfolio priorities and enterprise objectives.
• Define key scientific pillars, messaging platforms, evidence generation priorities, and insight-gathering strategies across all stages of development.
• Lead medical strategy development for programs ranging from early-stage clinical development through launch readiness and commercialization.
• Ensure medical plans evolve appropriately as assets advance through development milestones and lifecycle stages.

Evidence Generation & Data Strategy

• Lead the global strategy and scientific rationale for clinical, real-world evidence (RWE), health economics and outcomes research (HEOR), and observational evidence generation activities.
• Collaborate closely with Clinical Development, Biometrics, Regulatory Affairs, Value & Access, and Evidence Generation teams to ensure decision-grade evidence addresses clinical, regulatory, payer, and stakeholder needs.
• Identify evidence gaps and proactively develop plans to address unmet scientific and clinical questions.
• Oversee global data dissemination activities, including congress strategy, publications planning, scientific communications, medical education activities, and advisory boards.
• Ensure evidence generation strategy supports both current and future franchise objectives.
• Maintain awareness of evolving stakeholder expectations and emerging evidence requirements across global markets.

Scientific Leadership & External Engagement

• Serve as a primary scientific and medical spokesperson for the neurometabolic franchise.
• Represent the organization at scientific meetings, advisory boards, investigator meetings, and external collaborations.
• Lead global KOL engagement strategy and scientific advisory planning to inform medical strategy, evidence generation priorities, and lifecycle decision-making.
• Build and maintain meaningful relationships with international thought leaders, investigators, patient advocacy groups, academic partners, and healthcare stakeholders.
• Maintain deep expertise in neurometabolic diseases, emerging science, competitive intelligence, treatment paradigms, and evolving patient needs.
• Drive development of clear scientific platforms and communication strategies that support long-term franchise objectives.

Cross‑Functional Integration & Launch / Lifecycle Excellence

• Partner with Regulatory Affairs, Clinical Development, Market Access, HEOR, Safety, Commercial (non-promotional), and Portfolio Strategy teams to ensure integrated lifecycle planning.
• Lead medical contributions to:
  • Global launch strategy and readiness activities
  • Medical launch planning and execution
  • Label expansion opportunities
  • Lifecycle management initiatives
  • Post-approval evidence generation plans
  • Development-stage asset planning
• Ensure medical insights are appropriately incorporated into development, access, and commercial decision-making.

Leadership & Matrix Team Development

• Lead and influence cross-functional and cross-regional teams in a global matrix environment.
• Provide strategic oversight and guidance to regional and local medical colleagues to ensure alignment with global strategy.
• Drive collaboration between US, European, and international stakeholders.
• Build strong partnerships with medical, commercial, clinical, and development organizations.
• Support development of future medical capabilities and organizational growth as the neurometabolic franchise expands.
• Mentor and develop medical colleagues while fostering a culture of scientific excellence and accountability.
• Influence without authority and effectively manage complex stakeholder environments.

Medical Operations, Governance & Scientific Integrity

• Lead governance processes supporting franchise medical strategy, evidence review, and scientific communications.
• Ensure all medical activities comply with global regulatory, legal, and ethical standards.
• Oversee scientific interactions and partnerships with academic institutions, investigators, registries, consortia, advocacy groups, and external vendors.
• Ensure scientific rigor, compliance, and transparency across all evidence generation and medical affairs activities.
• Contribute to budget development and management for global medical initiatives.
• Establish processes that support efficient global execution while maintaining flexibility for regional adaptation. Qualifications and Experience

Qualifications and Experience

• Advanced scientific or medical degree required (MD, PhD, PharmD strongly preferred).
• 8-15+ years of Medical Affairs, Clinical Development, or related biopharmaceutical experience.
• Significant experience in rare disease required; neurometabolic, neurological, genetic, metabolic, pediatric, or specialty disease experience strongly preferred.
• Demonstrated success developing and executing global medical strategies.
• Experience supporting both development-stage and commercial-stage assets preferred.
• Experience preparing for or supporting global product launches.
• Proven expertise in evidence generation, scientific communications, and stakeholder engagement.
• Strong experience interacting with international KOLs, investigators, and scientific experts.
• Demonstrated ability to lead complex cross-functional teams and influence without direct authority.
• Prior global or regional medical affairs leadership experience preferred.
• Experience operating effectively within matrix organizations and high-growth environments.
• Willingness and ability to travel internationally on a regular basis.

Core Competencies

• End-to-end asset leadership
• Scientific excellence and strategic medical insight
• Global medical affairs expertise
• Launch readiness and commercialization support
• Strong cross-functional and matrix leadership
• Global mindset and cultural agility
• Executive communication skills
• Ability to simplify complexity and drive clarity
• Stakeholder engagement and influence
• Strategic problem solving
• Patient-centric decision making
• Adaptability in evolving and fast-paced environments

Success Measures

• High-quality, scientifically robust, globally aligned medical strategy across the neurometabolic franchise
• Successful development and execution of evidence generation programs supporting regulatory, clinical, and payer needs
• Effective establishment of scientific platforms supporting launch readiness and lifecycle objectives
• Strong partnerships with global asset teams and cross-functional leadership
• Consistent execution of global medical priorities across regions
• Recognition as a trusted scientific authority among internal and external stakeholders
• Meaningful contribution to patient outcomes and advancement of care within neurometabolic diseases
• Successful support of both near-term launch objectives and long-term franchise growth strategy

Additional Information

The base compensation range for this role is: $295,000 - $380,000. Compensation is determined through an analytical approach utilizing a combination of factors including, but not limited to, relevant skills & experience, job location, and internal equity.