Pharmaceuticals; Biotechnology; Research

Sr. Director, Medical Affairs, Inherited Neurometabolic Disorders (IMD)

Concord, MAOnsiteFull TimeDirector$290,000–$320,000 /yrPosted 24 days agoVisa sponsorship available

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Required skills

MD
PharmD
rare disease
specialty products
neurometabolic disease
lysosomal storage diseases
matrixed global organization
scientific integrity
patient centricity
healthcare professionals

Nice to have

insight generation
customer engagement platforms
CRM systems
departmental budgets
resource planning
global organizational environment

Role summary

The Sr. Director, Medical Affairs, Inherited Neurometabolic Disorders (IMD) will lead the U.S. Medical Affairs strategy for ITF Therapeutics' rare neurometabolic disease portfolio. This senior role involves developing and executing integrated medical strategies across pre-launch, launch, and lifecycle stages, ensuring alignment with global plans. Responsibilities include leading launch excellence, scientific communications, evidence generation, and external stakeholder engagement with KOLs, payers, and patient advocacy groups. The role also requires strong cross-functional collaboration and team leadership, fostering scientific excellence and a patient-centered culture. A minimum of 12 years of progressive pharmaceutical or biotechnology experience in Medical Affairs or Clinical Development is required, with a strong preference for rare disease or neurometabolic expertise.

Reports to: VP, US Medical Affairs & Patient Advocacy

About This Role:

The Sr. Director, Medical Affairs, Inherited Neurometabolic Disorders (IMD) is responsible for leading the U.S. Medical Affairs strategy for ITF Therapeutics' rare neurometabolic disease portfolio. Reporting to the Vice President of Medical Affairs & Patient Advocacy, this role serves as the senior U.S. medical leader for the neurometabolic therapeutics portfolio, ensuring effective translation of global medical strategy into impactful U.S. plans and successful launch execution.

As a member of the US Medical Affairs leadership team, the Senior Director provides strategic and scientific leadership across product development and commercialization, with responsibility for U.S. medical strategy, launch readiness, scientific engagement, field medical effectiveness, medical communications, education, payer engagement, and cross-functional execution.

Working closely with the Global Medical Director, this individual serves as the primary U.S. medical voice for the neurometabolic portfolio, ensuring global evidence generation, scientific communications, and medical activities address the needs of U.S. patients, healthcare professionals, payers, and other stakeholders.

The successful candidate will bring deep rare disease expertise, strong scientific and strategic leadership, and the ability to influence within a matrixed global organization. This leader will foster scientific excellence, collaboration, patient centricity, and innovation while helping establish ITF Therapeutics as a trusted partner within the neurometabolic community.What You'll Do:

U.S. Medical Strategy & Leadership

Lead development and execution of integrated U.S. Medical Affairs strategies and tactical plans across pre-launch, launch, and lifecycle stages for the neurometabolic therapeutics portfolio.

Serve as the senior U.S. medical leader for target neurometabolic diseases, ensuring medical strategies support appropriate patient identification, disease knowledge, product knowledge, and improved patient outcomes.

Medical Launch Excellence

Lead all U.S. medical launch planning and execution activities related to the neurometabolic therapeutics portfolio.

Establish launch readiness plans spanning field medical, scientific communications, medical education, insight generation, congress strategy, advisory boards, and stakeholder engagement.

Partner with Commercial, Market Access, Regulatory, Patient Advocacy, Global Medical Affairs, and Clinical Development teams to ensure coordinated launch execution.

Identify and mitigate launch risks while addressing emerging opportunities and competitive dynamics.

Scientific Communications, Evidence Generation & Insights

Lead U.S. scientific communication and medical education strategies, including congress activities, scientific exchange, educational programs, and content development.

Ensure alignment of U.S. communication plans with global publication and evidence dissemination strategies.

Review and approve U.S. medical materials and ensure communications address stakeholder educational needs.

Drive insight generation processes and translate stakeholder insights into actionable recommendations that inform development, launch, and lifecycle strategies.

Provide U.S. strategic input into evidence generation priorities, study concepts, real-world evidence initiatives, publication planning, and data dissemination activities.

Monitor scientific, clinical, regulatory, policy, and competitive developments to inform decision-making.

External Engagement & Market Access

Build and maintain relationships with KOLs, investigators, healthcare professionals, patient advocacy organizations, professional societies, payers, and other stakeholders.

Lead advisory boards and expert consultation initiatives to inform medical strategy and evidence priorities.

Represent ITF Therapeutics at scientific, advocacy, and industry forums.

Partner with Market Access and HEOR to support generation and communication of evidence demonstrating clinical, economic, and patient-centered value.

Support payer-facing medical engagements and patient access objectives.

Cross-Functional & Global Collaboration

Serve as a strategic partner to Commercial, Clinical Development, Regulatory Affairs, Market Access, Safety, Patient Advocacy, and Global Medical Affairs teams.

Provide scientific leadership across key business decisions and strategic initiatives.

Act as the primary U.S. partner to the Global Neurometabolic Medical Director, contributing U.S. market insights to disease strategy, product strategy, lifecycle planning, evidence generation, publication priorities, and scientific platform development.

Ensure alignment between U.S. and global stakeholders, incorporating U.S. insights into global plans while maintaining the highest standards of scientific integrity and patient focus.

Team Leadership & Organizational Development

Build, lead, and develop a high-performing U.S. Medical Affairs neurometabolic team.

Coach and mentor team members, fostering accountability, collaboration, innovation, and scientific excellence.

Establish priorities, performance expectations, and development plans to support organizational growth and succession planning.

Promote a patient-centered culture that balances scientific rigor with meaningful healthcare impact.

Required Skills:

MD, PharmD, or PhD in a relevant scientific, medical, or healthcare discipline.

12+ years of progressive pharmaceutical or biotechnology experience within Medical Affairs, Clinical Development, or related functions.

Demonstrated success leading U.S. Medical Affairs organizations and launch readiness activities for rare disease and/or specialty products. Experience in neurometabolic disease or lysosomal storage diseases preferred.

Experience partnering effectively with Global Medical Affairs teams in a matrixed multinational organization.

Proven ability to influence global strategy through U.S. market insights, external stakeholder engagement, and evidence generation planning.

Strong understanding of evidence generation, publication planning, scientific communications, medical governance, and healthcare decision-making processes.

Significant experience engaging KOLs, researchers, advocacy organizations, payers, and healthcare systems.

Demonstrated ability to operate strategically while driving flawless execution.

Preferred Skills:

Experience supporting product launches and pre-launch Medical Affairs activities.

Expertise in neurometabolic disorders, rare diseases, neuroscience, genetics, or related therapeutic areas.

Experience with insight generation, customer engagement platforms, and CRM systems.

Demonstrated success managing departmental budgets and resource planning.

Experience working in a global or matrixed organizational environment.

Additional Information:

The base compensation range for this role is: $290,000 - $320,000. 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.

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