Global Medical Affairs Therapy Area Lead - PKU (East Coast Based Candidates)

Role Summary

Global Medical Affairs (GMA) at PTC is focused on supporting health care providers with evidence-based decisions for patients with rare diseases. The Global Therapeutic Area Lead, PKU, defines the global medical affairs vision, strategy and tactics to support the PKU business, collaborates with regional heads to align local needs, and partners with enabling functions to execute the medical strategy for PKU and lifecycle management. The role serves as the medical affairs subject matter expert for PKU, educating KOLs, physicians, and internal colleagues, and ensuring regulatory compliance.

Responsibilities

• Develops and leads strategic global medical affairs activities related to PKU to accelerate commercialization in launched markets and support launches in other markets.
• Drives collaboration among regional/local Medical Affairs, GMA enabling functions, and other teams to ensure PKU strategic plans are well developed and executed; fosters cross-functional partnerships.
• Serves as the PKU subject matter expert in Global Medical Affairs; partners with medical and commercial training to educate internal and external colleagues on PKU.
• Develops and finalizes global evidence generation plan in collaboration with regional/country medical heads and enabling functions; ensures alignment with internal stakeholders.
• Plans and leads the Investigator Initiated Studies (IIS) strategy for PKU.
• Engages PKU KOLs, stakeholders, and healthcare professionals to implement the medical strategy; identifies and facilitates scientific exchange with PKU experts.
• Represents PKU medical affairs in Clinical Development, Commercial Development, and Life-Cycle Management teams.
• Represents Global Medical Affairs at international/regional scientific congresses.
• Collaborates with Marketing to provide medical input for commercial activities related to PKU therapy.
• Plans and leads the PKU publication strategy.
• Supervises medical information processes from the PKU perspective; ensures appropriate utilization of Medical Services for PKU.

Qualifications

• Advanced Degree Required (PharmD, PhD or MD) with at least 10 years of progressively responsible experience in the pharmaceutical/biotech sector, and ideally 7+ years in medical affairs.
• Global or Regional Medical Lead experience (supporting the U.S.) in a rare disease; metabolism experience strongly preferred.
• Experience supporting key markets (including US).
• Proven leadership and progression in medical affairs, with strategic and tactical expertise.
• Ability to evaluate medical literature and develop effective medical/communication strategies.
• Ability to analyze data and craft a compelling scientific narrative for relevant audiences.
• Strong understanding of compliance in medical affairs.
• Track record in commercializing orphan/rare disease or specialized drugs; payer experience.
• Experience building cross-functional teams; ability to influence KOLs and key customers; engage patient organizations.
• Proficiency with Microsoft Office; excellent written and verbal communication; ability to convey complex science clearly to diverse audiences.
• Ability to work in a global, multicultural environment; independent and collaborative work style in a fast-paced matrix setting.
• Analytical, with strong problem-solving, prioritization, and time management skills.
• Fluent in English (verbal and written); PKU or related metabolism expertise preferred.
• Experience in orphan/rare diseases, neurology and/or genetics; knowledge of US payor interactions.

Additional Requirements

• Travel: 40% global travel.
• Location: Office-based or remote with travel to PTC offices; candidates based in the U.S., preferably East Coast.