Global Medical Affairs Therapy Area Lead - PKU; East Coast

PTC Therapeutics is a global commercial biopharmaceutical company. For over 25 years our team has been deeply committed to a unified purpose:
Extending life’s moments for children and adults living with a rare disease.

At PTC, we cultivate an inclusive culture where everyone feels valued, respected, and empowered. We welcome candidates from all backgrounds to join our team, fostering a strong sense of belonging.

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Global Medical Affairs (GMA) at PTC is committed to helping health care providers make evidence-based decisions to optimize health outcomes in their patients with rare diseases. The Global Medical Affairs – Global Therapeutic area Lead, PKU is responsible for defining the Global Medical Affairs vision, strategy and tactics to support PTC’s PKU business. He/she will partner with the Regional Medical Directors to identify and develop relationships with key opinion leaders (KOLs), identify treatment centers, ensure that the global medical affairs strategies and tactics for PKU are adopted and implemented at the local level and to ensure that the Global vision, strategy and tactics meet local needs.

He/she collaborates with marketing colleagues, to ensure that the strategic PKU medical affairs plan is aligned with the brand strategy. He/she will partner with the global medical affairs (GMA) enabling functions (i.e. Publications, Medical Information, Medical Education, Medical Communications, etc.) to lead and execute the medical strategy for sepiapterin, which includes: updating/executing established medical plan, maintaining the scientific communication platform (publications, congresses etc.),

planning and executing scientific advisory boards, review and approval of all grants (including IIS requests) and creating and delivering educational presentations both internally and externally. This individual will represent GMA on existing program team for sepiapterin, providing input into clinical development initiatives related to sepiapterin and will also support all life-cycle management initiatives. This role is viewed as the medical affairs subject matter expert on PKU and is expected to play a key role in educating KOLs, physicians, and internal colleagues.

This position ensures adherence to relevant regulatory requirements and company SOPs as appropriate.

• Develops and leads strategic global medical affairs activities related to PKU to accelerate commercialization efforts in launched markets and support successful commercialization in other markets to launch.
• Drives and supports communication and collaboration amongst regional and local Medical Affairs functions, GMA enabling functions and other teams in Commercial, Market Access, etc. to ensure well developed and executed PKU strategic plans; drives internal cross-functional partnerships for all PKU medical initiatives.
• Serves as the PKU subject matter expert in Global Medical Affairs. Partners with medical and commercial training to educate internal and external colleagues on the scientific and clinical aspects of PKU.
• Develops and finalizes global evidence generation plan in close collaboration with regional and country medical heads, GMA enabling functions and ensures alignment with other key internal stakeholders.
• Plans and leads the Investigator Initiated Studies (IIS) strategy for PKU.
• Effectively engages and builds relationships with PKU KOLs, internal and external stakeholders and healthcare professionals for the successful implementation of medical strategy for PKU; identifies and engages in scientific exchange with medical experts in the PKU therapeutic area.
• Serves as the strategic partner with respect to PKU to Clinical Development, Commercial Development and Life-Cycle Management (LCM) teams.
• Represents Global Medical Affairs at international and regional scientific congresses.
• Collaborates closely with the Marketing function to provide medical input for commercial activities related to therapy.
• Plans and leads the publication strategy for PKU.
• Supervises medical information process and service from the PKU indication…