Director, Regional Medical Lead, US Pompe

SUMMARY OF POSITION Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases.  The Regional Medical Lead (RML) will serve as the medical director for the US region exclusively dedicated to Pompe Disease.  This is a strategic medical leadership position that partners with the Global Medical Lead (GML), and the International RML to develop the medical plans for novel therapeutic products for patients with Pompe Disease and ensures the implementation and alignment of medical activities across the US. As an MD/PharmD/PhD with experience as a Medical Director at the global or regional level, the US RML will play a key role in developing and executing our wider vision for Medical Affairs. The US RML will represent US Medical Affairs (USMA) in key governance meetings, review committees and cross-functional leadership teams related to the Pompe franchise and will be responsible for communicating updates to the USMA team. The US RML supports the GML to establish the Global Medical Strategic Imperatives for products in Pompe Disease. In addition, the US RML will work collaboratively with Global Medical Operations and the International RML to execute Pompe data generation - IISs, Phase IV studies, disease registries, collaborative research studies and non-interventional studies - and drive US Pompe publication planning and medical communications. The US RML will ensure that medical activities delivered across the region are aligned to the overall Global Strategy and objectives in Pompe Disease. The US RML will partner with USMA, US Pompe Marketing and US Patient Advocacy to ensure scientifically accurate content development, support advice-seeking activities, and assist with training development and delivery as needed. The US RML will be responsible for establishing and maintaining strong working relationships and partnerships with external Global and National Neuromuscular and Genetic KOLs, Pompe Centers of Excellence and Patient Advocacy Organizations. 

Roles And Responsibilities

• Responsible for the development and execution of the Pompe medical activities within the US Medical Plan and supporting delivery of medical activities when required.
• Ensure that the medical activities are aligned to the Global Medical Strategy for the disease area and product(s) and adapted to fit US regional needs.
• Accountable to deliver clinical and scientific input from the US region into the development of plans at the Pompe Integrated Evidence Planning Team, Global Pompe Publication Planning Team, Global Pompe Brand Leadership Team, and other cross-functional teams as required.
• Proactively cascade decisions from Governance meetings and committees to the USMA team and other cross functional teams.
• Develop and lead US scientific advice seeking events, coordinating with insights from the field, and delivering outputs through appropriate channels.
• Support Global scientific advice seeking events as appropriate.
• Develop and lead US scientific educational programs and events for healthcare providers in partnership with cross functional teams.
• Establish networks with external experts in Pompe Disease to expand potential research opportunities aligned with the Global and US Medical Strategy.
• Act as a scientific expert in the review of investigator-initiated research proposals and independent medical education submissions.
• Develop and lead US Pompe collaborative research proposals, propose and support US RWE and HEOR analyses, and US focused sub-analyses from ongoing and completed clinical research studies
• Support the development and execution of a US Pompe Digital Strategy for Pompe and collaborate with the International Pompe RML to jointly leverage digital content
• Responsible for the clinical and scientific training in Pompe including disease and product related training events for USMA and cross-functional stakeholders.
• Develop, communicate, and maintain in-depth scientific knowledge of Amicus Therapeutics Pompe products and pipeline and other products within the Pompe therapeutic area.
• Assist the US MSL team to deliver accurate and timely medical information related to Amicus Therapeutics Pompe products and Pompe disease to US healthcare professionals.
• Partner with US Marketing on US Pompe thought leader engagement planning and support
• Ensure that all activities are conducted in a fully compliant manner in accordance with Amicus policies and country laws and regulations.
• Contribute to the development and management of US Medical Affairs Pompe budget.
• Provide clinical and scientific input for the US reimbursement dossiers as needed.
• Serve as Medical Reviewer for US Pompe scientific and promotional materials/activities and support Global reviews as needed.

Educational Requirements

• Advanced doctorate level degree (e.g., PharmD, PhD, MD) or equivalent 

Professional Work Experience

• 10 years of Medical Affairs or related experience within the biotech/pharmaceutical industry required
• 2+ years as Associate Medical Director/Medical Director within Medical Affairs required
• Prior experience in orphan drugs and rare diseases preferred
• Prior experience in Genetic Disorders, Respiratory, Neurology or Neuromuscular Medicine preferred
• Experience working in cross-functional teams at the brand or disease area levels in country, regional, or global organizations is preferred
• Experience in digital health and AI technologies use in Medical Affairs preferred
• Knowledge of US pharmaceutical regulations and guidelines preferred

Required Attributes

• A passion for helping patients with rare conditions.
• Demonstrates Amicus Core Values
• Ability to think strategically and tactically from a medical perspective
• Excellent leadership skills
• Demonstrated business acumen and analytical thinking skills
• Ability to work independently and collaboratively within diverse cross-functional teams
• Strong organizational skills with the ability to prioritize and manage multiple time-sensitive projects simultaneously.
• Strong verbal and written skills with ability to explain complex, challenging topics across various audiences
• Fluent in English, written and spoken

Travel

• Travel will be required (up to 20%)

Compensation And Benefit Summary

• The U.S. base salary range for this full-time position is between $230,000 and $260,000 year.  Within the range, individual pay is determined by work location and additional factors, including job-related skills, experience, and relevant education or training.  In addition to the base salary, employees may be eligible for various benefits, including health insurance, retirement plan (401K), paid time off, performance-based bonuses, stock options, restricted stock units, and other discretionary awards. Details of participation in these benefit plans will be provided during the hiring process.

We value an inclusive environment where individual experiences and perspectives are celebrated, and voices from the rare disease community are heard. Amicus is an equal opportunity employer and will judge all applicants based on their qualifications for the job, without regard to race, color, sex, religion, national origin, age, disability, sexual orientation, gender identity, protected veteran, disability status or any other characteristics protected by applicable federal, state or local law.