Amicus Therapeutics

Director

• Serve as the medical director for the US region exclusively dedicated to Fabry Disease.
• Partner with the Global Medical Lead (GML) and International RML to develop medical plans.
• Represent US Medical Affairs (USMA) in key governance meetings, review committees, and cross-functional leadership teams.
• Establish and maintain strong working relationships with external Global and National Fabry KOLs, Fabry Centers of Excellence, and Patient Advocacy Organizations.
• Support Global scientific advice seeking events and US scientific educational programs and events for healthcare providers.

Director, Regional Medical Lead

• Serve as the medical director for the US region exclusively dedicated to Pompe Disease.
• Partner with the Global Medical Lead (GML) to develop medical plans for therapeutic products.
• Ensure alignment of medical activities across the US region.
• Represent US Medical Affairs (USMA) in governance meetings and cross-functional leadership teams.
• Support Global Medical Operations to execute Pompe data generation and drive publication planning.
• Establish networks with external experts in Pompe Disease for research opportunities.
• Deliver clinical and scientific input into medical strategy and activities.

Director, Regional Medical Lead – Pompe

Operations

• Supporting the U.S. Market Access team in contract administration, execution, and performance of Trade Account purchase, distribution, and service agreements.
• Liaise between Amicus Assist and the third-party logistics provider, ICS, customer service function for Trade account set-up, pricing and chargebacks.
• Facilitate account set-up with the third-party logistics provider for Hospital, 340B, infusion clinic and other “Buy & Bill” accounts based on Pombiliti+Opfolda prescriber site of care requirements.
• Collaborating cross-functionally to partner with US Finance and FP&A functions by providing advice on government pricing matters for current and potential products and provide support for Gross-to-Net assumptions and estimates to establish government pricing and be in compliance with Government Programs.
• Submitting monthly, quarterly, and periodic product pricing information and other pertinent data to CMS, the Department of Veterans Affairs and States requiring price transparency. This would include reporting of Best Price, Average Manufacturer Price, Average Sale Price, Non-Federal Average Manufacturer Price and Public Health Service pricing for applicable products.
• Staying current on State Price Transparency pending and enacted legislation and maintain standard operating procedures and Amicus policies on price reporting, including updates for new products.
• Manage the outsourced service provider, currently IntegriChain for the modeling and preparation of report submissions and ensures timely approval by Controller or CFO.
• Developing a strong understanding of contract structure and strategy of the Commercial Operations business segments and their impact on Medicaid Average Manufacturer Price, Best Price, and Average Sales Price calculations, as well as the Non-Federal Average Manufacturer Price and Public Health Service pricing to support updates to Reasonable Assumptions and updates to legislative changes.
• Analyzing Payer contract requests for business and pricing impact and support business case development.
• Monitoring the activity of contracts, including varied price concessions such as rebates, fees etc., to evaluate the impact on AMP, Best Price, ASP, NFAMP, and PHS calculations in accordance with the policies of CMS, the VA, and the PHS.
• Understanding the Amicus class of trade and market segment structure.
• Preparing periodic and ad hoc pricing reports with interpretive commentary for management review, including competitor analysis on similar products and services.
• Providing accurate analyses in support of numerous internal and external audit inquiries.
• Maintaining and operationalizing the VA/Federal Supply Schedule Agreement through compliance with the Federal Government Regulations (FAR) and submit modifications as necessary, including updating annual submissions (SAM.gov, etc.).
• Working cross-functionally and cross-departmentally to resolve data, system and processing issues prior to calculations being processed.
• Managing historical pricing, monitoring CPI, implementing product price updates with trade partners, new product launch and pricing compendia notifications and track competitor products pricing.
• In support of Amicus Assist, this position maintains the integration of Specialty Pharmacy dispense data with Patient CRM and supports the implementation and maintenance of Patient CRM, liaises with Quality in support of audits and with Market Access on patient status.
• Developing and supporting data infrastructure and reporting for the Amicus Assist team and for Sr Leadership pertaining to Patient CRM.
• Assisting with other ad-hoc projects, as needed

Senior Manager, Trade Operations, Access Analytics & Government Pricing

Clinical Research

• Lead the clinical development strategy and prepare Clinical Development Plans (CDP) for clinical programs and clinical trial protocols
• Responsible for assets from early phase through to registration and approval process
• Oversee and potentially supervise junior clinical team members; provide hands-on tactical work in project teams
• Collaborate with clinical/preclinical teams and cross-functional partners across Development Organization
• Work with Clinical Trial Investigators and Clinical Research Organizations on trial site selection, training and ongoing study conduct
• Ensure studies are performed with highest quality; perform data review and analysis and present clinical data
• Lead generation of clinical study reports and participate in preparation of regulatory documents (Investigator Brochures, CSRs, Briefing documents)
• Participate in face-to-face meetings with regulatory agencies and contribute to global regulatory submissions
• Maintain high level of medical science and clinical development expertise
• Collaborate on business development activities
• Perform responsibilities in compliance with company policies, SOPs and guidelines
• Represent Clinical Research function on program-aligned governance, operational committees and corporate projects as required
• Ability to travel, both domestic and international (25%)

Executive Director, Clinical Research

• Lead the development and execution of CMC analytical strategies for large- and small-molecule programs
• Oversee analytical method development, validation, and characterization testing for clinical and commercial products
• Provide subject matter expertise for large molecules and support investigations and regulatory information requests
• Collaborate cross-functionally with Tech Ops, Quality, CMC Regulatory Affairs, and Global Regulatory Affairs to drive program execution
• Oversee outsourced analytical packages and partner with CDMOs to ensure scientific rigor and regulatory compliance
• Partner with QC to implement continuous improvement initiatives and ensure timely project progression
• Provide strategic oversight of analytical content for regulatory submissions and represent Analytical Sciences on the CMC Leadership Team
• Lead, manage, and develop a team of 3–5 direct reports
• Engage with external industry organizations and consortia to benchmark best practices

Senior Director, Analytical Sciences

Marketing

• Lead KOL identification, mapping, and strategies across Amicus’ U.S. therapeutic areas
• Organize and coordinate advisory boards, expert panels, educational events, and other initiatives to gather insights
• Lead US Congress KOL engagement planning
• Continuously monitor and synthesize external scientific trends, expert feedback, and market dynamics
• Collaborate closely with cross-functional teams to translate thought leader insights into strategies, plans, and activities (short- and long-term KOL plans for Pompe, Fabry, and FSGS)
• Lead execution of strategic plans for engaging established and emerging KOLs aligned to commercial and product objectives across the patient journey
• Identify and engage emerging clinicians, researchers, and academics with potential to become future leaders
• Support professional growth of emerging thought leaders via experiential leadership and engagement opportunities
• Ensure all KOL engagement activities comply with Amicus policies, industry codes, and applicable laws and regulations
• Collaborate with Legal, Compliance, and Regulatory to identify, assess, and mitigate compliance risks
• Lead training and communication initiatives to reinforce compliance standards
• Report to the Executive Director, Marketing and partner with US Therapeutic Brand leads, US Medical Affairs, Program Management, Legal, Compliance, Regulatory, Global Marketing, and Commercial Leadership
• Execute both field and home office responsibilities; travel up to 50% to support KOL engagement and HQ meetings

Senior Director, Key Opinion Leader Engagement and Marketing

• Lead multi-product rare disease product portfolio and aligned team to deliver financial, strategic and tactical objectives
• Develop and implement US commercial strategic and operating plans; convey clear vision to stakeholders
• Translate market changes and data into executable business and marketing plans, anticipating and reacting to competitive landscape
• Provide leadership in selection, expansion, motivation and retention of a high-performing Marketing organization; mentor and coach direct reports
• Manage the US brand planning process, including brand optimization, launch readiness, budget planning and quarterly business reviews (QBRs)
• Build and manage resources including annual objectives, budgets and agency management
• Steer strategy and execution of marketing and branding activities; ensure coordination and consistency of brand positioning, messaging and differentiation globally
• Establish strong relationships and collaborative joint ventures with key stakeholders to create global advocacy
• Ensure marketing activities comply with internal policies, industry codes (e.g., PhRMA, EFPIA) and applicable laws and regulations
• Partner with Legal, Compliance, and Regulatory teams to proactively identify, assess and mitigate risks associated with marketing campaigns, product launches and stakeholder engagement
• Serve as a key member of the US Leadership Team and work closely with Sales, Commercial Strategy and Operations, Market Access, Patient Experience and global marketing to align priorities and support the sales force

Executive Director, Marketing

Manager

• Work closely with patient/caregiver to support access to therapy
• Provide day-to-day oversight and coordination of caseload to ensure timely completion of activities and tasks
• Accurately document and maintain case history for all assigned patients
• Act as single point of contact performing preliminary benefits investigations, prior authorization and appeal support
• Communicate patient benefits and responsibility timely and accurately to patients and healthcare providers
• Complete a pre-screen to determine eligibility for financial support
• Troubleshoot complex cases while interfacing with internal and external stakeholders to ensure optimal start to therapy
• Professionally field incoming telephone calls and answer patient and medical office inquiries
• Provide ongoing adherence and compliance support for patients
• Provide back-up coverage for other Case Managers
• Attend patient and commercial strategy meetings where applicable
• Ensure compliance with legal and regulatory mandates and serve as a compliance role model

Case Manager

Compliance

• 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 Quality Lead, Quality Compliance and Operational Excellence will oversee the Global Process for Product Complaints for Marketed Products and Clinical Trails and will also assist to drive organizational performance through workforce empowerment using collaboration, proven lean six-sigma tools, and change management methodologies.
• This role will emphasize compliance with SOPs and applicable regulations. They will interact with key stakeholders in the organization for timely and accurate receipt of complaint reports. This role will lead/facilitate complaint investigations, report complaint trends and escalate issues to Management. They will lead the training of Amicus personnel for the responsibilities and requirements for the identification and handling of complaints. This role will also interact with Health Authority inspectors as the Global Complaints Process Lead/SME.
• They will assist with supporting and developing a Quality Compliance and Continuous Improvement culture ensuring that Global Quality Operations are carried out in a compliant and efficient manner including leading and/or facilitating internal audits.
• They will compile and author Annual Product Quality Review (APQR) global summary reports for all commercial products.