Senior Director, Health Economics and Outcomes Research

Location: Lexington, MA Education level: PhD

Job category: Medical Affairs Target start date: 4/1/2025

Work Location: Hybrid Shift: 1st

uniQure is dedicated to bringing innovative therapies or unmet medical needs to patients. In doing so each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility should be part of our day-to-day working life by (i) living up to high quality standards and (ii) by recognizing the sense of urgency in areas of high medical need.

This position will be responsible for global Health Economics and Outcomes Research strategy and tactics across uniQure’s pipeline and will serve as the functional lead for HEOR. The Senior Director, Health Economics and Outcomes Research, will be a key member of the Global Medical Affairs team. Working under the leadership of the Global Head of Medical Affairs, this individual will develop strategies to demonstrate the clinical, economic, and humanistic value of uniQure’s products and lead execution of health outcomes research and economics modeling tactics and evidence generation plans, creation of global value dossiers, as well as provide input into clinical trial design to deliver robust market-relevant evidence to support broad and appropriate access to uniQure’s therapies.

Responsibilities

Key result areas (major duties, accountabilities and responsibilities)

• Define the HEOR strategy for uniQure’s gene therapy programs
• Lead and conduct methodologically rigorous and feasible studies that address priority US HEOR evidence needs. Such studies may include:
• Longitudinal retrospective studies of existing databases to assess patient characteristics, treatment patterns, and associated clinical, economic and/or patient-reported outcomes
• Retrospective or prospective evaluations of disease natural history and treatment patterns, including drug utilization and adherence
• Cross-sectional surveys of patients, caregivers, health care providers/practitioners, and/or payers
• Assessments exploring the burden of illness, patient preference studies and/or comparative effectiveness
• Models of cost-effectiveness and budget impact; indirect treatment comparison models and methodologies
• Collaborate with Clinical Development to incorporate HEOR perspectives and strategies in the clinical development plan and provide input into clinical protocol design to capture HEOR endpoints relevant to payers, physicians and patients
• Lead development of health economics models in collaboration with Market Access
• Develop product value narrative and work with colleagues across the organization to educate on the value proposition of the company’s products to internal and external stakeholders
• Prepare global value and reimbursement dossiers and oversee submission to relevant HTAs; support HTA/payer interactions in conjunction with Market Access as needed
• Manage external vendors and HEOR budget as required to achieve deliverables
• Understand HTA/payer requirements globally and actively monitor the changing global health care landscape to anticipate future resource needs of internal and external stakeholders and prepare the organization
• Serve as HEOR subject matter expert in cross-functional teams; provide methodological & analytical expertise regarding the utilization of real-world data.
• Develop HEOR publications for dissemination in appropriate scientific congresses and peer reviewed journals in alignment with program publication strategy
• Maintain a high level of knowledge with regards to the scientific, clinical and commercial developments in the therapeutic area and an understanding of pharmacy management/medical policy trends/techniques used by payers to make informed decisions about payer strategy and OR plans and tactics
• Advanced degree (MD, PhD, MS/MPH, or PharmD) in a health economics, epidemiology, health services research, public health, scientific discipline, or related fields.
• 8-10 years of experience in HEOR and/or market access in the pharmaceutical/biotechnology industry; experience in rare disease and gene therapy strongly preferred
• Demonstrated technical and strategic excellence in designing, conducting, and interpreting health economics and outcomes research, with a particular emphasis on quality of life and other patient-reported outcomes assessments
• Background in Huntington’s Disease is a plus, but not required
• Significant experience with both drug development and marketed products
• In-depth knowledge and experience in or with a variety of US/EU payer segments, including but not limited to: HTAs, health plans, PBMs, providers, commercial, and government
• Must be fully cognizant and adhere to all regulatory, legal, and compliance standards for Medical Affairs activities
• Ability to travel up to 30% of the time, both US and internationally
• Demonstration of an ability to embody uniQure’s values
• Strong interpersonal skills and demonstrated leadership in working in cross functional environment and influencing management decisions
• Effective planning, prioritization, management, and organizational skills
• Ability to navigate ambiguous, complex, and difficult situations
• Ability to interface with multiple stakeholders, including clinicians and payers
• Possess business and analytic skills and an in-depth understanding of pharmaceutical business practices that impact asset development and commercialization
• Show expert awareness and the ability to understand and make recommendations concerning the evolving health care systems