Director, Global HEOR, Duchenne Muscular Dystrophy, Gene Therapy

New York City, New York
Jul 30, 2021
Required Education
Position Type
Full time


This Director, Health Economics & Outcomes Research (HEOR) role is a unique opportunity for a seasoned HEOR professional to innovate while launching the next era of pharmaceutical innovation, Gene Therapies.  Novel solutions are required for unique issues such as:

  • How to create an integrated & comprehensive natural history across all elements of patient experience in a rare disease with disparate Real World data sources
  • How to conduct economic modelling of a one-time, high value medicine with undetermined long-term durability that will replace widely different standards of care depending on the country
  • How to create outcomes-based agreements that give payers confidence in the value of therapy, in a situation where the metrics are unclear, in a way that is feasible given clinical practice and data systems

This role will support the strategic goals of the Rare Disease (RD) Business Unit (BU) by leading the creation of data, analysis and tools for optimal reimbursement and access for Duchenne Muscular Dystrophy (DMD) GTx, currently in Phase 3 development. This role directly impacts the ability to achieve business objectives on a global basis by creating new innovative approaches to HEOR & Access issues, providing strategic guidance and robust evidence development to secure optimal global patient access and demonstrate the value of our product.

This role will serve as the Global Director, HEOR to support product development and eventual launch of DMD GTx worldwide. The HEOR Director will function with a one-Patient and Health Impact (PHI) mindset and work in a closely aligned fashion with the full team to ensure there is a single and coordinated view on strategy and delivery from PHI to the RD business. The Director will collaborate with Global medical, commercial, and other cross-functional teams to develop and implement health outcomes research strategies to support DMD GTx globally.


  • Lead the development of the Health Economics & Outcomes Research (HEOR) strategy to support the value of DMD GTx in close partnership with Market Access and the cross-functional team.
  • Lead the execution of HEOR studies and projects in alignment with the global HEOR strategy and demonstrate the value proposition. This may include some or all of the following:
  • Lead the timely development of pre-launch deliverables in alignment with Bold Move 2.3 requirements for Early Stakeholder Engagement in a Create archetype market. 
  • Lead the development of pre launch deliverables including global value dossiers (GVDs), evidence blueprints, to successfully support global launch and post-launch reimbursement and access requirements in conjunction with the regions/countries.
  • Lead the development of economic models and innovative tools capable of illustrating the value of GTx with the rigor of analysis expected by payers.  This will include dealing with novel modelling issues
  • Take a leadership role in the design of Outcomes Based Agreements including clinical end point selection, data analysis to inform the agreements and leading the information systems implementation in partnership with a diverse group of internal and external stakeholders
  • Develop real world evidence generation strategies and execute studies (e.g., burden of illness, comparative effectiveness studies) in an environment of incomplete data sets where integrating disparate data sets in a robust way will be key.  Conduct this work in partnership with the Real World Evidence workstream which involves leveraging clinical and regulatory work to abide by FDA & EMA requirements
  • Innovate by leveraging the humanistic and economic endpoints in the clinical trials to build comprehensive trial-based evidence in line with overall clinical strategy. Provide strategic input in the communication of relevant patient population and comparators within clinical trials to enable appropriate patient access with payer and regulatory decision makers.
  • Validate and communicate new Patient Reported Outcomes measures, as appropriate, for inclusion across clinical trials, registries, and prospective real-world studies.
  • Lead the development of appropriate data dissemination plans and communicate evidence generated via publications and conference presentations.
  • Identify opportunities to partner with external customers to conduct HEOR projects in support of asset strategies
  • Lead the coordination of input from local country teams on evidence needs and ensure that the input is appropriately incorporated to develop and integrated evidence strategy to support global market access needs at time of launch.
  • Collaborate with local health and value affiliates and enable local model adaptations and updates throughout the product life cycle.
  • Provide strategic guidance and content guidance in reimbursement negotiations for Pfizer products.
  • Assess, synthesize, and interpret relevant literature and communicate findings to internal and external stakeholders
  • Execute and manage research projects with external collaborators/vendors to ensure timely completion and quality of deliverables as well as budget/legal compliance.


Candidate demonstrates a breadth of diverse leadership experiences and capabilities including: the ability to influence and collaborate with peers, develop and coach others, oversee and guide the work of other colleagues to achieve meaningful outcomes and create business impact.

  • Minimum 9+ years Masters degree (MSc, MPH) in health services research, public health, epidemiology, or health economics with a preferred Doctoral degree 7+ years (PhD, PharmD, DrPH, ScD) in health services research, public health, epidemiology, or health economics.
  • Minimum of 7+ years of experience in HEOR or relevant related fields
  • Innovative, self-starter who can address challenges with a minimum of supervision while working in aligned fashion within the PHI & DMD cross functional teams
  • Able to independently engage all levels of stakeholders through a strong customer orientation, strategic thinking, leading change, and building collaborative partnerships in a matrix environment
  • Working knowledge of the technical and methodological aspects of registries and observational study design and implementation. Capable of managing registry and non-interventional study projects.
  • Strong methodological skills (study design, data analysis and interpretation) in clinical trials, epidemiology, or health services research is required
  • Understanding of Pharmacoeconomics and Health-related Quality of Life
  • Excellent oral and written English communication skills required
  • Skilled in functioning within a matrix organization where managing through influence is required
  • Excellent interpersonal skills required: ability to understand and respond to multiple internal and external customers
  • Strong project management abilities (contracting, budgeting, vendor management) essential.
  • Demonstrated ability to manage multiple projects involving complex processes, significant budget, competing deadlines and rapidly shifting priorities
  • Ability to influence key members of medical and commercial teams constructively and without conflict


  • Knowledge and experience in Rare Disease area is desirable
  • Experience with HTA organizations such as NICE, SMC, PBAC is desirable
  • Understanding of emerging Japan HTA methodology also desirable


Other Job Details:

Last Date to Apply for Job: August 15th 2021

Additional Location Information: North America-remote, Europe-remote


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EEO & Employment Eligibility

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Market Access