Director, Global Health Economics and Outcomes Research and Real World Evidence (HEOR & RWE)

Bannockburn, IL, US
Sep 08, 2019
Required Education
Position Type
Full time

Ready to join a team committed to moving gene therapies into the clinical and commercial settings for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science to treat rare and life-threatening genetic diseases starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101 (ZOLGENSMA®). We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, Inc., a Novartis Company , is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality, and for the treatment of SMA Type 2. The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

The Director, Health Economics and Outcomes Research and Real-World Evidence (HEOR & RWE), is responsible for developing and implementing (hands on) global strategic research and for managing the planning, implementation and publishing of HEOR and RWE focusing on disease registries, observational and retrospective epidemiologic studies in disease areas of interest to AveXis and through the product life cycle. The Director will oversee registry operations and vendors management globally. The Director will be responsible for providing insights to Early Development, Clinical R&D Commercial/Marketing/Market Access, Medical Affairs regarding the development of registries and HEOR and RWE to support development and commercialization of AveXis products.


  • Develop and implement Global HEOR research plans and coordinates fulfillment of strategic research plan across all commercial, R&D, and medical affairs.
  • Implement innovative approaches to the design of rare disease registries.
  • Leverage existing registries and EHR databases to support regulatory requirements, address payer evidence generation and help reimbursement activities.
  • Research and perform critical analyses of medical and scientific evidence including systematic reviews, RWE, and economic evaluations as the basis for HTA assessments, disease management guidelines, and formulary evaluations.
  • Oversee and conduct pre-, peri-, and post-launch HEOR research, either through external vendors and consultants or internal staff.
    • Ensure development and implementation of RWE research program to support value proposition including:
    • Perform non-interventional studies focusing on Registries, observational and longitudinal database studies
    • Manage vendors in supporting the SMA disease registry
    • Oversee the day to day registry operation
    • Engage external KOLs to provide insights to AveXis registries and observational research
    • Provide input into registry data analytics and methodologies
    • Document unmet needs by demonstrating economic and humanistic burden of illness in planned/approved indications for products
    • Contribute to the design, HEOR endpoint selection, reporting and analysis of clinical studies including subgroup analysis
    • Demonstrate product value versus comparator through mixed treatment comparison or indirect treatment comparison methods as appropriate
    • Develop cost-effectiveness model-based economic evaluations, budget impact analyses, cost-offset analyses and burden of illness studies
    • Support scientific content output (abstracts/manuscripts/posters) based on HEOR & RWE analyses for products
    • Conduct patient and caregiver surveys to support the value proposition
    • Develop high quality core value and AMCP dossiers
  • Attend medical/scientific, pharmacy, and HEOR professional meetings to keep up with changing trends and remain current within the field relating to new research and medical trends affecting each product area.
  • Possesses excellent communication and interpersonal skills especially over departmental and geographical boundaries.
  • Responsible for operation, resourcing and management of the HEOR activities.


  • PhD, PharmD, MD or DO required.
  • 7 years of HEOR and RWE (i.e., registries) experience.
  • Management experience is preferred.
  • Possesses excellent clinical and reimbursement knowledge.
  • Experience working in a global organization highly desirable.
  • Experience working in a matrixed environment highly desirable.

The level of this position will be based on the final candidate's qualifications.

Please note this job description is not designed to cover or contain a comprehensive listing of activities, duties or responsibilities that are required of the employee for this job. Duties, responsibilities and activities may change at any time with or without notice.

AveXis is committed to creating a diverse environment and is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation or veteran status.