Director, Global Real-World Evidence
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 Real-World Evidence (RWE) will be responsible for the execution of a regional portfolio of RWE initiatives and the operational leadership of the AveXis SMA Disease registry. Responsibilities include strategic registry deployment and the management of efficient and compliant processes in support of registry operations.
The Director will lead registry operations in assigned region (Americas, EMEA, APAC) and manage vendor and stakeholder interactions. This role is expected to partner with Early Development, Clinical R&D Commercial/Marketing/Market Access, and Medical Affairs colleagues regarding the further development of registries and observational research initiatives to support development and commercialization of AveXis products.
- Directs high-impact RWE initiatives (incl. observational studies, analyses of electronic health records (EHRs) and claims databases).
- Responsible for the deployment, day-to-day operations, resourcing, and support of the AveXis SMA Registry.
- Independently manages relationships with internal and external key stakeholders in support of observational studies activities.
- Anticipates and solves operational challenges independently and escalates issues for resolution.
- Drives internal and external publications including abstracts and manuscripts.
- Supports and participates in registry governance activities (steering committee, publications committee) and develops and implements global policies to improve the quality and efficiency of registry data; participates in the development and maintenance of relevant SOPs and Working Instructions.
- Partners with investigators and develops site guidelines for formulary evaluations.
- Leverages RWE to support regulatory requirements, addresses payer evidence generation and assist with reimbursement activities.
- Solves complex technical problems and partnership/clientele issues related to registry data generation with advanced experience and expertise.
- Attend medical/scientific, pharmacy, and other 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.
- Responsible for routine data management and advances AveXis RWE initiatives including regular performance reporting on data.
- Manages and conducts data integrity audits either through external vendors and consultants or internal staff, develops and implements related departmental policies and procedures and provides guidance in the proper use of registry data and data sharing.
- Works with the cross-functional representatives for each study to ensure that the database captures all critical data.
- Possesses excellent communication and interpersonal skills especially over departmental and geographical boundaries.
- BS, MS, required.
- PhD, PharmD, MD or DO highly desirable.
- Minimum 7 years of observational/epidemiological (i.e., registries) experience.
- Management and health informatics experience is preferred.
- Possesses excellent observational and epidemiological research experience.
- 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.