The Senior Director, Biostatistics – Gene Therapy provides technical leadership and operational oversight for the design and implementation of clinical development programs and the associated clinical trials for the Gene Therapy therapeutic areas and associated gene therapies in aromatic Amino Acid Decarboxylase (AADC) deficiency and Friedreich Ataxias. This includes, but is not limited to, identifying optimal study designs, defining data analysis strategies, implementing statistical analysis plans, interpreting the resulting findings and crafting and communicating key messages to internal audiences and selected key external stakeholders. The incumbent also provides strategic direction, and assumes accountability for, biostatistical activities on on-going BLA. S/He may interact with regulatory agencies on matters related to the data for assigned clinical programs and/or trials.
The incumbent works cross-functionally with internal departments and external resources on Biostatistics related issues.
The Senior Director, Biostatistics – Gene Therapy ensures adherence to relevant regulatory requirements and company Standard Operating Procedures (SOPs) as appropriate.
Ensures the timeliness and quality of statistical deliverables for clinical trial results, exploratory and meta-analyses, publications activities, scientific presentations and promotional material.
Develops solutions to statistical and data analysis issues for clinical, regulatory, and commercial teams. Communicates solutions cross-functionally.
Participates in vendor evaluation, selection, and management as required.
Collaborates on protocol development including study design, endpoint selection, and power and sample size assessment.
Authors and manages the implementation of statistical analysis plans for clinical studies and integrated summaries for regulatory submissions.
Authors and reviews statistical and outcome sections of study reports and submissions.
Provides support for statistical deliverable(s) for BLAs and other submissions.
Participates in due diligence evaluation of design and clinical trial data of external partnership opportunities as needed.
Performs other tasks and assignments as needed and specified by management.
Advanced degree in Statistics or a related statistical discipline and a minimum of 9 years of progressively responsible statistical experience in a pharmaceutical, biotechnology, contract research organization (CRO).
Hands-on statistical experience supporting clinical studies, working with clinical teams and interacting with regulatory agencies.
Demonstrated understanding and applied knowledge of statistical theory and its application in the pharmaceutical/biotechnology industry.
Ability to influence without direct authority.
Excellent verbal and written communication and skills.
Ability to work independently and collaboratively, as required, in a fast-paced, matrixed, team environment consisting of internal and external team members.
Analytical thinker with excellent problem-solving skills and the ability to adapt to changing priorities and deadlines.
Excellent planning, organization and time management skills including the ability to support and prioritize multiple projects.
Thorough/detailed understanding of regulatory requirements and guidance documents.
Prior BLA, NDA and/or MAA experience.
Travel: 5- 15%
This position can be based out of our South Plainfield, NJ or Lynnfield, MA locations.
PTC Therapeutics is an equal opportunity employer. We welcome applications from all individuals, regardless of race, color, national origin, gender, age, physical characteristics, social origin, disability, religion, family status, pregnancy, sexual orientation, gender identity, gender expression, disability, veteran status or any unlawful criterion under applicable law. We are committed to treating all applicants fairly and avoiding discrimination.