Director, Biostatistics

Sarepta Therapeutics•Cambridge, MA

2d•Hybrid

About The Position

The Director, Biostatistics will work with a cross-functional team as the project biostatistician responsible for statistical activities at the compound level or study level in DMD or siRNA platforms. This role requires a hands-on analyst who can explore data deeply, partner with clinical development to shape scientific questions, and deliver rapid insights using modern analytical tools. This is an opportunity to work at the forefront of precision genetic medicine, making an impact on the development of transformative therapies that may change the lives of patients. The ideal candidate is a good communicator, high-energy, self-motivated, and forward thinking. Experience working in rare disease is desirable, but not required.

Requirements

Ph.D. (strongly preferred) with 9+ years or Master’s degree with 10+ years of experience in statistics or related discipline in the pharmaceutical or biotech industry
Demonstrated ability and experience in the design, analysis, and reporting of clinical trials
In-depth knowledge of statistical methods for clinical trials, including both frequentist and Bayesian approaches
In-depth knowledge of FDA, EMA, and ICH regulations and guidelines
Proficient in statistical programming in SAS and R; Python and other languages a plus
Proficient in statistical design software such as EAST
Ability to concurrently lead statistical efforts for multiple studies
Understanding of data standards, including SDTM and ADaM
Ability to oversee statistical services provided by CROs and/or contractors
Ability to work independently, guide team and junior biostatisticians, and act with initiative to address issues
Ability to collaborate effectively with colleagues from all other functions
Excellent written and oral communication skills, including the ability to effectively communicate statistical concepts in a clear and concise manner
Willingness and ability to perform hands-on data exploration using R and/or Python, in addition to SAS
Enthusiasm for learning and harnessing modern AI tools and workflows to enhance analytical productivity and scientific insight
Demonstrated experience partnering with clinical development teams to shape analytical strategy, not just execute pre-defined plans

Nice To Haves

Experience in NDAs, MAAs or other regulatory submissions preferred
Knowledge of gene therapy, rare disease, or neuromuscular diseases is desirable, but not required
Familiarity with external control arms, real-world evidence, or observational study methods is a plus

Responsibilities

Lead collaboration with internal and external (e.g., CRO) team members to coordinate the planning and execution of statistical deliverables
Lead/conduct statistical modeling to enable robust and efficient statistical designs and to address identified or potential statistical issues arising in studies or programs
Contribute to clinical protocol development, including authoring of the Statistics section and reviewing of other sections by applying statistical principles
Author or review statistical analysis plans for clinical trials and for integrated summaries of safety/effectiveness (ISS/ISE), and author or oversee the development of shells for tables, figures and listings
Review case report form (CRF) designs to ensure data collection meets the requirements of statistical analyses
Design and specify randomization schedules; review and approve test randomization lists
Provide statistical input to data monitoring committee (DMC) charters, independent review charters, and other study-level documents
Review analysis dataset specifications
Perform QC/QA of statistical deliverables including validation of key analysis results
Perform ad hoc and exploratory statistical analyses as needed
Contribute to clinical study reports, including authoring of statistical methods and interpretation of the study results
Be accountable for assigned statistical activities in support of IND/NDA/MAA or other regulatory submissions
Address statistical questions/comments from FDA and other regulatory agencies, and review and address comments by IRB/ECs
Support and contribute to the preparation of publications, including manuscripts, posters and oral presentations
Contribute or lead standardization and process improvement efforts for Biostatistics and contribute to cross-functional process improvement efforts
Represent Sarepta regarding statistical issues in meetings with external parties including regulatory agencies and corporate partners
Serve as a mentor to junior team members
Conduct hands-on exploratory analyses beyond pre-specified SAP deliverables, probing unexpected results, testing sensitivity scenarios, and generating rapid insights to inform program decisions
Partner proactively with Clinical, Medical, and Regulatory colleagues to identify and prioritize scientific questions that warrant exploratory investigation, bringing Biometrics perspective early, not only at the analysis stage
Leverage modern analytical tools (R, Python, interactive visualization apps) alongside SAS to accelerate data-to-insight timelines, while maintaining clear documentation and traceability of exploratory work
Contribute to fit-for-purpose validation practices, distinguishing between outputs intended for internal learning versus those that require production-grade validation for external communication or regulatory use