Director, Biostatistics

The Importance of the Role

The person in this position will be working 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. Beyond traditional SAP-driven deliverables, this role calls for 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.

The ideal candidate is a good communicator, high-energy, self-motivated, and forward thinking. Experience working in rare disease is desirable, but not required.

The Opportunity to Make a Difference

• 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
• 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

More About You

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