Sr. Director, Biostatistics & Statistical Programming

BioSpace; Malvern (Chester County); On-site; USD 210,000 - 320,000; Full time

Job summary

Ocugen in Malvern, PA seeks a Senior Director of Biostatistics & Statistical Programming to lead all biometrics activities across late-stage clinical development, regulatory submissions, and health authority interactions. You will shape trial design, endpoints, and regulatory documentation with expertise in ophthalmic gene therapies.

The role requires 15+ years in biostatistics and programming, with leadership experience and a strong track record in BLA/NDA submissions and regulatory strategy.

Qualifications

• PhD or MS in Biostatistics, Statistics, Mathematics, or a related quantitative discipline; PhD strongly preferred.
• 15+ years of progressive experience in biostatistics and/or statistical programming in biopharmaceutical development, with at least 5 years in a senior leadership role.
• Demonstrated success leading biometrics activities supporting late‑stage clinical development and regulatory submissions.
• Deep expertise in ophthalmology clinical development and regulatory submission strategies.
• Experience supporting gene therapy clinical development programs, preferably AAV‑based or other viral vector platforms.
• Expert‑level knowledge of GCP, ICH E9/E9(R1), ICH E6, CDISC standards and FDA/EMA regulatory expectations for advanced therapies.

Responsibilities

• Lead biometrics contributions to late-stage development, BLA/NDA submissions, and health authority interactions.
• Provide statistical leadership for protocol development, endpoint selection, sample size, and adaptive design considerations.
• Oversee SAP development, DMC interactions, interim analyses, and regulatory documentation.
• Ensure CDISC-compliant SDTM/ADaM datasets and regulatory submission readiness.
• Drive statistical innovation including Bayesian approaches and rare-disease trial design.
• Build and develop internal biometrics team and manage CRO/vendor oversight.

Skills

Biostatistics

Statistical Programming

Clinical Trial Design

Regulatory Submissions

FDA/EMA interactions

Education

PhD in Biostatistics

MS in Biostatistics

Job description

Purpose

The Director, Biostatistics & Statistical Programming serves as Ocugen’s most senior biometrics leader, providing executive-level scientific and operational oversight across all biostatistics and statistical programming activities in support of Ocugen’s ophthalmology-focused gene therapy pipeline. As a core member of the R&D leadership team, this individual is accountable for the strategic direction, design, execution, and regulatory delivery of biometrics across Ocugen’s portfolio including OCU400, OCU410, OCU410ST, and future development programs.

Description

This role leads all biometrics contributions to late-stage clinical development, BLA and NDA submissions, and health authority interactions, ensuring delivery of high-quality, submission-ready datasets, analyses, and regulatory documentation compliant with global standards. The Senior Director will be a recognized expert in ophthalmic endpoints, retinal imaging assessments, and the statistical methodology specific to rare inherited retinal diseases and AAV-based gene therapies. This individual will shape critical development and regulatory decisions and serve as Ocugen’s primary statistical authority in interactions with the FDA, EMA, and other global health authorities.

Strategic & Executive Leadership

• Serve as Ocugen’s senior biometrics authority, shaping the strategic direction for all biostatistics and statistical programming activities across the R&D portfolio.
• Translate corporate and product development goals into rigorous, operationally executable biometrics strategies that balance scientific defensibility, regulatory compliance, and speed of execution.
• Represent Ocugen as the senior biometrics lead in interactions with the FDA, EMA, and other global health authorities — including BLA/NDA submission meetings, Type A/B/C meetings, and advisory committee preparations.
• Provide authoritative statistical leadership for gene therapy development programs, with expertise in rare disease trial design, small patient populations, longitudinal assessments, and long‑term follow‑up.
• Establish and maintain biometrics standards, SOPs, and governance aligned with GCP, ICH, CDISC, and evolving regulatory expectations for advanced therapies.
• Contribute to R&D governance, portfolio prioritization, and executive-level strategic planning as a member of the senior leadership team.

Biostatistics Leadership — Late-Stage & Regulatory

• Lead statistical design, analysis planning, interpretation, and reporting for all late-stage clinical programs, including Phase 2/3 trials and pivotal studies supporting BLA and NDA submissions.
• Provide scientific leadership for protocol development, ophthalmic endpoint selection (BCVA, LLVA, full-field stimulus testing, ellipsoid zone integrity, microperimetry), sample size determination, randomization, and adaptive design considerations.
• Oversee development, review, and final approval of Statistical Analysis Plans (SAPs) across all programs.
• Serve as the primary statistical resource for Data Monitoring Committees (DMCs), interim analyses, executive-level data reviews, and governance committee presentations.
• Ensure statistical methodologies are scientifically defensible, clinically interpretable, and aligned with current FDA and EMA regulatory expectations for ophthalmic gene therapies.
• Lead statistical innovation efforts, including Bayesian adaptive designs, novel estimands (ICH E9 R1), natural history-informed analyses, and patient-level simulation approaches relevant to rare disease programs.

Statistical Programming Leadership

• Provide strategic and hands‑on operational oversight of all statistical programming activities, including CDISC‑compliant SDTM and ADaM dataset development, TLF production, integrated summaries, and electronic regulatory submission deliverables.
• Establish and enforce programming standards, validation procedures, and quality control processes consistent with inspection readiness and global regulatory compliance.
• Direct oversight of CRO and statistical programming vendors, ensuring quality, timeliness, and regulatory defensibility of all deliverables.
• Champion implementation of automation, modern programming tools, and operational efficiencies to improve quality and execution speed across programs.
• Ensure all programming deliverables meet FDA and EMA eCTD submission requirements, including electronic Common Technical Document formatting standards.

Clinical Data Review & Development Decision Support

• Partner closely with Clinical Development, Clinical Sciences, Medical Affairs, and Regulatory Affairs to interpret emerging clinical and biomarker data and inform program‑level decisions.
• Lead integrated reviews of efficacy, safety, imaging, and functional assessments — including retinal imaging (OCT, fundus), electrophysiology (ERG/mfERG), and visual function measures specific to inherited retinal diseases.
• Present data analyses and strategic biometrics recommendations to C‑suite leadership, Board of Directors, and external governance bodies.
• Serve as an internal expert on the natural history of inherited retinal disease and gene therapy endpoints, providing scientific context for statistical interpretations.

Operational Excellence & Team Development

• Build and develop Ocugen’s internal biometrics function, including hiring, mentorship, and performance development of biostatistics and programming staff.
• Drive accountability for external vendors and CRO partners; establish governance structures for vendor oversight and issue escalation.
• Implement scalable, milestone‑driven processes supporting current late‑stage programs and future pipeline growth.
• Maintain the highest standards of data integrity, quality, and regulatory defensibility across all programs.
• Ensure perpetual inspection readiness in compliance with FDA 21 CFR Part 11, GCP, and global expectations for advanced therapy submissions.

Qualifications

Required

• PhD or MS in Biostatistics, Statistics, Mathematics, or a related quantitative PhD or MS in Biostatistics, Statistics, Biometrics, or a related quantitative discipline; PhD strongly preferred.
• 15+ years of progressive experience in biostatistics and/or statistical programming in biopharmaceutical development, with at least 5 years in a senior leadership role.
• Demonstrated success leading biometrics activities supporting late‑stage clinical development and regulatory submissions — including at least one BLA or NDA filing with FDA or equivalent global submission — with a strong record of successful health authority interactions.
• Deep expertise in ophthalmology clinical development, including ophthalmic endpoints (BCVA, LLVA, FSST, microperimetry, EZ integrity on OCT), retinal imaging assessments, central reading center oversight, and ophthalmology‑specific regulatory submission strategies.
• Experience supporting gene therapy clinical development programs, preferably AAV‑based or other viral vector platforms.
• Expert‑level knowledge of GCP, ICH E9/E9(R1), ICH E6, CDISC standards (SDTM, ADaM, DEFINE‑XML), and FDA/EMA regulatory expectations for advanced therapies.
• Demonstrated experience overseeing external statistical programming vendors and CROs in a late‑stage, submission‑critical environment.
• Exceptional leadership, scientific communication, and cross‑functional influencing skills; proven ability to operate effectively in a lean, agile biotechnology environment.

Preferred

• Experience supporting rare inherited retinal disease (IRD) programs, including natural history‑informed trial design and endpoint selection for diseases such as retinitis pigmentosa, Stargardt disease, or geographic atrophy.
• Familiarity with FDA’s guidance framework for rare disease drug development, including Accelerated Approval, Breakthrough Therapy Designation, and endpoint qualification processes.
• Experience with Bayesian adaptive trial designs, estimand frameworks (ICH E9 R1), and novel statistical methods applicable to small patient populations.
• Experience supporting FDA advisory committee (AdCom) preparations and presentations.
• Prior experience operating as the senior biometrics’ leader within a small‑to‑mid‑stage biotechnology company, including direct involvement in building or scaling an internal biometrics function.

Working Conditions

Incumbent will primarily work Monday – Friday during normal business hours out of our Malvern, PA corporate headquarters. This position operates in an office setting, and the incumbent may require to be available outside of normal hours to handle priority business needs. We do not offer remote work for this role.

Ocugen is an equal opportunity employer. In order to provide equal employment and advancement opportunities to all individuals, we make all of our employment decisions based upon merit, qualifications, abilities, and an individual’s conduct and performance. We will not make any of our decisions, and will not discriminate against any employee or applicant, on the basis of race, color, religion, creed, national origin or ancestry, ethnicity, sex (including pregnancy), gender (including sexual orientation, gender identity and status as a transgender or transsexual individual), age, physical or mental disability, citizenship, past, current or prospective service in the uniformed services, genetic information, or any other characteristic protected from discrimination under the law. Ocugen complies with applicable federal, state and local laws governing nondiscrimination in employment.