Biostatistics

Proven track record from Phase I through Phase IV clinical trials.

Expertise with early‑phase exploratory designs, dose‑finding studies, pivotal Phase II/III trials, and real‑world/post‑marketing analyses

An experienced group of SAS programmers, biostatisticians, and methodologists—primarily senior profiles—providing strong scientific grounding and hands‑on regulatory understanding

Low turnover, ensuring continuity, knowledge retention, and consistent project delivery

ICH guidance and more specifically ICH-E9(R1) EMA and FDA guidance as appropriate
CDISC standards

A biotech sponsor developing a therapy for an ultra‑rare neonatal genetic disease faced a major challenge: extremely limited patient availability and the need to treat newborn's shortly after birth. Demonstrating efficacy while keeping the sample size as small as possible was essential.

Our Approach

Our senior biostatistics team designed a tailored two‑stage Phase I/II trial that minimized patient exposure while preserving statistical robustness. The first stage evaluated safety across two dose levels, and the second confirmed efficacy using the selected dose. Because fewer than 20 infants were expected worldwide, we implemented a Bayesian statistical framework to enable early decisions on futility or efficacy, reducing patient burden and supporting faster progression.

Drawing on extensive experience in rare disease trials, our team aligned the design with regulatory expectations and provided a statistically defensible path forward despite severe sample size constraints. Our methodological guidance ensured that the study remained feasible, ethical, and scientifically credible.

The proposed low‑sample, two‑stage design was positively reviewed by multiple regulatory bodies. The sponsor is now on track to file for conditional approval based on this design—representing a critical milestone in bringing the therapy to patients with this devastating condition.