Case Study: How a Better Biostatistics Fractional Biostatistician Saved a Sponsor Hundreds of Thousands of Dollars

At a glance

Without the right statistical leadership, this Sponsor might still be stuck in regulatory limbo. Instead, with support from Better Biostatistics, they found a viable, FDA- aligned pathway forward and launched their pivotal trial. This is the value of partnering with experts who understand both statistics and regulatory strategy.

Why It Worked

Better Biostatistics did more than provide statistical calculations. We provided regulatory strategy, negotiation support, and deep FDA insight. A fractional or consulting biostatistician with FDA experience can:

Translate FDA feedback into actionable design options

Propose scientifically defensible alternatives when the Agency’s request isn’t feasible

Prevent stalled development programs due to unresolved design disagreements, saving months (or even years) of lost time

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CHALLENGES

A small, oncology-focused pharmaceutical company was preparing for its pivotal Phase 3 trial. The science was strong, but they repeatedly hit a wall with the FDA. After multiple rounds of feedback, the trial design remained unresolved, putting timelines, funding, and investor confidence at risk.
  • The FDA insisted on a very specific primary endpoint for the Phase 3 trial. The Sponsor strongly disagreed. The drug was not developed for that endpoint, and there was no regulatory precedent for using it in this indication.
  • Despite several attempts, the Sponsor could not convince the Agency to consider alternatives. The development program stalled, delaying trial initiation and jeopardizing the company’s path to approval and future financing.

SOLUTIONS

Better Biostatistics brought in a senior statistical consultant with deep expertise in oncology trial design, direct experience interacting with the same FDA review division, and a clear understanding of how to negotiate scientifically sound alternatives with the Agency. This expert helped:

Analyze and challenge the Agency’s requested endpoint utilizing robust scientific, statistical, and regulatory arguments

Reframe the conversation from resistance to collaboration, helping the Agency focus on patient benefit, feasibility, and interpretability

Develop several alternative endpoint and study design options grounded in clinical relevance and statistical validity, with clear, data-driven justifications for each to present to the FDA

RESULTS

1

The Sponsor gained alignment with the Agency and was then cleared to proceed with their pivotal Phase 3 trial.

2

The development program was unblocked, avoiding indefinite delays or potential trial redesign after initiation.

3

The company preserved investor confidence and maintained its path toward approval.