Pentara to Present Rare Disease Statistical Innovations at NORD Summit Aligning with FDA’s New Framework Initiative

Posters on Global Statistical Tests, Basket Trials and Natural History Controls address clinical evidence gaps at the center of the FDA’s new regulatory effort.

SALT LAKE CITY, UT, UNITED STATES, October 23, 2026 /EINPresswire.com/ -- Pentara Corporation, an independent biostatistics and regulatory consulting firm, will present research on recovering statistical power in small rare disease trials at the NORD^® Rare Diseases & Orphan Products Breakthrough Summit^®, October 25–27 in Washington, D.C. The presentation follows the U.S. Food and Drug Administration’s launch of its Rare Disease Framework Initiative, which aims to align regulatory expectations with the evidence that small patient populations can realistically generate.

Founder and CEO Suzanne Hendrix, PhD, and Sam Dickson, Senior Vice President, Statistics, will present three posters:

- The 100-to-1 Imbalance: Recovering Statistical Power in Rare Disease Trials Through Global Statistical Tests (GSTs). Highlights that under current trial designs, we are at least 178 times more likely to reject a rare disease treatment that works than to approve one that does not. The research examines how capturing multi-domain efficacy through a single pre-specified GST eliminates "endpoint guesswork" and recovers the power small trials need.

- Statistical Considerations for Assessing Rare Events in Single-Arm Studies. Demonstrates that trials can be significantly smaller and shorter if natural history data is allowed as the comparison group. It describes how leveraging endpoints with low, well-defined background response rates allows smaller cohorts to detect treatment effects when statistical significance barriers are adjusted appropriately.

- Optimizing Power and Sample Size for Biomarker-Driven Basket Trials in Neurodegenerative Diseases. Evaluates how testing several indications under one biomarker-driven protocol can reduce total enrollment and give sponsors a structured basis for selecting which indication advances to Phase 3.

“Standard evidentiary standards severely penalize rare disease patients by passing up treatments that work in order to guard tightly against approving an ineffective treatment," said Suzanne Hendrix, PhD, founder and CEO of Pentara. "The FDA’s new framework is a critical step forward. By adopting Global Statistical Tests to see the whole effect of a treatment, and allowing use of natural history data to run shorter, smaller single-arm studies, we can capture the evidence patients desperately need. These approaches are ready now, and they belong in the framework from the start.”

Attendees can meet the Pentara team at the posters during the Summit. Copies are available on request after the event at pentara.com https://www.pentara.com/contact.

About Pentara
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Pentara Corporation is an independent biostatistics and regulatory consulting firm based in Salt Lake City, Utah. Founded by Suzanne Hendrix, PhD, Pentara specializes in measuring change in slow, heterogeneous, hard-to-measure diseases, with a focus on neurodegenerative conditions including Alzheimer’s disease, Parkinson’s disease, ALS and frontotemporal dementia, as well as rare disease. Its statisticians develop and publish composite endpoints and novel analytic methods, including Global Statistical Tests, built to detect real treatment effects in small and variable patient populations.

Pentara supports sponsors across the clinical development program, from study design and statistical analysis through clinical programming, data management, trial data integrity and fraud detection, medical writing and FDA interaction. Independent of any CRO parent or software platform, Pentara keeps senior statisticians on a program from first protocol to regulatory submission. Learn more at pentara.com.

Brett Walker
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+1 801-361-6600
bwalker@pentara.com
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