Beyond Patient Centricity: What Rare Disease Research Gets Right

Hierarchical composite endpoints analyzed through pairwise comparisons more accurately reflect multifaceted treatment benefit than time-to-first-event composites, but transparent outcome prioritization, patient involvement in ranking, and reporting of Net Treatment Benefit remain underutilized despite their importance to interpretation.

In Episode 3 of The Human Side of Clinical Trials, hosts Brian S. McGowan, PhD, FACEHP, chief learning officer and co-founder of ArcheMedX, Inc., and Kelly Ritch, chief operating officer of ArcheMedX, Inc., speak with Jason Riis, PhD, CEO and chief behavioral scientist at Behavioralize, about why a well-written protocol doesn’t always translate into consistent execution, and how designing for the way people actually think and learn can reduce site burden and the need for costly unlearning.

A new white paper and public engagement process outline the FDA’s commitment to building fit-for-purpose evidentiary standards, stronger patient voice integration, and greater regulatory predictability across rare disease drug development and review.

Real-time clinical trials require standardized digital data capture at the point of care through scaled eSource-EDC-EHR integration, but progress stalls because competing standards lack regulatory enforcement and one-off system integrations cannot scale beyond single studies without industry-wide alignment and legal clarity on data ownership.

In this video interview, Saamir Pasha and Carole Berini, PhD, of Ontada, describe what operationalizing regulatory-grade quality frameworks requires—and why fit-for-purpose evidence generation is a transparency and documentation discipline, not just a data cleanliness exercise.

Trialbee CEO Matt Walz explores the current impact of AI in delivering practical gains in clinical development and reflects on its future potential, where enrollment predictability may be the biggest prize of all.

A biomarker test now decides who enters a precision medicine trial, yet results often go unused and assays are not ready when screening opens.

Real-time RBQM requires proportionate analysis frequency matched to data arrival and issue criticality, not continuous reanalysis, because signal churn from over-frequent runs creates alert fatigue and operational noise that obscures meaningful deterioration.