Your Strategy For Patient-Forward Rare Disease Research

Collectively, rare diseases make up the most challenging area in clinical research. Individually, they present unique hurdles for pharmaceutical companies and CROs.

Poorly understood natural history of disease coupled with widely varying phenotypes often hinders rare disease diagnosis and documentation of treatment impact. Biomarkers are not yet available for many conditions, and end point selection must often be carefully customized, as most conditions have no widely accepted clinical outcomes measures or precedence for trial design. And by virtue of being rare, many of these diseases lack the patient numbers to enable robust recruitment — let alone statistically significant analyses and control groups. With all of these challenges potentially at play in any given rare disease trial, maintaining ethical, efficient trial design and execution is paramount.

When selecting a CRO for your rare disease study, however, a global footprint and fancy biostatistics capabilities are not enough. Worldwide believes that success in rare disease clinical trials stems from recognizing each program’s particular needs and getting the priorities right: Patients come first. Patients come second. And patients come third. Everything else will follow.

We believe in going beyond being patient-friendly, placing patients at the center of it all for truly patient-forward research.

Our eBook explains how to develop creative strategies to plan and operationalize rare disease programs that put the patient first. And second. And third.