Optimizing Rare Disease Trials From An Operational Point Of View

Today’s rare and orphan disease landscape is comprised of close to 6,000 rare diseases affecting a population of approximately 25M people in the U.S. and another 30M people in Europe, although many believe these numbers to be significantly higher.¹ Regulatory developments over the past 40 years have helped to improve the viability and accessibility of rare disease clinical trials, increasing both the ROI for pharmaceutical companies and patient access to treatment.

However, it is important to note that the operational strategy for rare disease trials is markedly more complex than those for traditional pharmaceuticals. The standard responsibilities of maintaining clinical trial protocols, short and long-term schedules and milestones, and project budgets are just a small part of the burden represented by each unique rare disease and its accompanying trial.

The key is to understand the clinical trial from an operational point of view, ensuring that critical challenges are addressed prior to program start to avoid foreseeable delays. By establishing a three-tiered strategy that accounts for 1) clinical trial setup, 2) the implications of multiple global settings, and 3) both physical and virtual site opportunities, pharmaceutical companies, sponsors, CROs, and patients can each better their own ROI in the end result.