Prepping For Success: Five Operational Considerations For Rare Disease Programs

By Jeff O’Hagan, Associate Director, Global Project Management, Worldwide Clinical Trials

As a veteran project manager, I’ve spent the majority of my career in operations management, either on the part of the sponsor or on the part of the CRO. I’ve provided expertise or oversight in a number of therapeutic disciplines ranging from cardiovascular to infectious disease to oncology to pain. I’ve participated and managed highly complex projects, such as an oncology program involving five different treatment arms, a stepwise approach for dose increases for maximum tolerability, tedious cohort assignment/management in phase 1 studies, high incidental SAE reporting, and delicate and intricate IP preparation scenarios requiring compounding for infusions, to name a few.

Despite my extensive operational expertise and experience, nothing could prepare me for the complexity of my first rare disease program, studying spinal muscular atrophy (SMA) in young pediatric populations. This project required that I draw upon all my understanding of complex study design, operational delays in study start-up, retention of patients, and, equally important, family engagement. And, all my previous expertise notwithstanding, I came away from the SMA study with five new learnings for managing a rare disease study: