Fewer than 5% of the more than 10,000 known rare diseases have approved treatments, yet unmet need alone does not move a patient from diagnosis to enrollment. The real obstacle is a cluster of five compounding barriers, and a sponsor that solves only one of them still loses most of its eligible pool to the other four.
The five barriers Medpace identifies are low awareness, protocol complexity, competition for a thin patient population, hesitancy, and logistics. Each one can independently kill enrollment, but they rarely arrive alone. A patient who finds a trial through social media may still drop out because travel reimbursement was unclear at the screening visit. A patient ready to enroll may reconsider when the informed consent document reads like a regulatory filing rather than a plain explanation of what the next 18 months will look like. The FDA’s own guidance on enhancing clinical trial participation calls out the particular pressure rare disease programs face: when the eligible population numbers in the hundreds nationally, a single site that fails to communicate clearly can cost the study months.
The hesitancy barrier deserves specific attention because it is easy to misread. In rare disease, many patients arrived at their current treatment regimen after years of misdiagnosis and failed options. Asking them to interrupt that routine for a placebo-controlled trial is not a neutral ask. Sponsors who treat hesitancy as a messaging problem tend to push clearer brochures; the ones who address it as a trust problem invest earlier in advocacy relationships and give investigators talking points that acknowledge the patient’s history rather than skating past it.
On the logistics side, Medpace’s IntelliPACE and ExcelliPACE model pairs site-selection data with concierge services covering travel, lodging, and caregiver support, recognizing that a willing patient who cannot afford the flight still counts as a dropout. The measure that matters most is not how many patients express interest at screening, but how many complete the protocol after confronting the actual cost and physical demands of participation.
Source link: https://www.medpace.com/blog/overcoming-the-greatest-barriers-in-rare-disease-patient-recruitment/
Moe Alsumidaie, MBA, MSF, is founder and Chief Editor of Vanguard Publications, which publishes Clinical Trial Vanguard, Pharma Vanguard and BullScope, and Head of Research at CliniBiz. He has two decades in clinical trial operations and data science, with earlier roles at Genentech, Abbott Vascular and Stanford University Medical Center, and is a guest lecturer in clinical trial sciences at Rutgers University.
