Bringing the Trial to the Patient: Nursing Support for Decentralized Clinical Trials in Pediatric Rare Disease

Recruiting and retaining patients for a clinical trial in pediatric rare disease can be challenging. Patient populations are small, families are geographically dispersed, and the burden of repeated travel to a trial site can stretch families thin over the course of a multi-year study. When a child also faces physical and cognitive challenges, the demands of a traditional site-based trial can become a barrier to participation.

Why nursing is central to a successful decentralized trial

For a decentralized or hybrid trial to support retention without compromising data integrity, every in-home visit needs to feel like an extension of the site, not a workaround. That means protocol-trained nurses, reliable supply and equipment management, and coordination across the sponsor, CRO, central pharmacy, and clinical sites so coordination stays intact between visits. When any one of those pieces is fragmented across vendors, the burden shifts back to the family, and study retention can suffer.

A six-year partnership in pediatric rare disease

Since 2020, Naven Health has provided in-home nursing care, high-touch program management and trial supplies management for a Phase 1/2 pediatric rare disease study, in partnership with the study sponsor, CRO, central pharmacy and clinical sites. The condition affects a small patient population and presents both physical and cognitive challenges, making consistent, in-home support an important part of keeping families engaged in the study.

Every nurse engaged in the study is individually trained on the trial protocol before seeing a patient, so each visit can adhere to the same standards across all 16 subjects. A dedicated program management team provides round-the-clock on-call availability, flexible scheduling support, and coordination across all care delivery partners. Naven also sources, stores, and manages trial supplies, including pumps and temperature monitors, to help support the success of each visit and reduce the risk of protocol deviation. Nurses provide weekly visits of five to six hours in the home, building the kind of consistency that long-duration pediatric studies depend on.

What protocol-driven care at home can deliver

Over six years and 16 unique pediatric patients, the program has delivered more than 2,000 in-home visits, maintained a 94% study retention rate, and recorded only three missed visits attributable to supply management.

For sponsors and CROs supporting rare disease studies, the patient experience between site visits can be a meaningful lever for retention, protocol adherence, and the speed at which a therapy moves through development.

Want to learn more? View our case study here.